Ketone Monoester Supplementation in Cystic Fibrosis: A Pilot and Feasibility Study
Phase: NA
Conditions: Cystic Fibrosis
Sponsor: University of Alabama at Birmingham

September 24, 2024 
Ketone Supplementation in Cystic Fibrosis 
NCT 04938726 
To Whom it May Concern, 
Please find the Study Protocol included in this information. 
 
Eric P. Plaisance 
 

IRB ePortfolio
IRB ePortfolio

1. GENERAL QUESTIONS
Protocol Number IRB-300007323
Principal Inves. gator Name Plaisance, Eric P.
Title of proposed project:
Ketone Monoester SupplementaƟon in CysƟc Fibrosis: A Pilot and Feasibility Study
Select the type of applicaƟon you are submiƫng to the IRB for review. ConƟnuing Review
NOTE: The ePorƞolio should NOT be used for ConƟnuing Reviews for protocols not INITIALLY submiƩed on the ePorƞolio. Please see instrucƟons for submiƫng the
appropriate form here.
The OIRB has developed a list of . ps and best pracĀces for IRB submissions. Review the document here for assistance in developing your applicaƟon.
IniƟal IRB Protocol ApplicaƟon
PURPOSE: In non-technical, lay language, provide the purpose of the project. The contents of this secƟon are copied to other areas of IRAP. As such, provide only
the purpose of the project here.
Assess the acceptability and tolerability of an exogenous ketone monoester supplement  and determine its eﬀects on reducing markers of inﬂammaƟon in the lungs and improving
clinical outcomes for paƟents with cysƟc ﬁbrosis. The exogenous ketone that will be used in this study is a commercially available nutriĀonal supplement known as KE4 from
KetoneAid, Inc. Biomarkers of inﬂammaƟon and nutriƟon  will be analyzed in the blood and sputum samples.
BACKGROUND: In 2-3 paragraphs, summarize the past experimental/clinical ﬁndings leading to the design of this project. Include any past or current research that
informed the study design and any previous results that are relevant to understanding the project. Lastly, list the study outcomes that will be measured to evaluate
the purpose of the project . It is not necessary to include methodology in this secĀon. NOTE: Technical terms must be deﬁned in simple language. AbbreviaƟons
must be spelled out. Provide references for any speciﬁc citaƟons.
Our group has invesƟgated the physiological and metabolic responses to a number of exogenous ketone supplements in both rodents and humans. Our
experience and that of our colleagues lead us to the conclusion that exogenous ketone supplements provide an exciƟng approach to increase circulaƟng
ketone concentraƟons. These ﬁndings suggest that administraƟon of exogenous ketones might hold promise in reducing inﬂammaƟon in a number of
chronic disease condiƟons that cause inﬂammaƟon, including CF.
METHODS: Describe the procedures for all aspects of your protocol. Tell us what you are doing.
Participants will be recruited by the Principal Investigator or Study Coordinator from the inpatient and outpatient  CF population at UAB. Following
admission to UAB Hospital for a routine CF exacerbation, or within the outpatient setting, the Study Coordinator will discuss the study with participants
and obtain written informed consent before initiating the study.
Patients will be randomly allocated in unbalanced numbers (2:1) (n = 15 ketone and n = 8 control) to receive a ketone monoester or an identical-tasting
placebo, respectively, for 8 days during the course of hospitalization for treatment of acute pulmonary exacerbation (One, 60 mL bottle of either a ketone
ester will be consumed twice daily  (30 mL or 15.0 g per dose) (KE4, KetoneAid, Falls Church, VA; 15.0 g or placebo (PL, flavored to closely match taste
of the ketone) will be administered following an overnight fast and again at 1500h.
ParƟcipants will be asked to drink about 1 ﬂuid ounce of the ketone or placebo supplement twice a day (once in the morning and once mid-late a晴ernoon)
for up to 7 days. Each day in the study, the parƟcipant  will be asked to fast overnight for about 8 hours before drinking the ﬁrst dose of ketone or placebo.
Following administraƟon of the ﬁrst dose of either the ketone or placebo and on either day 5, 6, or 7 a small drop of blood will be obtained from their
ﬁnger before (Ɵme 0 min) and 15, 30, 60, 90, and 120 min a晴er  drinking the supplement. On days in between visit 1 and visit 2, we will check their blood
sugar before drinking the supplement and 30 minutes a晴er they drink the supplement. We will use a commercially available ketone meter (Keto-Mojo,
Napa, CA) that provides a rapid assessment of  blood ketone and glucose (sugar) levels. The Keto-Mojo device requires a ﬁnger sƟck with a lancet,
idenƟcal to those used to measure blood sugar, and uses only a small drop of blood. PaƟents will be educated on how to use the device and self measure
ketone and blood sugar.
 
Blood sampling will occur from their indwelling catheter or venipuncture  at day 1 and at day 5, 6, or 7 before administraĀon of the ketone or placebo. We
will try to coordinate this with any blood sampling done for clinical care.
 
 The Keto-Mojo device requires a finger stick with a lancet, identical to those used to measure blood sugar, and uses only a small drop of blood. The device
provides values within about 15 seconds following sampling. An alcohol prep will be used to cleanse the surface of the skin prior to each stick and a
bandage will be applied to reduce risk of infection.
Participants will remain on all CF chronic care medications and/or standard exacerbation therapy (intravenous antibiotics and increased airway clearance
throughout the course of the study. The study pharmacist will provide the ketone or placebo supplements by computer-generated random assignment in
blocks of three participants and will have sole access to the randomization code.
Spirometry measurements, CRP, and, history/physical exam and Sputum bacteriology  will be conducted at the beginning of hospitalization as part of
standard of care (these results will be pulled from the medical record for study purposes.  Spirometry measurements and history/physical exams  will also
be performed at the end of hospitalization as part of standard of care and will be pulled from the medical record for study purposes.
For outpatient subjects, these results will be pulled from the medical record and study procedures will also be perfomred at visit 1 and visit as according to
the consent
Spirometry will also be be per formed in addtion to those for standard care just before the participant drinks the ketone supplement or placebo control and
one hour after on day 1 and either day 5, 6 or 7.
Additional sputum and blood will be collected at baseline before the treatment and at patient-discharge for trial-specific readouts.
The parƟcipants will also be asked to complete the CFQR quesƟonnaire and a food diary every night. They will also be asked to complete the symptom
quesƟonnaire on hour a晴er receiving the morning ketone or placebo.
*
*

2. CONTINUING REVIEW
REQUIRED - SELECT ONE OR BOTH: Will this be a retrospecƟve study and/or a prospecƟve study?
Yes 
No
RetrospecƟve
Yes 
No
ProspecƟve
Yes 
No
Will the study involve the prospecƟve collecƟon or analysis of data, documents, or records?
Yes 
No
Will the study involve the prospecƟve collecĀon or analysis of biospecimens?
What is the expected end date of the study (including data analysis)? 01-May-2023
Provide the total number of subjects to be included at all sites, both retrospecƟvely (including records) and prospecƟvely. 25
Provide the total number of subjects to be included at UAB, both retrospecƟvely (including records) and prospecƟvely. 25
Select the status of the Principal InvesƟgator.
Faculty/Staﬀ
Student/Trainee
Yes 
No Are any of the invesƟgators listed on the IRB Personnel Form students using this research for their capstone project, thesis, or dissertaƟon?
Yes 
No Is the project to be conducted internaƟonally?
Procedure List
Yes 
No Does the protocol involve any procedures not described in the above table?
Describe the procedure(s), including whether the procedure(s) are research only or rouƟne.
spirometry- protocol driven
Research Area
Select the type of research.
Biomedical (non-oncology)
Behavioral (non-oncology)
Oncology
Project InformaƟon
StarƟng date of project 01-Sep-2021
Current IRB expiraƟon date 10-Oct-2024
Yes 
No Do any of the invesƟgators have a ﬁnancial conﬂict of interest management plan issued from the Conﬂict of Interest Review Board?
Indicate the status of your study.
Not yet open
Open
On hold
Closed to enrollment
30-Apr-2023
Date enrollment closed
On protocol procedure
In long-term follow-up
*
*
*
*
*
*
Select all study procedures and indicate whether the procedures are research only or rouƟne.
Procedure
Select whether the procedure is research or rouƟne.
 
Blood drawing
Protocol Driven
Biological sampling (other than blood)
Protocol Driven
Placebo
Protocol Driven
RandomizaƟon
Protocol Driven
Pregnancy tesƟng
Protocol Driven
Record review (which may include PHI)
Protocol Driven
Placebo
Protocol Driven
Physical Exam
Protocol Driven
Surveys, quesƟonnaires, or interviews (one-on-one)
Protocol Driven
Diet, exercise, or sleep modiﬁcaƟons
Protocol Driven
Food supplements
Protocol Driven
*

In data analysis only
Completed
Provide the number of subjects currently acƟve in the study (receiving study intervenƟon, acƟve follow-up, etc.). NOTE: Enter 0 if the study only involves secondary
data analysis.
Total number of acĀve parƟcipants.
Provide the number of subjects enrolled since the start of the study by parƟcipant groups, sites and/or sub-study. NOTE: Enter 0 if the study only involves secondary
data analysis.
Total number of parĀcipants enrolled since the start of the study 15
Provide the number of subjects enrolled since the last conƟnuing review by parƟcipant groups and sub-study.
Total number of parƟcipants enrolled since the last conƟnuing review 0
Yes 
No Are there preliminary results from the study?
Provide the preliminary results from the study.
Preliminary analysis suggests that paƟents tolerated the ketone ester well with no adverse eﬀects. We also show that the ketone ester increases circulaƟng ketone
concentraƟons and decreased sputum and some plasma inﬂammatory markers. There were no signiﬁcant eﬀects on pulmonary funcƟon as measured by spirometry,
but paƟents did report an improvement in their percepƟon of respiratory funcƟon as determined by the CFQ-R
Yes 
No
Has there been any new substanƟve literature relevant to the risks, procedures, or intervenƟons of the study?
Yes 
No
Since the last IRB review, has your study received an audit by an internal or external group (e.g., UAB for-cause audit or government enƟty)? NOTE:
This does not include study team self-audit, sponsor monitor visits, or sponsor audits.
Yes 
No Have there been any changes to the study since the last conƟnuing review? If there has been no conƟnuing review, have there been changes to study
acĀviĀes since the iniĀal review?
ProspecƟve
Have any UAB subjects voluntarily withdrawn or been administraƟvely withdrawn since the last IRB review?
Yes 
No
Over the life of the protocol, have there been any Serious Adverse Events (SAEs)/unanƟcipated problems, and/or unanƟciapted problems?
Yes 
No
Over the life of the protocol, have there been any problems NOT meeƟng the UAB criteria for reportable problems but the sponsor requests IRB
noƟﬁcaƟon?
Yes 
No
Have there been any protocol deviaƟons since the last IRB approval, including both previously reported and unreported?
Yes 
No Has there been an IND Annual Report submiƩed to the FDA since the last approval?
Indicate whether any of the following have occurred since the last IRB Approval.
You have had one or more problems obtaining informed consent.
You have received complaints about the research.
If you wish to add any informaƟon for the IRB to consider (e.g., any informaƟon the IRB has not already asked for or the research team has not already provided and
the research team believes will aid the IRB in their review), provide it here.
Yes 
No
Is this a mulĀ-site study?
Provide the number of subjects screened and enrolled at UAB since the last IRB review.
0
Total screened
0
Total enrolled
Complete the table for parƟcipants who are not of Hispanic or LaĀno ethnicity.
Complete the table for parƟcipants who are of Hispanic or LaĀno ethnicity.
ParƟcipant Group/Sub-study/Site
Number AcĀve
 
ParƟcipant Group/Sub-study/Site
Number Enrolled
 
ParƟcipant Group/Sub-study/Site
Number Enrolled Since Last ConƟnuing Review
 
*
*
*
*
*
*
Racial Categories
Female
Male
Unknown/Not Reported
 
White
7
7
Black or African American
1
0
Racial Categories
Female
Male
Unknown/Not Reported
 

5. RESEARCH DETERMINATION
6. NON-AFFILIATED PERSONNEL
7. SPONSORS AND ENTITIES
Complete the table for parƟcipants whose ethnicity is unknown or not reported.
Research DeterminaƟon Form
Yes 
No
Is the acĀvity a systemaƟc invesƟgaƟon?
Yes 
No
Is the acĀvity designed to develop or contribute to generalizable knowledge?
Yes 
No
Does the project involve obtaining informaƟon about living individuals?
Yes 
No
Does the project involve an intervenƟon or an interacƟon with parĀcipants?
Yes 
No
Does the project involve an FDA regulated test arƟcle?
Yes 
No
Is the project deﬁned as a clinical invesƟgaƟon?
Brieﬂy describe the proposed project, including what materials are being obtained and their sources. If you provided this informaƟon on Page 1 (General
QuesƟons), enter "See above."
Spirometry measurements, CRP, and, history/physical exam and Sputum bacteriology will be conducted at the beginning of hospitalizaƟon as part of standard of care
(these results will be pulled from the medical record for study purposes. Spirometry measurements and history/physical exams will also be performed at the end of
hospitalizaƟon as part of standard of care and will be pulled from the medical record for study purposes.
AddiĀonal spirometry, queasƟonaire, sputum and blood will be collected at baseline before the treatment and at paƟent-discharge for trial-speciﬁc readouts
for out paƟents medical history will also be pulled from the medical record
Risk Level - FDA Regulated
REQUIRED: Select whether the project involves minimal risk or greater than minimal risk to parƟcipants.
Greater than Minimal Risk
Sources of Private InformaƟon
Indicate the informaƟon that may be obtained, accessed, used, disclosed, or shared from an individual (living or deceased). Select all that apply.
An individual's geneƟc tests
GeneƟc tests of family members of a parƟcular individual (including an embryo or fetus)
GeneƟc manifestaƟon of a disease or disorder in family members of a parƟcular individual
Any request for, or receipt of, geneĀc services, or parĀcipaĀon in a clinical study which includes geneĀc service, by the individual or any family member of the
individual
EducaƟon records of an individual
Alcohol or substance abuse informaƟon, including diagnosis, treatment, or referral of treatment for alcohol abuse, substance abuse, or chemical dependency
Yes 
No Will the PI or others obtain, review, or make other use of parƟcipants' “protected health informaƟon” (i.e., informaƟon, whether oral or recorded in
any form or medium that (a) is created or received by a health care provider and (b) relates to past, present, or future physical or mental health or
condiƟon of an individual; or provision of health care; or payment for provision of heath care)?
IdenƟﬁable, but not private, informaƟon
Other idenƟfying informaƟon
Yes 
No
Is the project subject to the 2023 NIH Data Management and Sharing (DMS) policy?
Yes 
No
Will informaƟon be received from outside of UAB?
Yes 
No
Will you share the informaƟon with an insƟtuƟon other than UAB?
Yes 
No
Will you need to obtain informaƟon from a department other than your own?
Provide any addiƟonal informaƟon for the IRB to consider. Upload any relevant ﬁles in the AƩachments secƟon.
Non-UAB/CoA Personnel
Yes 
No
Are there any NON UAB, Children's of Alabama (CoA), Lakeshore FoundaƟon, or Birmingham Veteran's Aﬀairs Medical Center (BVAMC) key
personnel on this study? NOTE: UAB, CoA, Lakeshore FoundaƟon, and BVAMC invesƟgators must be listed on the IRB Personnel Form. AddiƟonally, this
page does not apply to adding sites for Single IRB or mulĀ-site research.
Funding, University Contracts, Subcontracts, MTAs, or DUAs
Racial Categories
Female
Male
Unknown/Not Reported
 
*
*
*
*
*
*
*
*
*
*
*
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9. PROSPECTIVE
1. <18
2. 18-89
3. >=90
Yes 
No
Is this project funded in any way?
Yes 
No
Is the funding internal?
Yes 
No Does the project involve any University Contracts, MTAs, DUAs, or subcontracts/subawards? NOTE: Subawards are idenƟﬁed by the OSP Assigned
Number with a three digit suﬃx (e.g., 000500000-001).
Yes 
No Will the project receive non-monetary support (i.e., drugs, devices, services, etc.) from another enĀty?
Ind
Yes 
No
Drugs/Dietary Supplements
Yes 
No
Biologics
Yes 
No
RadiopharmaceuĀcals
Yes 
No
Medical Devices
Yes 
No
In-vivo imaging, image guided intervenĀons (e.g., CT, PET/CT, MRI, PET/MRI, x-ray, DEXA, ﬂuoroscopy, nuclear imaging, ultrasound) or radiaƟon
therapy
Yes 
No Is this a mulĀ-site study?
Yes 
No Does the study involve access, review, or disclosure of Protected Health InformaƟon (PHI) ?
Yes 
No Does the study involve randomizaƟon?
Include the risks and beneﬁts of randomizaƟon in the consent form.
Yes 
No Does the study require clinical services at any of these sites?
Yes 
No Will any of the services be billed to either parĀcpants or to the study account through the Hospital Billing Oﬃce (PFS) or the HSF Billing Oﬃce (MSO)?
Yes 
No Will this study involve direct interacƟon with parƟcipants who have an infecƟous disease?
Target Accrual InformaƟon
35
Approximately how many subjects will need to undergo screening procedures to determine whether they qualify to be enrolled? Enter 0 if not applicable.
NOTE: This number is an approximaƟon based on previously completed projects.
25
How many subjects do you intend to enroll at UAB? Enter 0 if not applicable.
Describe how the screening and enrollment numbers above are determined (e.g., provide a power analysis, explain the pilot data, or reference prior studies.
UAB Hospital admits approximately 70 unique cases each year and has a outpaƟent populaƟon of around 250 paƟents, so we anƟcipate, over the 2-years of funding,
we will have the appropriate number of paƟents available to address our quesƟons.
What are the target age range(s) of the potenƟal subjects with which you will interact?
Indicate which of the following populaƟons you will be targeƟng (enrolling, interacƟng, or intervening with) for your study. Select all that apply.
CogniĀvely impaired adults
Economically or educaƟonally disadvantaged
UAB employees
Fellows
Individuals with Limited English Proﬁciency (LEP) or non-English speakers. Recruitment of study with LEP or non-English speakers is generally required, if the study
holds the prospect of a direct therapeuƟc beneﬁt to the parƟcipant.
Individuals of speciﬁc racial, religious, or ethnic groups
Individuals living outside the 50 US states
Pediatric and neonates
PaƟents (This includes exisƟng paƟents)
Persons who are insƟtuƟonalized
Pregnant women or fetuses
Prisoners
Medical Residents
Study Staﬀ or invesƟgators named on this applicaƟon
Students
Others
None of the above, not targeƟng speciﬁc populaƟons
How will the study team idenƟfy potenĀal subjects? Select all that apply.
Non-UAB physicians
Non targeted/unknown (e.g., chart review)
Other
If chart reviews will be used to idenƟfy potenƟal subjects that are not your own paƟents, request a parƟal waiver of HIPAA AuthorizaƟon for the purpose of
idenƟfying individuals for recruitment.
*
*
*
*
*
*
*

Describe.
ParƟcipants will be recruited by the principal invesƟgator or Study Coordinator from the inpaƟent CF populaƟon at UAB. Dr. Amit Gaggar is a UAB physician and part of
the UAB CF Care team. He is a Sub InvesƟgator on this project and will assist in recruiƟng paƟents.
*Describe parƟcipant inclusion/exclusion criteria, including sex, race/ethnicity, age, and health status.
Inclusion criteria will include diagnosis of CF, age > 19 years, colonizaƟon with Pseudomonas Aeruginosa, and acute pulmonary exacerbaƟon requiring
inpaƟent care or out-paƟents .
Exclusion criteria include concurrent or recent (within 28 days of enrollment) use of corƟcosteroids, acute respiratory failure requiring the use of invasive
or noninvasive venƟlaƟon, chronic liver or renal disease, and pregnancy
Describe the esĀmated Āme commitment of each subject. Examples: 1) One hour once a week for 52 consecuƟve weeks; 2) Twenty minutes to complete a one-Ɵme
survey; 3) One interview lasƟng 60 minutes. Enter "N/A" if parƟcipants are not directly involved.
The parƟcipant involvement will include 2 visits over 7 days of data collecƟon
Yes 
No
Will a CerƟﬁcate of ConﬁdenƟality be obtained from the NIH?
What recruitment materials/methods will be used to recruit subjects. Select all that apply.
Flyer
Where will the ﬂyer be distributed?
UAB CF Clinic
Internet posĀng/website
ResearchMatch
Subject pool/repository
Printed brochure
LeƩer to healthcare providers
LeƩer to potenƟal subjects
Printed ad
Radio/TV ad
Email solicitaƟons
Direct subject contact
Describe the methods used to recruit the subject (i.e., how, when). If parƟcipants without an exisĀng relaƟonship with study personnel will be recruited via
telephone, provide a copy of the required “Phone Script Template for Recruitment of PaƟents from an Electronic Health Record” and complete the IRB Training -
Telephone Recruitment for Human Subject Research ParƟcipants course.
ParƟcipants will be recruited by the principal invesƟgator or Study Coordinator from the inpaƟent and outpateint CF populaƟon at UAB. Dr. Amit Gaggar is a UAB
physician and part of the UAB CF Care team. He is a Sub InvesƟgator on this project and will assist in recruiƟng paƟents. Upon admission, Dr. Gaggar will alert the
research team of the admission and a member of the research team will contact the paƟent to assess interest.
Electronic Medical Record query using i2b2, Electronic Data Warehouse, or IMPACT
Other
*Describe all acƟviĀes to idenĀfy and recruit prospecĀve parĀcipants.
(For assistance with the development of a Recruitment Plan, please contact CRSP.)
Select the types of data collecĀon tools that will be used in this study. Select all that apply.
Survey/quesƟonnaire(s) (UAB recommends using Qualtrics or RedCap for electronic surveys)
Audio or video recordings
Interview guides/scripts
Subject diaries
Mobile or web applicaƟon
Electronic Medical Record query using i2b2, Electronic Data Warehouse, or IMPACT
Other
ProspecĀve Biospecimens
What type of biospecimens will be collected? Select all that apply. NOTE: If blood will be taken for screening and/or enrollment purposes, indicate them speciﬁcally.
AddiĀonally, mulƟple specimens taken from the same person should be counted individually (e.g., 3 parĀcipants with 4 blood draws per parƟcipant equals 12
specimens).
Tissue
Bone marrow aspirates
Blood
How many Ɵmes will blood be collected? 2
Number of biospecimens. 2
Volume of blood per draw.
5-8 ml
Describe the blood collecĀon to include when, how oĀen, how, and volume (in teaspoons and milliliters).
*

addiƟonal blood will be collected at baseline and at visit 2 s for inpaƟents . This will be a single draw of 5-8 ml at two Ɵmepoints.
For outpaĀents blood will be collected at visit 1 and visit 2
Fecal
Semen
Urine
Number of biospecimens. 1
Biopsy
Other
Specify. Include number of biospecimens. NOTE: If mulĀple types specimens (e.g., blood, urine, etc.) are obtained from the same person, they count as one.
If the paƟent can expectorate addiƟonal sputum will be collected at visit 1 and visit 2
How will biospecimens be obtained, processed, distributed, and stored?
Blood samples will be collected from parĀcipants aĀer signing informed consnet. Sampes will be stored and processed in Dr. Plaisance's laboratory (Shelby Hall, 876A)
for subsequent analysis.
How will biospecimens be labeled (e.g., unique idenĀﬁer, medical record number, social security number, name, date of birth)?
Specimens will be labeled with a parĀcipant code and the date of the sample. The parĀcipant code will be a randomly assigned, unique alphanumeric idenĀﬁer
How will clinical data associated with the biospecimens be collected and stored?
Clinical data will be collected and stored using the same alphanumeric idenƟﬁer as described above and will be
entered into a secure database maintained on a ﬁrewall- and password-protected network drive
What parƟcipant idenƟfying informaƟon will be collected and linked to the biospecimens?
Samples will be labeled with a parƟcipant code and the date of the sample. InformaƟon which would reveal the idenĀty of a parƟcipant code
number or link data to an individual will be extremely limited.
What steps will be taken to maximize the conﬁdenƟality of linked idenƟﬁers? For example, procedures could include using a password-protected computer database
to link idenƟﬁers, with limited personnel knowledgeable of the password, or coded idenƟﬁers released without the ability to link to clinical data (also called
“stripped” or “anonymized” biospecimens).
ConﬁdenĀality of all parĀcipants will be fully protected and invesĀgators will only use unique idenĀﬁcaĀon numbers for labeling study samples and for compiling data
ﬁles. Access to parĀcipant data will be strictly limited under control of the
PI. Any publicaĀon of these data will not idenĀfy the parƟcipant by name.
Describe how the above numbers were determined (e.g., provide a power analysis, explain the pilot data, or reference prior studies.
AllocaƟon of parƟcipants in a 2:1 (ketone:placebo control) fashion in blocks of 3 was chosen because this is a pilot study designed to provide preliminary data for an
extramural grant. DescripƟve staƟsƟcs will be calculated for all quanƟtaƟve measures, including assessments of acceptability and tolerability. We will have the
appropriate number of paƟents available to address our quesƟons.
Where are the specimens coming from (i.e., from the parƟcipant, repository, etc.)?
the parĀcipant
Indicate UAB's role. Select all that apply.
Serving as central laboratory
Central biospecimen repository
Other
Yes 
No
Will any geneƟc analyses be performed on the biospecimens?
Yes 
No
Will the biospecimens include remnant material from a clinical procedure that would otherwise be discarded?
Yes 
No
Will the biospecimens include extra material that would NOT have been taken for clinical purposes?
Yes 
No
Are the biospecimens linked during the storage process, a晴er processing?
Yes 
No
Could the study yield clinically relevant informaƟon?
Yes 
No
Will the biospecimens be stored for future research?
What type of study is to be done with the biospecimens?
bio-specicems will be stored and distributed for the study of lung disease only a晴er veriﬁcaƟon of appropriate approval are in place
Describe how and where biospecimens will be stored. NOTE: If biospecimens will be stored in a BVAMC locaƟon, describe what IRB is responsible for overseeing the
operaĀons of the biospecimen bank (i.e., local IRB or other mulĀ-site or central IRB?).
Sampes will be stored  and processed  in Dr. Plaisance's laboratory (Shelby Hall, 876A) for subsequent analysis.
Describe how the privacy of subject and the conﬁdenƟality of data will be protected.
A master subject log will be maintained by the PI. The Log will be maintained on a UAB encrypted computer which are password protected
Who will have access to the biospecimens, including the requirements for access, and who has control of this access?
Only Dr. Plaisance and his team will have access to the database and the samples. Only Dr. Plaisance will oversee distribuƟon and he is responsible to ensure that all
IRB approvals are in place before distribuƟng any samples for future research
Describe the procedures in place for subjects to withdraw their biospecimens or whether deidenƟﬁcaƟon makes subject withdrawal impossible.
Should the parƟcipant wish to withdraw their consent or specimen they can reach out to Dr. Plaisance . His contact informaƟon is provided in the Informed consent.
Yes 
No
Is the banking of the biospecimens opĀonal?
Include in the consent the applicable secƟons of the Storage for Future Use language.
Yes 
No Will biospecimens be shared with other invesƟgators in the future?
Yes 
No
Will the biospecimens be stored indeﬁnitely?
*
*
*
*
*
*
*
*

11. LOCATIONS
12. DEVICES
13. DEVICE SELECTION
14. DRUGS
Describe the methods that will be used to discard specimens at the end of the study life cycle.
A晴er analysis for protocol described outcomes the samples will be banked and stored for future research if the parƟcpant consents. If not they will be destroyed
Yes 
No Will specimens be used and/or shared for commercial proﬁt?
Yes 
No Will parƟcipants be informed of the results of the specimen tesĀng?
Yes 
No Are there any implicaƟons for family members based on specimen tesƟng results?
Clinical Trials
Select whether this protocol meet the deﬁniĀon of a clinical trial.
Clinical Trial 
Non-clinical trial 
This protocol must be registered on clinicaltrials.gov. Provide the NaƟonal Clinical Trial (NCT) idenĀﬁer. NCT04938726
NCT idenƟﬁers are required for IRB approval of a clinical trial; however, if all other informaƟon is complete and accurate, pending NCT idenƟﬁers can be obtained in
parallel with the IRB's review of the submission.
All key personnel must complete Good Clinical PracƟces (GCP) training.
InsƟtuƟonal Biosafety CommiƩee (IBC)
Yes 
No
Does this project involve gene transfer, recombinant DNA, or CAR T cells?
Ind
UAB Hospital
UAB Hospital - Highlands
The Kirklin Clinic of UAB Hospital
The Kirklin Clinic at Acton Road
UAB Callahan Eye Hospital
UAB Clinical Research Unit
Children's of Alabama (CoA)
Jeﬀerson County Department of Health (JCDH)
Jeﬀerson County Department of Health (JCDH)
Other (i.e., any performance site not listed above, including those covered by subawards related to this protocol)
Yes 
No
Is this a ﬁeld study?
Ind
Comparison
Randomized
Open Label
Control
Other
Yes 
No
Is the trial blinded?
Is the trial single or double blinded? Double Blinded
Add a Device Review Sheet for each device the study involves.
Drugs, Biologics, and Supplements
What phase is the project? NOTE: If the drug is not being studied directly (i.e., an anxiolyƟc given to reduce anxiety before an MRI), state "Drug is not being studied
directly."
proof of concept pilot trial
Yes 
No
Will this study involve the use of radioisotopes managed by the UAB Radiopharmacy?
*
*
*
*
*
Device Name
Device Review Sheet
 
Keto Mojo
DEVICE REVIEW SHEET Complete
*

15. DRUG SELECTION
18. PATIENTS
25. COST AND PAYMENT
26. RISKS
Add a Drug Review Sheet for each drug the study involves.
Pa
mi
Yes 
No
Will the invesƟgators (any invesƟgator listed as key personnel) be recruiƟng their own paƟents?
Provide the raƟonale for including paƟents of invesƟgators.
ParƟcipants will be recruited by the principal invesƟgator or Study Coordinator from the inpaƟent and outpaƟent CF populaƟon at UAB . Dr. Amit Gaggar is a UAB
physician and part of the UAB CF Care team. He is a Sub InvesƟgator on this project
What safeguards are in place to ensure that invesƟgators are not unduly coercing their paƟents into parƟcipaƟon?
PaƟents will be consented by trained study team members and will not be pressured. Voluntary parƟcipaƟon will be emphasized to all parƟcipants
Cost, Reimbursement, or CompensaƟon
Yes 
No
Will subjects (or their insurance providers) experience costs associated with any of the procedures, drugs, biologics, devices, tests, or any aspect of
their parĀcipaĀon in the study?
Yes 
No
Will subjects receive any reimbursement or compensaƟon for their parƟcipaƟon (compensaƟon should not be an amount that could be considered
coercive or create undue inﬂuence)?
Select whether parĀcipants will receive payment and/or reimbursement.
Payment
Provide the payment details, including amount, frequency of payment (e.g., $20 at visits one, two, and three), and the method of payment (e.g., cash, check, direct
deposit, etc.).
The parƟcipant will receive $300.00. We will use the UAB greenphire system or checks to deliver the study payment.
NOTE: If subjects are being paid for their parƟcipaƟon (not reimbursed for study-related expenses) in an amount of $600 or more in a single calendar year, their
informaƟon will be reported to the IRS in accordance with federal requirements. This must be clearly stated in the informed consent form.
Reimbursement
Risks and Risk MinimizaĀon
Determine any anƟcipated risks or potenƟal discomforts experienced by subjects for this study. Select all that apply.
Physical risks (e.g., pain, bruising, and infecƟon associated with venipuncture, adverse reacƟons to drugs, muscle soreness and pain as a consequence of exercise
tesƟng, heart aƩack induced by maximal exercise test, radiaƟon risk (e.g., x-ray, CT scan, radiaƟon therapy, radioisotopes, ﬂuoroscopy))
Provide details of the risks, including expected frequency and severity.
ParƟcipants may experience some side eﬀects from taking the study supplement. This includes gastrointesƟnal distress such bloaƟng, belching, or nausea. These side
eﬀects are usually mild and may only last up to a few hours.
Describe any steps taken to miƟgate the expected risks.
The described side eﬀects are mild in nature and do not present any health concerns. However if a paƟent should experience concerning discomfort they will be
evaluated by the study doctor.
Psychological risks (e.g., depression and confusion as a result of administraƟon of drugs, feelings of guilt precipitated by a sensiƟve survey)
Social risks (e.g., invasion of privacy, breach of conﬁdenƟality, loss of community standing)
Economic risks (e.g., loss of employment, loss of potenĀal monetary gain)
Other risks
Indicate how safety is being monitored in this study.
Data Safety Monitoring Board (DSMB)
Independent medical monitor
Data will be monitored by PI.
Describe how safety will be monitored locally and, if this is a mulƟ-center study, how data and safety will be monitored across sites. AddiƟonally, describe how oĀen
data will be monitored. If the only risk is breach of conﬁdenƟality, descrbe the method of monitoring for breaches.
Drug Name
Drug Review Sheet
 
Ketone ester
DRUG REVIEW SHEET Complete
*
*
*

27. BENEFITS
28. PRIVACY AND CONFIDENTIALITY
The study PI will evaluate all adverse events from each enrolled parƟcipant as they occur in order to ensure subject safety.
Data will not be monitored.
Describe.
The parƟcipant may or may not experience beneﬁts from parƟcipaƟng. The potenƟal beneﬁts include a reducƟon of inﬂammaƟon in the lungs and blood which could
improve health outcomes for the parƟcipant.
What are the potenĀal beneﬁt(s) of the study (e.g., beneﬁts to society, increased knowledge of the parĀcular disease, increased scienĀﬁc knowledge, etc.)?
The potenƟal beneﬁts include a reducƟon of inﬂammaƟon in the lungs and blood which could improve health outcomes for the parĀcipant.
Select the idenƟﬁers.
Names
All geographical subdivisions smaller than a state, including street address, city, county, precinct, zip code, and their equivalent geocodes, except for the iniƟal
three digits of a zip code, if according to the current publicly available data from the Bureau of the Census: (1) The geographic unit formed by combining all zip
codes with the same three iniĀal digits contains more than 20,000 people; and (2) The iniĀal three digits of a zip code for all such geographic units containing
20,000 or fewer people is changed to 000
All elements of dates (except year) for dates directly related to an individual, including birth date, admission date, discharge date, date of death; and all ages over
89 and all elements of dates (including year) indicaƟve of such age, except that such ages and elements may be aggregated into a single category of age 90 or
older
Phone Numbers
Fax Numbers
Email Addresses
Social Security Numbers
Medical Record Numbers
Health plan beneﬁciary numbers
Account numbers
CerƟﬁcate/license numbers
Vehicle idenƟﬁers and serial numbers, including license plate numbers
Device idenƟﬁers and serial numbers
Web universal resource locators (URLs)
Internet protocol (IP) addresses
Biometric idenĀﬁers, including ﬁnger and voice prints
Full face photographic images and any comparable images
Any other unique idenĀfying number, characterisƟc, or code
Medical history
Surgical history
Laboratory results
Other
Provide the length of Ɵme the idenƟﬁers will be stored or retained.
one year past study close out.
Describe how data will be collected, recorded, and shared, including the speciﬁc safeguards to protect the conﬁdenƟality of data. Select all that apply.
Working with an enƟty outside of UAB to collect, record, share, or otherwise uƟlize the data.
Data will be stored in REDCap.
Data will be stored in ShareFile
Data will be kept on a UAB encrypted device (i.e., computer, tablet, etc.).
Data will be stored on a UAB encrypted server.
Data will be stored in the UAB Cheaha Supercomputer
Data will be recorded on paper.
Data will be coded.
Yes 
No
Will a key to the code be maintained?
*

29. CONSENT
Who will have access to the key?
THe PI
Data will be kept in a locked oﬃce/ﬁling cabinet.
Data will be stored on a password protected computer/drive not maintained by UAB.
Data will be stored in a locaƟon not described above.
Data will NOT be coded.
Describe the plan to destroy the subject idenƟﬁers at the earliest opportunity consistent with the conduct of research, unless retenƟon is required for reasons of
health, research, or law (including the methods that will be used to discard data at the end of the study life cycle). NOTE: Research records must be retained and
subsequently discarded in accordance with applicable regulatory guidelines.
HIPAA AuthorizaƟon
Indicate whether you will obtain HIPAA AuthorizaƟon, a HIPAA waiver, whether the data meet the speciﬁcaƟons for a Limited Data Set, or whether the study does not
involve access, review, or disclosure of Protected Health InformaƟon (PHI). Select all that apply.
Request for parƟal HIPAA waiver for recruitment/screening purposes.
HIPAA AuthorizaĀon will be obtained for some or all of the subjects.
NOTE: Ensure the appropriate HIPAA authorizaƟon language has been added to the consent form.
All
Will HIPAA authorizaĀon be obtained from all or some of the subjects?
Yes 
No
Will the study team ensure HIPAA authorizaƟon is obtained in a private seƫng?
Request for HIPAA waiver for some or all of the subjects (e.g., for retrospecƟve review of PHI, and/or to review PHI for recruitment purposes and obtain HIPAA
authorizaƟon during enrollment)
The data meet the speciﬁcaƟons for a limited data set. NOTE: A limited data set will only apply if you receive data from an outside source as a limited data set or if
you are sending data to an outside source as a limited data set (in which case, documentaƟon of authorizaƟon or a Waiver of AuthorizaƟon will be required). A
Data Use Agreement (DUA) is required for this opƟon.
HIPAA Covered EnƟƟes
Indicate which of the enƟƟes would provide health informaƟon for this protocol, maintain health informaƟon as it was collected for this protocol, and/or store health
informaƟon a晴er it has been collected for this protocol.
UAB Hospital or UAB Hospital - Highlands
The Kirklin Clinic of UAB Hospital or Acton Road (and/or associated clinics)
UAB Callahan Eye Hospital
Children's of Alabama
Jeﬀerson County Department of Health
School of DenĀstry
School of Health Professions
School of Medicine
School of Nursing
School of Optometry
University of Alabama Health Services FoundaĀon
Valley FoundaƟon
Medical West - UAB Health System Aﬃliate
Birmingham Veteran's Aﬀairs Medical Center
Yes 
No Is the principal invesƟgator requesƟng that the UAB IRB waive paƟent HIPAA authorizaƟon from another insƟtuƟon or enƟty (e.g., insurance company,
collaboraƟng insƟtuƟon)?
Informed Consent
Indicate all that apply to the informed consent process.
Informed consent with wriƩen documentaƟon will be obtained from all or some of the subjects.
All
Will wriƩen informed consent be obtained from all or some of the subjects?
Describe how wriƩen informed consent will be obtained, including conﬁrmaĀon that a copy of the signed consent document will be given to the individual who will
sign it.
Consent will be obtained in a private seƫng by trained research staﬀ and a signed copy will be provided to the parƟcipant.
Describe how the study team will ensure the subjects understand the informaƟon presented.
*
*
Select all types of consent forms that will be used.
Consent Form Types
DescripƟon of Document (50 words or less)
Clean Copy
Tracked Copy
 
*

31. ATTACHMENTS
The team will ask frequent quesƟons to the parƟcipant during the consent process to ensure they understand
What language will the prospecƟve parƟcipant understand?
English
What language will be used to obtain consent?
English
Yes 
No Will any potenƟal parƟcipants be, or have been, in a stressful, painful, or drugged condiƟon before or during the consent process?
Request a Waiver of DocumentaƟon of Informed Consent (e.g., subject may not sign an informed consent document, but will be given an informaƟon sheet)
Request a Waiver or AlteraƟon of some or all elements of informed consent or a FULL waiver of informed consent.
AƩachments
Updated By: Eric P. Plaisance @ 24-Sep-2024 12:26:21 PM
University of Alabama at Birmingham
Oﬃce of the IRB
Phone: 205-934-3789 | Fax: 205-934-1301
www.uab.edu/irb
QuesƟons?
Form Version: 2.0 | Date: 01/04/2021
Document Name
Document Type
DescripĀon of Document (50 words or
less)
Clean
Copy
Tracked
Copy
 
Protocol Oversight Review
Form
IRB Submission
Form
Data CollecƟon Sheet
Data CollecƟon
Response Memorandum
Other
Response Memorandum
Other
Survey/QuesƟonnaire
Other
CFQR
Survey/QuesƟonnaire
Other
symptom quesƟonnaire
Survey/QuesƟonnaire
Other
Food Diary
Flyer
Other
ﬂyer
DSMB Charter
Other
DSMP plan

DEVICE SELECTION
Devices
Device Name Keto Mojo
Yes 
No Is an InvesƟgaƟonal Device ExempƟon (IDE) required?
Yes 
No IDE Exempt
Select the applicable IDE Exempt categor(ies).
Category 1: A legally marketed device when used in accordance with its labeling.
Provide jusƟﬁcaƟon for the device meeƟng this category
This meter is commercially available without a prescripƟon
Category 2: A diagnosƟc device if it complies with the labeling requirements in §809.10(c) and if the tesĀng: a) is
noninvasive; b) does not require an invasive sampling proedure that presents signiﬁcant risk; c) does not by design or
intenƟon introduce energy into a subject; and d) is not used as a diagnosƟc procedure without conﬁrmaƟon by another
medically established diagnosƟc product or procedure. AddiƟonal guidance for in vitro diagnosƟc device studies can be
found here.
Category 3: Consumer preference tesƟng, tesƟng of a modiﬁcaƟon, or tesƟng of a combinaƟon of devices if the device(s)
are legally marketed device(s) [that is, the devices have an approved PMA, cleared Premarket NoƟﬁcaƟon 510(k), or are
exempt from 510(k)] AND if the tesƟng is not for the purpose of determining safety or eﬀecƟveness and does not put
subjects at risk.
Category 4: A device intended solely for veterinary use.
Category 5: A device shipped solely for research with laboratory animals and contains the labeling "CAUTION – Device for
invesƟgaƟonal use in laboratory animals or other tests that do not involve human subjects.
Yes 
No Is the device a Humanitarian Use Device (HUD)?
Yes 
No Has the FDA made a device study determinaƟon?
Submit any applicable documentaƟon from the FDA.
Yes 
No Are you requesƟng the IRB make a Nonsigniﬁcant Risk determinaƟon for this device?
Describe the plan for the storage, dispensing, and disposal of the device being studied. Note: If the device is not
FDA-approved, the plan must include methods to segregate the invesƟgaƟonal devices from other devices available for
general use.
The ketone meter is mulƟ use and will be kept by the research coordinator, this devise is not given out to paƟents
Describe how safety will be monitored for this device.
There is no safety monitoring for this device
Refer to the following FDA guidances for helpful informaƟon and
examples.
1) Frequently Asked QuesƟons About Medical Devices
2) Signiﬁcant Risk and Nonsigniﬁcant Risk Medical Device Studies
3) In Vitro DiagnosƟc Device (IVD) Studies - Frequently Asked QuesƟons
Updated By: Eric P. Plaisance @ 24-Sep-2024 12:26:21 PM
Device Review Sheet
Device Review Sheet
DescripĀon
Document
 

University of Alabama at Birmingham
Oﬃce of the IRB
Phone: 205-934-3789 | Fax: 205-934-1301
www.uab.edu/irb
QuesƟons?
Form Version: 2.0 | Date: 01/04/2021

DRUG SELECTION
Drugs
Drug name exogenous ketone ester
Yes 
No Is an InvesƟgaƟonal New Drug ApplicaƟon (IND) required?
Yes 
No Has the requirement for an IND been waived?
Yes 
No Has the drug received FDA Approval?
Provide the name of the person holding the IND.
NA This a supplement and does not require FDA approval
Submit a copy of the Release of Drugs for Human Research Use (UAB Pharmacy (FOR 217), Children's of Alabama (FOR 218).
From where will the drugs be obtained?
A company called Ketone Aid will provide the blinded supplement or placebo. The inpaƟent parƟcipants will receive the
ketone supplement and placebo from the pharmacy but the outpaƟent parƟcipants will receive the ketone supplement and
placebo from the research staﬀ.
Yes 
No Do you have an invesƟgator's brochure available?
Describe the plan for the storage, dispensing, and disposal of the drug(s)/biologic(s).
For the outpaƟent parĀcipants, the PI will store, monitor and dispense the blinded product to the research staﬀ for
dispensing to the paƟent per the randomizaƟon code. The PI will maintain a product accountability log.
For the inpaĀent parĀcipants, the research pharmacy will store and dispense the supplement to the parĀcipant. The research
pharmacy will maintain a product accountability log for the inpaƟents.
Yes 
No Is this a combinaƟon therapy (e.g. an invesƟgaƟonal product in combinaƟon with commercially approved
agent(s), two or more commercially approved agents are being combined, etc.)?
Describe the route of administraƟon.
oral
Yes 
No Is the drug commercially available?
The drug will be used for: A new indicaƟon, new populaƟon, new route of administraƟon, or new dosage
Explain.
Evidence exist that ketone supplement decrease systemic inﬂammaƟon
Describe how safety will be monitored for this drug.
Adverse Events will be monitored throughout the course of the study and the team will meet weekly to discuss paƟent safety
Updated By: Eric P. Plaisance @ 24-Sep-2024 12:26:21 PM
University of Alabama at Birmingham
Oﬃce of the IRB
Phone: 205-934-3789 | Fax: 205-934-1301
www.uab.edu/irb
QuesƟons?
Form Version: 2.0 | Date: 01/04/2021
Drug Review Sheet
Drug Review Sheet

Appendix 1
EForm Name: IRB EPORTFOLIO
Page:
31. ATTACHMENTS
Section:
Question:
Clean Copy
File Name:
PORF_210614.docx

SCHOOL OF EDUCATION
Human Studies
1150 10th Ave S
 BIRMINGHAM AL 35294-0110
phone: 205.996.7909  
www.uab.edu
Institutional Review Board
Protocol Oversight Review Form
Date Submitted to IRB: 06/14/2021
Title of Project: Ketone monoester supplementation in cystic fibrosis: A pilot and feasibility study
Name of Principal Investigator: Eric P. Plaisance
Signature of Principal Investigator: 
School: University of Alabama at Birmingham
Department: Human Studies
Division: School of Education
Review Process (as determined by Department Chair or Dean):
School Review
Divisional Review (Division Director or Designate)
Center or Departmental Protocol Review Committee Review
Project Review Panel (PRP)—Appointed by the Department Chairman or Division 
Director (PRP report attached)
I have reviewed the proposed research and concluded that the following apply:

The research is scientifically valid and is likely to answer the scientific question; 

The researcher and the study team are qualified and/or credentialed to conduct the 
procedures proposed; 

The researcher has identified sufficient resources in terms of experienced research 
personnel, facilities, and availability of medical or psychological services that may be 
necessary as a consequence of participation in the research to protect the research 
participants.
Name of Official: Dr. Michelle Robinson    Title: Interim Dean
(type or print)
Signature: 
Date: _06/10/21_________________

Appendix 2
EForm Name: IRB EPORTFOLIO
Page:
31. ATTACHMENTS
Section:
Question:
Clean Copy
File Name:
DATA SHEET.210614.doc

            
Version Date: 06/14/21
 Pre / Post Assessment (Circle)
Physiological Data Sheet   
P A R T I C I P A N T  I N F O R M A T I O N
Name
ID
Today's Date
           /            /  
Sex
Age
Height
Weight
               yrs
                  inches                        lbs
Medications "On Board" / Special Precautions
KETONE CONCENTRATIONS
Time
BHB
Glucose
Comments
    0 
  15
  30
  60
  90
120
PULMONARY FUNCTION TESTING
Time
Comments
FEV1
FVC
FEV1/FVC
Notes:

Appendix 3
EForm Name: IRB EPORTFOLIO
Page:
31. ATTACHMENTS
Section:
Question:
Clean Copy
File Name:
response.210827.doc

470 Administration Building
701 20th Street South
Birmingham, AL 35294-0104
205.934.3789 | Fax 205.934.1301 | irb@uab.edu
DETERMINATION LETTER
TO:
Plaisance, Eric P.
University of Alabama at Birmingham Institutional Review Board
Federalwide Assurance # FWA00005960
IORG Registration # IRB00000196 (IRB 01)
IORG Registration # IRB00000726 (IRB 02)
FROM:
IORG Registration # IRB00012550 (IRB 03)
DATE:
25-Aug-2021
IRB-300007323
RE:
Ketone Monoester Supplementation in Cystic Fibrosis: A Pilot and Feasibility Study
The IRB reviewed the Initial Application submitted on 05-Aug-2021 for the above referenced 
project. The review was conducted in accordance with UAB’s Assurance of Compliance 
approved by the Department of Health and Human Services.
Type of Review:
Full - Institutional Review Board 02 (UAB)
Determination:
Additional information required
Determination Date: 18-Aug-2021
Please respond to the items listed below. The response must be submitted through IRAP. Visit 
the IRAP page on the UAB IRB website for guidance on responding through IRAP. Click on “How 
to Respond to an IRB Request for Changes”. 
Items to Address: 
1. All key personnel involved in the research must complete the initial IRB training (4 credit requirement), 
then continuing IRB training (refresher) every 3 years. Please check the IRB web site at 
https://www.uab.edu/research/home/irb-training-page for more information.
The following individuals must complete continuing IRB training: 

Amit Gaggar 

2. Make sure the e-Personnel Form includes all research staff that will be working on this study including 
Amit Gaggar and Respiratory Therapists. Amit Gaggar has been removed from the protocol and will be 
added when he completes his training.  All respiratory therapist  and everyone else on the IRB has 
current training.
The following pertains to the e-Portfolio:
3. SECTION 1 GENERAL QUESTIONS, Background 
a. Add a description of what will be analyzed in the blood (5-8mls) and sputum for research 
purposes. Mentioning biomarkers of inflammation will suffice. added
The following pertains to the consent form: 
4. In the Purpose of the Research Study, add a description of what will be analyzed in the blood (5-8mls) 
and sputum for research purposes. Mentioning biomarkers of inflammation will suffice. added
5. In the bullets for Sputum and Blood Collection in the Study Participation & Procedures section, it 
states that each of these biospecimens will be collected for research twice (during days 1-3 and 5-7). 
The IRB thinks this may be confusing for participants. Revise the section to state collection will take 
place once on day 1, 2, or 3, and then another sample collected on days 5, 6, or 7. done
6. In the Information for Women of Childbearing Potential, Nursing Mothers, and/or Men Capable of 
Fathering a Child section, remove the third paragraph since the study agent (ketones) is expected to be 
cleared from their systems within a few hours after ingestion. This appears to have been a “cut and 
paste” error from another consent document. done
7. In the third sentence of the Payment for Participation section, revise the sentence to the following: 
“The total payment you will receive is $300.00.” done
The IRB or designated reviewer must review your response submission before you receive formal IRB 
approval. No activity related to this project may occur until the Office of the IRB issues the IRB Approval Letter 
and, if applicable, any IRB-stamped consent forms.
RESPONDING TO COMMENTS FROM THE OIRB IN IRAP
ONLY RESPOND WHEN ALL ITEMS ARE COMPLETE, INCLUDING OUTSTANDING TRAINING

Navigate to the project record in IRAP and open the record.

The project will open to the Submissions folder. The submission for which revisions are required will 
have a blue “Respond” hyperlink. Click that link to create a Response submission.

After clicking Respond, a new window will open. Click the drop down, in which the only option will 
be Response to Info/Mod Request. Choose Response to Info/Mod Request and click Save.

The Response submission will then be created, which will include a copy of the previously 
completed IRB ePortfolio.

Generate your response memo
o
Save this document as “response.yymmdd”
o
Type in your response in the designated areas using the formatting provided (bold text, 
italics).
o
Use full sentences and responses; avoid “completed”, especially when addressing the 
question differently than requested, when questions are asked or clarity is needed.

o
If adding any additional changes that were not requested, add them to the end of your 
response memo by inserting a new row and using bold and italics for your response.
o
Upload this file with the other revised documents in the applicable sections of the IRB 
ePortfolio
o
NOTE: Failure to provide a response memo may result in a delay in your project and an 
additional request for its provision.

Make any requested changes to the IRB ePortfolio.

Replace attachments that require revisions.

Revise the needed documents or gather missing documents:
o
Use MSOffice’s tracked changes functionality.
o
Submit both a tracked version of the document and a clean version. NOTE: Failure to provide 
a tracked version may result in a delay in your project and an additional request for its 
provision.
o
Save the revised file by changing the YYMMDD suffix and adding “.tracked” or “.clean” to any 
document changed.
o
Do not save files as PDFs. PDFs should only be submitted when the document requires a 
signature or is not available in its native format.  All clean and tracked versions should be 
submitted in the format in which they were created if at all possible.
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Upload the newly revised documents.
 
 

Appendix 4
EForm Name: IRB EPORTFOLIO
Page:
31. ATTACHMENTS
Section:
Question:
Clean Copy
File Name:
response.210831.doc

470 Administration Building
701 20th Street South
Birmingham, AL 35294-0104
205.934.3789 | Fax 205.934.1301 | irb@uab.edu
ADMINISTRATIVE PRE-REVIEW
TO:
Plaisance, Eric P.
University of Alabama at Birmingham Institutional Review Board
Federalwide Assurance # FWA00005960
IORG Registration # IRB00000196 (IRB 01)
IORG Registration # IRB00000726 (IRB 02)
FROM:
IORG Registration # IRB00012550 (IRB 03)
DATE:
30-Aug-2021
IRB-300007323
RE:
Ketone Monoester Supplementation in Cystic Fibrosis: A Pilot and Feasibility Study
The Office of the IRB staff has completed the administrative pre-review of the Response to 
Info/Mod Request submitted on 27-Aug-2021 for the above referenced project. Please respond 
to the items listed below. The response must be submitted through IRAP. 
Visit the IRAP page on the UAB IRB website for guidance on responding through IRAP. Click on 
the “Quick Step by Step Instructions for How to Respond to an IRB Review”. 
 
 
Due to Dr. Gaggar's role in the study, we cannot issue formal approval until his training is 
completed.  Once his training is completed, revise the Personnel eForm and attach a copy of his 
training where indicated (training certificate) section of the Personnel eForm.  Also include the 
degrees for each person listed. Dr. Gaggar has been added and his training certificate has been 
uploaded.  All degrees have been added as well.  Thanks! 
Let me know if you have any questions.
Thanks,
Margie Lawson (mlawson@uab.edu)

Appendix 5
EForm Name: IRB EPORTFOLIO
Page:
31. ATTACHMENTS
Section:
Question:
Clean Copy
File Name:
Queastionaire(CFQR).220110.pdf

 
 
 
 
 
C y sti c
C y sti c
Fi br o si s
F o u n d ati o n
Fi br o si s
F o u n d ati o n
R
2 0 0 0, Q uitt n er, M o di, W atr o us a n d M ess er.  R evis e d 2 0 0 2.  C F Q- R T e e n/ A d ult, E n glis h V ersi o n 2. 0 
P a g e 2 
 
A d olesce nts a n d A d ults
( P atie nts 1 4 Ye ars Ol d a n d Ol der)
 
CY S TI C F I B R O SI S Q U E S TI O N N AI R E - R E VI S E D  
  
 
 
 
D uri n g t he p ast  t w o wee ks, t o w h at exte nt h ave y o u h a d diffic ulty: 
A l ot of 
diffic ult y 
S o me 
diffic ult y 
A little 
diffic ult y 
N o 
 diffic ult y 
  1.  Perf or mi n g vi g or o us acti vities s uc h as r u n ni n g or pla yi n g s p orts .....................  

 

 

 

 
  2.  W al ki n g as fast as ot hers ...................................................................................  

 

 

 

 
  3.  Carr yi n g or lifti n g hea v y t hi n gs s uc h as b o o ks, gr oceries, or sc h o ol ba gs ........  

 

 

 

 
  4.  Cli m bi n g o ne flig ht of stairs ..............................................................................  

 

 

 

 
  5.  Cli m bi n g stairs as fast as ot hers .........................................................................  

 

 

 

 
 
D uri n g t he p ast t w o wee ks , i n dic ate h o w ofte n: 
Al w a ys  
Ofte n  
S o meti mes  
Ne ver 
  6.  Y o u felt well ....................................................................................................... 

 

 

 

 
  7.  Y o u felt w orrie d .................................................................................................. 

 

 

 

 
  8.  Y o u felt useless ................................................................................................... 

 

 

 

 
  9.  Y o u felt tire d ....................................................................................................... 

 

 

 

 
1 0. Y o u felt e ner getic ................................................................................................ 

 

 

 

 
1 1. Y o u felt e x ha uste d .............................................................................................. 

 

 

 

 
1 2.   Y o u felt sa d ......................................................................................................... 

 

 

 

 
Ple ase circle t he n u m ber i n dic ati n g y o ur a ns wer.  Ple ase c h o ose o nly o ne a ns wer f or e ac h q uesti o n. 
T hi nki n g a b o ut t he st ate of y o ur he alt h over t he l ast  t w o wee ks:  
  
 
1 3.  T o w hat e xte nt d o y o u ha ve diffic ult y wal ki n g? 
1.  Y o u ca n wal k a l o n g ti me wit h o ut getti n g tire d 
2.  Y o u ca n wal k a l o n g ti me b ut y o u get tire d 
3.  Y o u ca n n ot wal k a l o n g ti me b eca use y o u get tire d q uic kl y 
4.  Y o u a v oi d wal ki n g w he ne ver p ossi bl e beca use it’s t o o tiri n g f or y o u 
1 4.  H o w d o y o u feel a b o ut eati n g? 
1.  J ust t hi n ki n g a b o ut f o o d ma kes y o u feel sic k 
2.  Y o u ne ver e nj o y eati n g 
3.  Y o u are s o meti mes a ble t o e nj o y eati n g 
4.  Y o u are al wa ys a ble t o e nj o y eati n g 
1 5.  T o w hat e xte nt d o y o ur treat me nts ma ke y o ur dail y life m ore diffic ult?  
 
1.  N ot at all 
2.  A little 
3.  M o deratel y 
4.  A l ot 
 
 
 
Secti o n II. Q u alit y of Life  
Ple ase c hec k t he b o x i n dic ati n g y o ur a ns wer . 

 
 
 
 
 
C y sti c
C y sti c
Fi br o si s
F o u n d ati o n
Fi br o si s
F o u n d ati o n
R
2 0 0 0, Q uitt n er, M o di, W atr o us a n d M ess er.  R evis e d 2 0 0 2.  C F Q- R T e e n/ A d ult, E n glis h V ersi o n 2. 0 
P a g e 3 
 
A d olesce nts a n d A d ults
( P atie nts 1 4 Ye ars Ol d a n d Ol der)
 
CY S TI C F I B R O SI S Q U E S TI O N N AI R E - R E VI S E D  
  
 
1 6.  H o w m uc h ti me d o y o u c urre ntl y s p e n d eac h da y o n y o ur treat me nts? 
1.  A l ot 
2.  S o me 
3.  A little 
4.  N ot ver y m uc h 
1 7.  H o w diffic ult is it f or y o u t o d o y o ur tr eat me nts (i ncl u di n g me dicati o ns) eac h da y? 
1.  N ot at all 
2.  A little 
3.  M o deratel y 
4.  Ver y 
1 8.  H o w d o y o u t hi n k y o ur healt h is n o w? 
1.  E xcelle nt 
2.  G o o d 
3.  Fair 
4.  P o or 
Ple ase select a b o x i n dic ati n g y o ur a ns wer. 
T hi nki n g a b o ut y o ur he alt h d uri n g t he p ast  t w o wee ks, i n dic ate t he 
exte nt t o w hic h e ac h se nte nce is tr ue or f alse f or y o u.  
Ver y 
tr ue 
S o me w h at 
tr ue 
S o me w h at
f alse 
Ver y 
f alse 
1 9.  I ha ve tr o u ble rec o veri n g after p h ysical eff ort ...................................................... 

 

 

 

 
2 0.  I ha ve t o li mit vi g or o us acti vities s uc h as r u n ni n g or pla yi n g s p orts .................... 

 

 

 

 
2 1.  I ha ve t o f orce m yself t o eat .................................................................................. 

 

 

 

 
2 2 . I ha ve t o sta y at h o me m ore t ha n I wa nt t o ............................................................ 

 

 

 

 
2 3. I feel c o mf orta ble disc ussin g m y ill ness wit h ot hers ............................................. 

 

 

 

 
2 4.  I t hi n k I a m t o o t hi n .............................................................................................. 

 

 

 

 
2 5.  I t hi n k I l o o k differe nt fr o m ot hers m y a ge ........................................................... 

 

 

 

 
2 6.  I feel ba d a b o ut m y p h ysical a p peara nce .............................................................. 

 

 

 

 
2 7.  Pe o ple are afrai d t hat I ma y be c o nta gi o us ........................................................... 

 

 

 

 
2 8.  I get t o get her wit h m y frie n ds a l ot ....................................................................... 

 

 

 

 
2 9.  I t hi n k m y c o u g hi n g b ot hers ot hers ...................................................................... 

 

 

 

 
3 0.  I feel c o mf orta ble g oi n g o ut at ni g ht..................................................................... 

 

 

 

 
3 1.  I ofte n feel l o nel y .................................................................................................. 

 

 

 

 
3 2.  I feel healt hy ......................................................................................................... 

 

 

 

 
3 3.  It is diffic ult t o ma ke pla ns f or t he f ut ure (f or e xa m ple, g oi n g t o c olle ge, getti n g 
marrie d, a d va nci n g i n a j o b, etc.) ......................................................................... 

 

 

 

 
3 4.  I lea d a n or mal life ................................................................................................ 

 

 

 

 
. 

 
 
 
 
 
C y sti c
C y sti c
Fi br o si s
F o u n d ati o n
Fi br o si s
F o u n d ati o n
R
2 0 0 0, Q uitt n er, M o di, W atr o us a n d M ess er.  R evis e d 2 0 0 2.  C F Q- R T e e n/ A d ult, E n glis h V ersi o n 2. 0 
P a g e 4 
 
A d olesce nts a n d A d ults
( P atie nts 1 4 Ye ars Ol d a n d Ol der)
 
CY S TI C F I B R O SI S Q U E S TI O N N AI R E - R E VI S E D  
  
 
 
Q uesti o ns 3 5 t hr o u g h 3 8 are a b o ut sc h o ol, w or k, or ot her d aily t as ks. 
3 5.  T o w hat e xte nt di d y o u ha ve tr o u ble kee pi n g u p wit h y o ur sc h o ol w or k, pr ofessio nal w or k, or ot her dail y acti vities d uri n g t he pa st 
t w o wee ks?   
1.  Y o u ha ve ha d n o tr o u ble kee pi n g u p 
2.  Y o u ha ve ma na ge d t o kee p u p b ut it’s bee n diffic ult 
3.  Y o u ha ve bee n be hi n d 
4.  Y o u ha ve n ot bee n a ble t o d o t hese acti vities at all 
3 6.  H o w ofte n were y o u a bse nt fr o m sc h o ol, w or k, or u na ble t o c o m plete dail y acti vities d uri n g t he last t w o wee ks beca use of y o ur 
ill ness or treat me nts? 
  
           Al wa ys  
      Ofte n                      S o meti mes                   Ne ver         
3 7. H o w ofte n d oes C F get i n t he wa y of m eeti n g y o ur sc h o ol, w or k, or pers o nal g oals  
                  Al wa ys  
             Ofte n         
        S o meti mes        
  Ne ver       
3 8 . H o w ofte n d oes C F i nterfere wit h getti n g o ut of t he h o use t o r u n erra n ds s uc h as s h o p pi n g or g oi n g t o t he ba n k? 
                         Al wa ys  
             Ofte n         
        S o meti mes        
  Ne ver       
 
 
 
 
 
I n dic ate h o w y o u h ave bee n feeli n g d uri n g t he p ast  t w o wee ks.  
A gre at de al 
S o me w h at  
A little  
N ot at all 
3 9. Ha ve y o u ha d tr o u ble gai ni n g wei g ht? ...................................................... 

 

 

 

 
4 0. Ha ve y o u bee n c o n geste d? ........................................................................ 

 

 

 

 
4 1. Ha ve y o u bee n c o u g hi n g d uri n g t he da y? ................................................. 

 

 

 

 
4 2. Ha ve y o u ha d t o c o u g h u p m uc us? ............................................................ 

 

 

 

 
 
 
 
 
G o t o 
Q uesti o n 4 4 
4 3.  Has y o ur m uc us bee n m ostl y:    Clear   Clear t o yell o w   Yell o wis h- gree n   Gree n wit h traces of bl o o d    D o n't k n o w 
H o w ofte n  d uri n g t he p ast  t w o wee ks: 
Al w a ys  
Ofte n  
S o meti mes  
Ne ver 
4 4.  Ha ve y o u bee n w heezi n g? ......................................................................... 

 

 

 

 
4 5.  Ha ve y o u ha d tr o u ble breat hi n g? ............................................................... 

 

 

 

 
4 6.  Ha ve y o u w o ke n u p d uri n g t he ni g ht beca use y o u were c o u g hi n g? .......... 

 

 

 

 
4 7.  Ha ve y o u ha d pr o ble ms wit h gas? ............................................................. 

 

 

 

 
4 8.  Ha ve y o u ha d diarr hea? ............................................................................. 

 

 

 

 
4 9.  Ha ve y o u ha d a b d o mi nal pai n? .................................................................. 

 

 

 

 
5 0.  Ha ve y o u ha d eati n g pr o ble ms? ................................................................. 

 

 

 

 
Ple ase be s ure y o u h ave a ns were d all t he q uesti o ns.  
 
Secti o n III. Sc h o ol, W or k, or D ail y Acti vities  
Secti o n I V. S y m pt o m Diffic ulties  
P le ase select a b o x i n dic ati n g y o ur a ns wer. 
T H A N K Y O U F O R Y O U R C O O P E R A TI O N!  

 
 
 
 
C y sti c
C y sti c
Fi br o si s
F o u n d ati o n
Fi br o si s
F o u n d ati o n
R
Q uitt n er, M o di, W atr o us a n d M ess er, 2 0 0 0. R evis e d 2 0 0 2.  C F Q- R — P ar e nt, E n glis h V ersi o n 2. 0 
P a g e 1  
 
P are nts/ C are gi vers ( C hil dre n A ges 6 t o 1 3)  
 
                               C Y S TI C F I B R O SI S Q U E S TI O N N AI R E -  R E VI S E D  
  
U n dersta n di n g t he i m pact of y o ur c hil d’s ill ness a n d treat me nts o n his or her e ver y da y life ca n hel p y o ur healt hcare tea m kee p trac k 
of y o ur c hil d’s healt h a n d a dj ust his or  her treat me nts.  F or t his reas o n, we have de vel o pe d a q ualit y of life q uesti o n naire s p ecificall y 
f or pare nts of c hil dre n wit h c ystic fi br osis.  W e t ha n k y o u f or y o ur willi n g ness t o c o m p lete t his q uesti o n naire.   
 
I nstr ucti o ns: 
T he  f oll o wi n g  q uesti o ns  are  a b o ut  t he  c urre nt  state  of  y o ur  c hil d’s  healt h,  as  he  or  s he  percei ves  it.  T his 
i nf or mati o n will all o w us t o better u n dersta n d h o w he or s he feels i n e ver y da y life. 
 
Please a ns wer all t he q uesti o ns. T here are n o ri g ht or wr o n g a ns wers!  If y o u are n ot s ure h o w t o a ns wer, c h o ose 
t he res p o nse t hat see ms cl osest t o y o ur c hil d's sit uati o n. 
 
 
 
A. 
W hat is y o ur c hil d’s date of birt h? 
Date 
 
 
 
 
 
 
 
 
  
M o  
Da y  
Year 
 
B. 
W hat is y o ur relati o ns hi p t o t he c hil d?   
 

 M ot her 
 

 Fat her 
 

 Gra n d m ot her 
 

 Gra n dfat her 
 

 Ot her relati ve 
 

 F oster m ot her 
 

 F oster fat her 
 

 Ot her ( please descri be) 
       _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ 
 
C.  W hic h of t he f oll o wi n g best descri bes y o ur  
       c hil d's racial or et h nic bac k gr o u n d?  
 

  Ca ucasia n 
 

  Africa n A merica n 
 

  His pa nic 
 

  Asia n/ Orie ntal or Pacific Isla n der 
 

  Nati ve A merica n or Nati ve Alas ka n 
 

  Ot her ( please descri be) 
_ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ _ 
 

  Prefer n ot t o a ns wer t his q uesti o n 
 
D.    D uri n g t he p ast t w o wee ks , has y o ur c hil d bee n o n      
       vacati o n or o ut of sc h o ol f or reas o ns N O T  relate d t o his  
       or her healt h? 
 
E. 
W hat is y o ur  date of birt h? 
Date 
 
 
 
 
 
 
 
 
  
M o  
Da y  
Year 
 
F.   W hat is y o ur c urre nt marital stat us? 
 

  Si n gle/ ne ver marrie d 
 

  Marrie d 
 

  Wi d o we d 
 

  Di v orce d 
 

  Se parate d 
 

  Re marrie d  
 

  Wit h a part ner 
 
G. 
W hat is t he hi g hest gra de i n sc h o ol y o u ha ve c o m plete d? 
 

  S o me hi g h sc h o ol or less 
 

  Hi g h sc h o ol di pl o ma/ G E D 
 

  V ocati o nal sc h o ol  
 

  S o me c olle ge 
 

  C olle ge de gree 
 

  Pr ofessi o nal or gra d uate de gree 
 
 
H.  W hic h of t he f oll o wi n g best descri bes y o u c urre nt w or k  
      stat us? 
 
       See ki n g W or k 
       W or ki n g f ull or part ti me (eit her o utsi de t he h o me or  
  
     at a h o me- base d b usi ness) 
       F ull ti me h o me ma ker 
       N ot w or ki n g d ue t o m y healt h 
       N ot w or ki n g f or ot her reas o ns
 

 Yes 

 N o 
 
 
 
Secti o n I. De m o gr a p hics  
P le ase fill i n t he i nfor m ati o n or c hec k t he b o x i n dic ati n g  yo ur a ns wer . 

 
 
 
 
C y sti c
C y sti c
Fi br o si s
F o u n d ati o n
Fi br o si s
F o u n d ati o n
R
Q uitt n er, M o di, W atr o us a n d M ess er, 2 0 0 0. R evis e d 2 0 0 2.  C F Q- R — P ar e nt, E n glis h V ersi o n 2. 0 
P a g e 2  
 
P are nts/ C are gi vers ( C hil dre n A ges 6 t o 1 3)  
 
                               C Y S TI C F I B R O SI S Q U E S TI O N N AI R E -  R E VI S E D  
  
 
Ple ase i n dic ate h o w y o ur c h il d h as bee n feeli n g d uri n g t he p ast t w o wee ks by c hec ki n g 
t he b o x m atc hi n g y o ur res p o nse. 
T o w h at exte nt h as y o ur c hil d h a d diffic ulty:  
A l ot of 
diffic ult y 
S o me 
diffic ult y 
A little 
diffic ult y 
N o 
 diffic ult y
  1.  Perf or mi n g vi g or o us acti vities s uc h as r u n ni n g or pla yi n g s p orts ........................ 

 

 

 

 
  2.  W al ki n g as fast as ot hers ...................................................................................... 

 

 

 

 
  3. Cli m bi n g stairs as fast as ot hers ............................................................................ 

 

 

 

 
  4.  Carr yi n g or lifti n g hea v y o bjects s uc h as b o o ks, a sc h o ol ba g, or bac k pac k ........ 

 

 

 

 
  5.  Cli m bi n g se veral fli g hts of stairs .......................................................................... 

 

 

 

 
Ple ase c hec k t he b o x m atc hi n g y o ur res p o nse.  
D uri n g t he p ast  t w o wee ks, i n dic ate h o w ofte n y o ur c hil d:  
 
Al w a ys  
Ofte n  
S o meti mes  
Ne ver 
  6.  See me d ha p p y ....................................................................................................... 

 

 

 

 
  7. See me d w orrie d .................................................................................................... 

 

 

 

 
  8. See me d tire d ......................................................................................................... 

 

 

 

 
  9. See me d s h ort-te m pere d .......................................................................................... 

 

 

 

 
1 0. See me d well .......................................................................................................... 

 

 

 

 
1 1. See me d gr o uc h y ................................................................................................... 

 

 

 

 
1 2. See me d e ner getic .................................................................................................. 

 

 

 

 
1 3.   W as  a bse nt  or  late  f or  sc h o ol  or  ot her acti vities  beca use  of  his/ her  ill ness  or  
treat me nts 
… … … … … … … … … … … … … … … … … … … … … … … … … … … … ….. 

 

 

 

 
Ple ase circle t he n u m ber i n dic ati n g y o ur a ns wer.  Ple ase c h o ose o nly o ne a ns wer f or e ac h q uesti o n. 
T hi nki n g a b o ut t he st ate of y o ur c hil d’s he alt h over t he p ast  t w o wee ks, i n dic ate: 
 
1 4. T he e xte nt t o w hic h y o ur c hil d partici pate d i n s p orts a n d ot her p h ysical acti vities, s uc h as g y m class 
1.  Has n ot partici pate d i n p h ysical acti vities 
2.  Has partici pate d less t ha n us ual i n s p orts  
3.  Has partici pate d as m uc h as us ual b ut wit h s o me diffic ult y 
4.  Has bee n a ble t o partici pate i n p h y sical acti vities wit h o ut a n y diffic ult y 
 
 
1 5. T he e xte nt t o w hic h y o ur c hil d has diffic ult y wal ki n g 
1.  He or s he ca n wal k a l o n g ti me wit h o ut getti n g tire d 
2.  He or s he ca n wal k a l o n g ti me b ut gets tire d 
3.  He or s he ca n n ot wal k a l o n g ti me, b eca use he or s he gets tire d q uic kl y 
4.  He or s he a v oi ds wal ki n g w he ne ver p ossi bl e, beca use it’s t o o tiri n g f or hi m or her 
 
 
 
 
Secti o n II. Q u alit y of Life  

 
 
 
 
C y sti c
C y sti c
Fi br o si s
F o u n d ati o n
Fi br o si s
F o u n d ati o n
R
Q uitt n er, M o di, W atr o us a n d M ess er, 2 0 0 0. R evis e d 2 0 0 2.  C F Q- R — P ar e nt, E n glis h V ersi o n 2. 0 
P a g e 3  
 
P are nts/ C are gi vers ( C hil dre n A ges 6 t o 1 3)  
 
                               C Y S TI C F I B R O SI S Q U E S TI O N N AI R E -  R E VI S E D  
  
 
Ple ase c hec k t he b o x t h at m atc hes y o ur res p o nse t o t hese q uesti o ns . 
T hi nki n g a b o ut y o ur c hil d’s st ate of he alt h d uri n g t he p ast  t w o wee ks, i n dic ate t he exte nt t o w hic h e ac h se nte nce is tr ue or f alse f or 
y o ur c hil d:  
 
Ver y 
tr ue 
S o me w h at 
tr ue 
S o me w h at 
f alse 
Ver y 
f alse 
1 6. M y c hil d has tr o u ble rec o veri n g after p h ysical eff ort ................................................. 

 

 

 

 
1 7.   Mealti mes are a str u g gle .............................................................................................  

 

 

 

 
1 8 .  M y c hil d’s treat me nts get i n t he wa y of his/her acti v ities .......................................... 

 

 

 

 
1 9. M y c hil d feels s mall c o m pare d t o  ot her ki ds t he sa me a ge ........................................ 

 

 

 

 
2 0. M y c hil d feels p h ysicall y differe nt fr o m ot her ki ds t he sa me a ge .............................. 

 

 

 

 
2 1.   M y c hil d t hi n ks t hat he/s he is t o o t hi n ........................................................................ 

 

 

 

 
2 2. M y c hil d feel s healt h y ................................................................................................ 

 

 

 

 
2 3. M y c hil d te n ds t o be wit h dra w n ................................................................................. 

 

 

 

 
2 4. M y c hil d lea ds a n or mal life ....................................................................................... 

 

 

 

 
2 5. M y c hil d has less f u n t ha n us ual ................................................................................. 

 

 

 

 
2 6.   M y c hil d has tr o u ble getti n g al o n g wit h ot hers ..........................................................  

 

 

 

 
2 7. M y c hil d has tr o u ble c o nce ntrati n g ............................................................................ 

 

 

 

 
2 8. M y  c hil d  is  a ble  t o  kee p   u p  wit h  his/ her  sc h o ol  w or k  or  s u m mer  acti vities  (e. g. 
ca m p) ......................................................................................................................... ........ 

 

 

 

 
2 9.   M y c hil d is n ot d oi n g as well as us ual i n sc h o ol or s u m mer acti vities (e. g. ca m p) .... 

 

 

 

 
3 0.  
 
M y  
c hil d  
s pe n ds  
a  
l ot  
of  
ti me  
o n  
his/ her  
treat me nts 
e ver y da y … … … … … … … … … …. 

 

 

 

 
Ple ase circle t he n u m ber i n dic ati n g y o ur a ns wer.  Ple ase c h o ose o nly o ne a ns wer f or e ac h q uesti o n. 
3 1. H o w diffic ult is it f or y o ur c hil d t o d o his/ he r treat me nts (i ncl u di n g medicati o ns) eac h da y?   
1.  N ot at all 
2.  A little 
3.  M o deratel y 
4.  Ver y 
 
3 2.  H o w d o y o u t hi n k y o ur c hil d’s healt h is n o w? 
1.  E xcelle nt 
2.  G o o d 
3.  Fair 
4.  P o or 
 
 
 

 
 
 
 
C y sti c
C y sti c
Fi br o si s
F o u n d ati o n
Fi br o si s
F o u n d ati o n
R
Q uitt n er, M o di, W atr o us a n d M ess er, 2 0 0 0. R evis e d 2 0 0 2.  C F Q- R — P ar e nt, E n glis h V ersi o n 2. 0 
P a g e 4  
 
P are nts/ C are gi vers ( C hil dre n A ges 6 t o 1 3)  
 
                               C Y S TI C F I B R O SI S Q U E S TI O N N AI R E -  R E VI S E D  
  
Secti o n III. S y m pt o m Diffic ulties  
 
 
 
T he ne xt set of q uesti o ns is desi g ne d t o deter mi ne t he fre q ue ncy wit h w hic h y o ur c hil d h as cert ai n res pir at ory .diffic ulties, s uc h as 
c o u g hi n g or s h ort ness of bre at h.                                                                                                                                                                               
 
Ple ase i n dic ate h o w y o ur c hil d h as bee n feeli n g d uri n g t he p ast  t w o wee ks. 
A gre at 
de al 
S o me w h at  
A little  
N ot at all 
3 3. M y c hil d ha d tr o u ble gai ni n g wei g ht .............................................................. 

 

 

 

 
3 4. M y c hil d was c o n geste d.................................................................................. 

 

 

 

 
3 5. M y c hil d c o u g he d d uri n g t he da y ................................................................... 

 

 

 

 
3 6. M y c hil d ha d t o c o u g h u p m uc us .................................................................... 

 

 

 

  
 
G o t o  
Q uesti o n 3 8
3 7.  M y c hil d’s m uc us has bee n m ostl y:  
 Clear       Clear t o yell o w      Yell o wis h- gree n   
 
 Gree n wit h traces of bl o o d        D o n't k n o w 
D uri n g t he p ast  t w o wee ks: 
Al w a ys  
Ofte n  
S o meti mes  
Ne ver 
3 8. M y c hil d w heeze d ........................................................................................... 

 

 

 

 
3 9. M y c hil d ha d tr o u ble breat hi n g ....................................................................... 

 

 

 

 
4 0. M y c hil d w o ke u p d uri n g t he ni g ht beca use he/s he was c o u g hi n g ................. 

 

 

 

 
4 1. M y c hil d ha d gas ............................................................................................. 

 

 

 

 
4 2. M y c hil d ha d diarr hea ..................................................................................... 

 

 

 

 
4 3. M y c hil d ha d a b d o mi nal pai n ......................................................................... 

 

 

 

 
4 4. M y c hil d has ha d eati n g pr o ble ms .................................................................. 

 

 

 

 
Ple ase be s ure y o u h ave a ns were d all t he q uesti o ns. 
 
 
 
 
T H A N K Y O U F O R Y O U R C O O P E R A TI O N!  

Appendix 6
EForm Name: IRB EPORTFOLIO
Page:
31. ATTACHMENTS
Section:
Question:
Clean Copy
File Name:
queastionaire(symtom)220220.docx

ID CODE __________ 
Date __________
Page 1 of 2
SYMPTOM QUESTIONNAIRE
How is your general health today? (Please check one box)
        Very Good
    Good
Fair
Poor
Please record any symptoms you may have had today (tick one box for each symptom experienced)
Absent
I did not have this symptom at all
Mild
I had this symptom occasionally, but it didn’t really bother me
Moderate
I had this symptom often, it bothered me quite a bit
Severe
I had this symptom very often, it bothered me a great deal
Absent
Mild
Moderate
Severe
Nausea 
Vomiting
Heartburn 
Stomach Pain 
Diarrhea 
Constipation 
Bloating/Feeling
of Fullness 
Belching 
If you experienced any of the above symptoms, at what point after supplementation did they occur?
        Immediately
          One hour
Two hours
Three or more hours
Nausea 
Vomiting
Heartburn 
Stomach Pain 
Diarrhea 
Constipation 
Bloating/Feeling
of Fullness 
Belching 

ID CODE __________ 
Date __________
Page 2 of 2
If you experienced any of the above symptoms, how long did they last? If applicable, please specify the 
number of times the symptom occurred.
        Less than 30 min
          One hour
Two hours
Three or more hours
Nausea 
Vomiting
Heartburn 
Stomach Pain 
Diarrhea 
Constipation 
Bloating/Feeling
of Fullness 
Belching 
Final Day Question (only). Which study condition do you think you were in?
                     Placebo condition (I did not drink the ketones)
       Ketone Condition (I did drink the ketones)
Additional Comments: 
_____________________________________________________________________________________
_____________________________________________________________________________________
_____________________________________________________________________________________
_____________________________________________________________________________________
_____________________________________________________________________________________
_____________________________________________________________________________________
_____________________________________________________________________________________
_____________________________________________________________________________________
_____________________________________________________________________________________
_____________________________________________________________________________________
_____________________________________________________________________________________
_____________________________________________________________________________________

Appendix 7
EForm Name: IRB EPORTFOLIO
Page:
31. ATTACHMENTS
Section:
Question:
Clean Copy
File Name:
queastionaire (DAILY FOOD RECORD)220220.doc

D a i l y  F o o d  R e c o r d

RECORD EVERYTHING YOU EAT AND DRINK INCLUDING SNACKS AND 
BEVERAGES.  

RECORD IMMEDIATELY AFTER FOOD IS CONSUMED

INDICATE PORTION SIZES.  MEASURE AMOUNTS OF EACH FOOD USING 
MEASURING CUPS OR SPOONS WHEN IT IS PRACTICAL.  RECORD PORTION 
SIZES IN GRAMS, OUNCES, CUPS, TABLESPOONS, TEASPOONS, OR PIECES.  
(example: 8 oz. orange juice, 1 piece wheat bread, 1 tbsp. butter)

INDICATE THE BRAND NAME. (3 oz. Ruffles BBQ Potato Chips, 1 cup Uncle Ben’s 
Long Grain Rice, McDonald’s Large French Fries)

INDICATE FORM OF PURCHASE. (fresh, frozen, canned, etc.)

RECORD TIME OF DAY MEAL WAS EATEN

RECORD AND CHECK THE NUMBER OF SERVINGS FOR EACH ITEM LISTED 
ST= Starch (bread, pasta, cereal, rice, etc.)
MT= Meat (poultry, beef, fish, eggs, nuts) 
V=Vegetable 
FR= Fruit 
D= Dairy (milk, yogurt, cheese, etc.) 
FT= Fat (butter, oil)
B= Beverage (regular soft drinks, sweet tea, sports drinks, etc.)
Please be as specific and thorough as possible with the 
dietary information you provide.  Thank You!

Time
Food
Quantity
Exchange

Appendix 8
EForm Name: IRB EPORTFOLIO
Page:
31. ATTACHMENTS
Section:
Question:
Clean Copy
File Name:
flyer.220525.pdf

We are seeking volunteers to participate in a study to test whether a
ketone ester supplement can reduce inflammation in the lungs for
patients with cystic fibrosis.
Cystic Fibrosis
Research Study
Diagnosed with cystic fibrosis
>19 years old
Concurrent or recent use of
corticosteroids
Acute respiratory failure
requiring invasive or
noninvasive ventilation
Chronic liver or renal disease
Pregnancy
Inclusion criteria:
Exclusion criteria:
Reduction of inflammation in
the lungs and blood
Improved health outcomes
Potential benefits:
Compensation:
Participants will receive $300.00
upon full completion of this 7-day
study. 
If you are interested in participating in this study, please contact:
Jonathan Bergeron
Email: jbergeron@uabmc.edu
Phone: (205)-638-2220

Appendix 9
EForm Name: IRB EPORTFOLIO
Page:
31. ATTACHMENTS
Section:
Question:
Clean Copy
File Name:
KETONE CF DSMP_17June2022.docx

Data safety monitoring plan for: Ketone monoester supplementation in cystic fibrosis: A pilot
and feasibility study
Principal Investigator: Eric P. Plaisance, Ph.D.
Grant Application: Pilot and Feasibility 
Introduction
This randomized controlled trial (RCT) aims to evaluate the effectiveness of a commercially 
available ketone supplement on pulmonary function and markers of inflammation and metabolic 
health in patients with cystic fibrosis (CF) admitted for acute pulmonary exacerbation and 
a cohort of stable outpatient CF subjects. To accomplish this goal, a 5-7 day RCT will be 
conducted to compare the effects of ketone supplementation to a placebo control. The 
primary outcome will be differences in pulmonary function, namely FEV1.0, FVC, and 
FEV1.0/FVC. Secondary outcomes will include quantitative assessment of circulating insulin, 
glucose, and a number of inflammatory markers associated with the NLRP3 inflammasome. 
Qualitative assessment of tolerability and the feasibility of delivery will also be extensively 
examined.  Compared to placebo control and standard care, we hypothesize that the 
ketone ester (KE4, KetoneAid, Falls Church, VA) will produce greater reductions in 
markers of inflammation and improvements in pulmonary function. We further hypothesize 
that ketone supplementation will be well-tolerated and prove to be feasible as it relates to 
administration and consumption of the ketone.  Of note, this Ketone supplement is considered 
GRAS and does not require FDA IND or FDA oversight.
The intervention and measurement protocols pose minimal risk to participants (as 
described below). Because of this low-risk status, the data safety monitoring plan (DSMP) 
for this trial focuses on close oversight by Medical Monitor, Dr. Bryan Garcia. We 
will also provide prompt reporting of excessive adverse events and any serious adverse events 
to the Cystic Fibrosis Center, IRB at the University of Alabama at Birmingham (UAB) and 
NIH. The data safety monitoring plan (DSMP) outlined below will adhere to the protocol 
approved by the UAB IRB.
Safety reports will be sent to the study statistician, the MPIs, and to medical oversight at the 
schedule specified below in the table. The project coordinator will be responsible for 
assembling the data and producing these reports, as well as assuring that all parties obtain 
copies of these reports. The frequency of data review for this study differs according to the 
type of data and can be summarized in the following table:
Data type
Frequency of review
Subject accrual (adherence to protocol regarding
demographics, inclusion/exclusion)
After every 5 patients that complete the
study
Adverse event rates (e.g. gastrointestinal distress 
or dizziness)
As they occur
(cumulative reports also reviewed quarterly)
Serious Adverse Events
Immediate reporting (within 24 hrs) to IRB
Compliance to treatment
After every 5 patients that complete the 
study

Qualifications and responsibilities of Medical/Safety Oversight
Dr. Bryan Garcia will serve as medical monitor for this study.  Dr. Garcia is an Assistant 
Professor of Medicine at UAB.  He has conducted numerous CF-related clinical trials and has 
extensive experience serving as a Co-Investigator for NIH-funded studies.  As Medical 
Monitor, Dr. Garcia will review the reports sent by the project coordinator (at the frequency 
outlined above) to determine whether there is any corrective action, trigger of an ad hoc 
review, or stopping rule violation that should be communicated to the study investigator, UAB 
IRB, and NIH.
Measurement and reporting of participant accrual, adherence to inclusion/exclusion 
criteria and site performance Review of the rate of participant accrual and adherence to pre-
specified inclusion/exclusion criteria will occur after the recruitment of every five patients 
throughout the study. During this phase, the target is to enroll approximately five patients 
per quarter.  No specific subgroups will be tracked.  We envision a high retention rate as this 
study will be conducted in an inpatient setting and will have close follow-up in the outpatient 
cohort. The quarterly review will assess this enrollment rate as well as to assure that 
participants meet eligibility criteria as outlined in the grant proposal. To accomplish this, the 
study statistician will oversee the preparation of data tables summarizing recruitment and 
enrollment status and demographic characteristics. In the event that participant accrual rates 
fall below projected levels, the study investigators will convene a conference call to discuss 
potential strategies to modify the recruitment, screening, and/or enrollment protocols for 
future recruitment efforts and will report any requested changes to the UAB IRB, 
medical/safety officer, and NIH.
Data integrity: All data will be collected by the study coordinator, Jonathan Bergeron, and 
inputted into Excel Redcap on secure UAB network.  This data can be converted to other 
statistical analysis software.  Relational logic checks, such as out-of-range values and 
internal inconsistencies, will be developed and automatically run in parallel with data entry. 
Double-data entry of study outcomes will be used to further minimize and detect entry errors. 
Statistical reports will be generated and include: 1) total number of participants screened, 
consented, and randomized on study entry and follow-up, and 2) a summary of demographic 
and baseline characteristics. This data will be password protected and only accessible by the PI 
and study coordinator. 
 
Confidentiality: Every effort will be made to maintain the confidentiality of participant data by 
the investigator and the Institutional Review Board (IRB) to the extent permitted by law. 
All participants will be assigned subject ID numbers so personal health information (PHI) will 
not be used to identify participants. 
Measurement and reporting of adverse events
We plan to collect adverse event data from all participants in both treatment conditions on a 
daily basis throughout the study. Potential symptoms include nausea, vomiting, 
lightheadedness, and abdominal pain.   Participants who report clinically relevant 
adverse events will be immediately referred to the PI (Dr. Plaisance) and the Medical 
Monitor (Dr. Garcia) for additional evaluation and referral as appropriate. After 

randomization, should the PI and research team determine that continued participation is 
contraindicated, the participant may be withdrawn from the study. Given that this is 
minimally invasive study, we do not anticipate that withdrawals will be common.
Other risks for this study are also judged minimal. Exogenous ketones have been shown to 
stimulate insulin secretion leading to mild reductions in blood glucose. We do not anticipate 
more than a 5-7% decrease in circulating glucose and do not anticipate symptomatic 
hypoglycemia. In the event that a patient presents with symptomatic hypoglycemia, our Study 
Coordinator, Jonathan Bergeron, will contact Dr. Garcia immediately. 
Measurement and reporting of participant compliance to treatment protocol
Once randomized participants begin the intervention, data on treatment adherence will 
be monitored by research staff on an ongoing basis. Adherence will be measured as the 
number of times the participant consumes the placebo or ketone supplement. Adherence 
will be reviewed after every 5 participants or quarterly (whichever comes first) and if the safety 
officer has concerns about whether compliance has reached a level that might inhibit the 
ability of the study to test its primary hypotheses, he will suggest a conference call for study 
investigators to discuss methods for improving compliance.
Statistical analyses and stopping rules
Although no formal power analysis was conducted for this study (due to exploratory nature of 
the study as pilot and feasibility), our 2:1 (ketone:placebo) recruitment strategy will provide 
opportunity to detect smaller differences in the ketone treated group.  We plan for no interim 
efficacy analyses and will not unblind until recruitment is complete. In this minimal-risk 
study, it is more likely that drop-outs or difficulty in recruiting adequate numbers of 
participants will require stopping the trial than that excess adverse events will occur and 
require stopping the trial. However, as outlined elsewhere, we will monitor adverse event 
rates in all participants and aggregate by randomized group. The safety officer, together with 
the PI, will alert the UAB IRB and the NIH if a larger than reasonably expected adverse 
event rate should occur in either treatment condition. Other issues relating to stopping rules 
for this trial include:
New Information
It is exceedingly unlikely that any new information will become available during this 
trial that would necessitate stopping the trial.
Limits of Assumptions
It is possible that baseline differences between the groups, excessive study dropouts, 
and/or missing data by the interim measurement time points will limit the value of 
data analysis of measurements. Baseline differences will be evaluated and effects on the 
power to detect differences in the primary outcome will be evaluated and 
communicated to the PIs, and safety office. Given the monitoring plans outlined 
elsewhere in this document as well as the randomized design of this study, it is 
exceedingly unlikely that there will be baseline differences between groups of any 
magnitude to threaten the validity of the study.
              

Limits of Rules
We acknowledge that there are other situations that could occur that might warrant 
stopping the trial and have a section in the safety report/checklist to identify other 
situations that might be of safety concern, which will allow for communication of 
concerns to the study MPIs, statistician, and safety officer.
Informed consent
After prospective participants have been preliminarily screened for eligibility and informed 
about the details of the study protocol, they will be asked to review the informed consent 
document. They will have the opportunity to ask questions and discuss all aspects of the 
study prior to providing consent. Informed consent will be obtained by the study coordinator 
or other designated research personnel with IRB clearance and appropriate training in the 
conduct of human research. It will be made clear that participation is voluntary and refusal to 
participate will not affect their eligibility or standing in other studies or programs at UAB 
nor will it affect the clinical care or  other services they receive from their primary care 
physician.