Conditions: Pediatric Obesity
Keywords: Albuterol Caffeine Lean Body Mass Fat Mass
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 12
Healthy volunteers: yes

Brief summary:
The purpose of this study is to determine whether taking a combination of caffeine and albuterol three times per day will increase muscle and decrease fat in your child's body and to determine how these medications make your child feel. Albuterol is approved by the FDA for the treatment of asthma. It is not approved to increase muscle and decrease body fat in children.

Detailed description:
Previous studies done at Pennington Biomedical have demonstrated that the equivalent of oral albuterol 4mg three times a day (tid) with oral caffeine 100mg tid reduces body fat and increases lean tissue in rodents more than the addition of the effect of the two components separately. The combination of albuterol with caffeine changed body composition without changing food intake. An adult male taking albuterol 4 mg orally tid plus caffeine 100mg orally tid increased lean mass by 1.25% and decreased fat mass by 1.2% over a two month period. These effects are expected to be even greater in a growing adolescent. This pilot project will take the first step towards trying to understanding the safety and potential efficacy of this drug combination. The prospect of using inexpensive medications already approved in the pediatric population for the treatment of asthma as a novel treatment for adolescent obesity addresses a medical need that is presently unmet.

Food restriction in adolescence is not only difficult to accomplish, but it also raises concerns about growth and development. A medication approved for the treatment of obesity in the adolescent age group that improves body composition by reducing body fat and increasing lean tissue without needing to restrict food intake would be a useful tool for physicians who address the treatment of obesity in adolescents. Albuterol is a medication, approved for ages 6 and older, used for the treatment of asthma and has also been shown to increase muscle strength and lean body mass in children with spinal muscular atrophy and in healthy young men during an exercise training program. A drug approved for the treatment of adolescent obesity that increases lean tissue, decreases fat tissue and can be given in conjunction with lifestyle modifications would be welcomed by both pediatricians who treat these adolescents and by adolescents who are stigmatized by their obesity.

A provisional patent has been submitted by Pennington Biomedical Research Center to protect the combination of caffeine and albuterol in a 1:25 ratio for synergistically increasing muscle mass and decreasing fat mass as a potential treatment for obesity in adolescents.

This study will be a double-blinded, randomized, placebo-controlled, pilot study in which subjects will be randomized to receive either placebo or a combination of Albuterol 4 mg and Caffeine 100mg three times per day orally for a total of 8 weeks. Each subject will continue on the study intervention for the entire duration of treatment

Arms:
arm 1: One capsule 3 times per day orally for a total of 8 weeks and family weight management counseling for a total of 8 weeks. arm 2: One capsule 3 times per day orally for a total of 8 weeks and family weight management counseling for a total of 8 weeks.

Interventions: intervention 1: Caffeine 100mg / Albuterol 4mg intervention 2: Placebo intervention 3: Family weight management counseling
Intervention descriptions: intervention 1: None intervention 2: None intervention 3: None

Protocol document:
Prot_SAP_000.pdf: 
Page 1 of 11 
 
Effect on Body Composition with Albuterol and 
Caffeine versus Placebo in Adolescents: A Pilot Study 
 
NCT02740660 
 
Protocol Version Date: 05/05/17 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


Page 2 of 11 
 
Protocol Title: Effect on Body Composition with Albuterol and Caffeine versus Placebo 
in Adolescents: A Pilot Study 
 
PI Name: Daniel S. Hsia, M.D. 
 
Sub-Investigator’s Name(s): Frank L. Greenway, M.D. 
 
Protocol Version Date: 05/05/17 
 
Objectives 
1. To assess the change in lean body mass and fat mass with the combination of 
albuterol and caffeine versus placebo in obese adolescents age 12-17 years. 
We hypothesize that the combination of medications will cause an increase in lean body 
mass and a decrease in fat mass, more than the placebo. 
 
2. To assess the safety of the combination of albuterol and caffeine versus placebo in 
obese adolescents age 12-17 years. 
We hypothesize that adverse events in the caffeine/albuterol group compared to 
placebo will be similar. 
 
Background 
Previous studies done at Pennington Biomedical have demonstrated that the equivalent 
of oral albuterol 4mg three times a day (tid) with oral caffeine 100mg tid reduces body 
fat and increases lean tissue in rodents more than the addition of the effect of the two 
components separately.(Liu, Arceneaux et al. 2015)  The combination of albuterol with 
caffeine changed body composition without changing food intake.  An adult male taking 
albuterol 4 mg orally tid plus caffeine 100mg orally tid increased lean mass by 1.25% 
and decreased fat mass by 1.2% over a two month period (unpublished).  These effects 
are expected to be even greater in a growing adolescent.  This pilot project will take the 
first step towards trying to understanding the safety and potential efficacy of this drug 
combination.  The prospect of using inexpensive medications already approved in the 
pediatric population for the treatment of asthma as a novel treatment for adolescent 
obesity addresses a medical need that is presently unmet. 
Food restriction in adolescence is not only difficult to accomplish, but it also raises 
concerns about growth and development.  A medication approved for the treatment of 
obesity in the adolescent age group that improves body composition by reducing body 
fat and increasing lean tissue without needing to restrict food intake would be a useful 
tool for physicians who address the treatment of obesity in adolescents.  Albuterol is a 
medication, approved for ages 6 and older, used for the treatment of asthma and has 
also been shown to increase muscle strength and lean body mass in children with 
spinal muscular atrophy (Skura, Fowler et al. 2008) and in healthy young men during an 
exercise training program.(Caruso, Hamill et al. 2005)  A drug approved for the 
treatment of adolescent obesity that increases lean tissue, decreases fat tissue and can 
be given in conjunction with lifestyle modifications would be welcomed by both 
pediatricians who treat these adolescents and by adolescents who are stigmatized by 
their obesity. 
A provisional patent has been submitted by Pennington Biomedical Research Center to 
protect the combination of caffeine and albuterol in a 1:25 ratio for synergistically 


Page 3 of 11 
 
increasing muscle mass and decreasing fat mass as a potential treatment for obesity in 
adolescents.   
 
Inclusion and Exclusion Criteria 
Participants will come to an initial screening visit (SV) after an overnight fast of 10 
hours.  Consent and assent will be obtained and the inclusion/exclusion criteria below 
will be assessed.  Screening labs (CBC; chem 26 including glucose, BUN, creatinine, 
sodium, chloride, potassium, carbon dioxide, uric acid, total protein, phosphorus, 
albumin, calcium, magnesium, total bilirubin, CPK, LDH, AST, ALT, alkaline 
phosphatase, GGT, amylase, iron, cholesterol, triglycerides, HDL, LDL; fasting insulin; 
and urinalysis with urine pregnancy test as needed) and an EKG will be done to help 
determine eligibility. 
 
Inclusion 
-Healthy males or females with a BMI ≥ 95th percentile  
-Between 12 and 17 years of age inclusive 
-Tanner Stage III and above 
 
Exclusion 
-Weigh less than 50 kg 
-Have a family history of sudden death or hypertrophic cardiomyopathy 
-Have a history of unexplained syncope 
-Have a marked baseline prolongation of QT/QTc interval (QTc interval >450 ms), a 
history of additional risk factors for torsade de pointes (e.g., heart failure, hypokalemia, 
family history of Long QT Syndrome), or use concomitant medications that prolong the 
QT/QTc interval 
-Have history of asthma, hypertension, thyroid disease, or significant 
neurologic disease such as seizure disorder 
-Are pregnant, planning to become pregnant, or nursing.  Females who are sexually 
active must be using adequate contraception. 
-Take a medication known to affect weight or body composition like systemic 
glucocorticoids, atypical anti-psychotics, or weight loss medications 
-Take beta-stimulators or beta-blockers on a regular basis  
-Take stimulants for attention deficit disorder  
-Take monoamine oxidase inhibitors, tricyclic antidepressants, or diuretics 
-Take any chronic medication that has not had a stable dose for 1 month or longer 
-Have type 1 or type 2 diabetes 
-Have any significant cardiac disease (such as heart failure, arrhythmias, or valve 
disease), uncontrolled pulmonary disease, chronic liver disease, chronic kidney 
disease, or chronic infectious disease 
-Have any significant psychiatric illness that is unstable or untreated such as bipolar 
disorder, severe depression, or severe anxiety 
-Have a history of suicidal ideation 
-Have an allergy or hypersensitivity to albuterol 
-Are unwilling to discontinue caffeine-containing products while in the study 
-Are deemed unfit to participate in the study based on evaluation by the medical 
investigator 
 


Page 4 of 11 
 
Number of Subjects 
We expect to enroll 20 adolescents to complete studies in 12 adolescents. 
 
Recruitment Methods 
The Recruitment Core is responsible for recruiting potential participants for clinical trials 
from a population that includes a wide range of people of various ages, ethnic 
backgrounds and with varying degrees of health.  The PBRC recruitment team creates 
and implements individualized, trial-specific advertising and awareness campaigns, 
including mass media, traditional advertising, and novel methods including social media, 
digital, and email marketing.  All advertising and awareness are approved by the PBRC 
IRB to ensure ethical and disclosure standards are met.  The PBRC database may also 
be utilized to find potential subjects for this study. 
 
Study Timelines 
Depending on scheduling, the total duration of this study is approximately 10 weeks 
(including screening) with 8 weeks of treatment. Following screening evaluations, 
eligible subjects will be randomized to the study intervention as follows: either 8 weeks 
of treatment with placebo or 8 weeks with the combination of caffeine/albuterol.  
The estimated length of time to complete the entire study (from enrollment of the first 
subject to completion of the last subject) is 6 months with study analysis to be complete 
6 months after the completion of the last subject. 
 
Study Endpoints 
The primary endpoint is to assess the effect of the combination of caffeine and albuterol 
on fat mass and lean body mass via DXA scan in obese adolescents. 
 
The secondary endpoints include assessing the effect of the combination of caffeine 
and albuterol on hunger and satiety as well as the safety of the combination of caffeine 
and albuterol compared to placebo. 
 
Procedures Involved 
This study will be a double-blinded, randomized, placebo-controlled, pilot study in which 
subjects will be randomized to receive either placebo or a combination of Albuterol 4 mg 
and Caffeine 100mg three times per day orally for a total of 8 weeks.  Each subject will 
continue on the study intervention for the entire duration of treatment. 
 
Overview 
Prior to study entry the following procedures will be performed: 
• 
obtain written informed consent 
• 
record medical history 
• 
verify conformance with entry criteria 
Twelve obese adolescents will be recruited through general advertisement and 
physician referrals.  The study will consist of 6 total study visits.  The first study visit will 
be a screening visit and include a medical history/physical, an electrocardiogram, and 
screening labs.  The Beck Depression Inventory will also be used to screen for 
depression in these adolescents.  A psychologist is available on site to assess anyone 
whose score indicates further evaluation is necessary, and the adolescent may be 
referred to outside sources if deemed necessary.    Adolescents who qualify will return 


Page 5 of 11 
 
for a baseline visit fasting within one month of the screening visit.  The adolescent will 
have a DXA scan for body composition as well as a Visual Analog Scale (VAS) test for 
hunger and satiety.  A parent will complete a Vanderbilt Assessment Scale for ADHD to 
determine if the adolescent has any signs of attention deficit or hyperactivity at baseline.  
The adolescent will then be randomized to either the placebo or treatment group 
(Albuterol 4 mg tid and Caffeine 100 mg tid orally) in a 1:1 ratio. The albuterol and 
caffeine will be obtained from Spectrum Chemical, Letco Medical, or a similar approved 
distributor where the pharmacy acquires FDA approved medications.  Albuterol is 
approved for the treatment of asthma in adolescents (12-18) at 4 mg three or four times 
a day. Starting with the baseline visit and at every 2 week visit, the adolescent and 
parent will meet with a registered dietitian certified in pediatric weight management.  
The information provided will include recommendations on healthy eating, increasing 
physical activity, and family participation as recommended by the Academy of Nutrition 
and Dietetics.  At week 2, 4, 6, and 8, compliance (via pill counts) and adverse events 
will be assessed.  At the week 4 visit, the screening labs will be repeated along with an 
electrocardiogram as safety measures.  In addition to these assessments, at week 4 
and 8, the adolescent will come fasting to clinic and having taken their morning dose of 
study medication. They willhave a repeat VAS scale for hunger and satiety, and a 
parent will fill out another Vanderbilt Assessment Scale for ADHD as a safety measure.  
If there are any concerns based on the Vanderbilt Assessment Scale, the intervention 
will be stopped and the adolescent will be referred to an appropriate health care 
provider for further evaluation.  At the last study visit after 8 weeks of intervention, the 
adolescent will have an electrocardiogram, a repeat DXA, and repeat labs to compare 
with the screening labs.  Approximately 7 days (± 2 days) later, someone from the study 
staff will call the participant to assess for any adverse events while off the study 
intervention.    
 
Study Evaluations 
The following study evaluations will be obtained at the screening visit (SV): 
• 
Body weight, height, body mass index (BMI), vital signs 
• 
Complete medical history and physical examination 
• 
Beck Depression Inventory 
• 
Standard 12-lead EKG 
• 
Urinalysis  
• 
Labs including fasting insulin, Chemistry 26 and CBC (~7.5 mL or 1.5 teaspoons 
of blood) 
• 
Collection of concomitant medications 
 
The following study evaluations will be obtained at the Baseline visit: 
• 
Body weight, height, body mass index (BMI), vital signs 
• 
Family weight management counseling 
• 
Pregnancy test (if applicable) 
• 
DXA scan for body composition 
• 
Randomization to either study intervention 
• 
Dispensing of study medication 
• 
Visual Analog Scale for hunger and satiety 
• 
Vanderbilt Assessment scale for ADHD  
• 
Collection of concomitant medications 


Page 6 of 11 
 
The following study evaluations will be obtained at the Week 2 visit 
• 
Body weight, height, body mass index (BMI), vital signs 
• 
Collection of concomitant medications 
• 
Family weight management counseling 
• 
Dispensing and collecting study medication 
• 
Assessment of tolerability and compliance (via pill counts) 
• 
Assessment of adverse events 
 
The following study evaluations will be obtained at the Week 4 visit 
• 
Body weight, height, body mass index (BMI), vital signs 
• 
Collection of concomitant medications 
• 
Family weight management counseling 
• 
Dispensing and collecting study medication 
• 
Assessment of tolerability and compliance (via pill counts) 
• 
Assessment of adverse events 
• 
Visual Analog Scale for hunger and satiety 
• 
Pregnancy test (if applicable) 
• 
Urinalysis  
• 
Labs including fasting insulin, Chemistry 26 and CBC (~7.5 mL or 1.5 teaspoons 
of blood) 
• 
Standard 12-lead EKG 
• 
Vanderbilt Assessment scale for ADHD  
 
The following study evaluations will be obtained at the Week 6 visit 
• 
Body weight, height, body mass index (BMI), vital signs 
• 
Collection of concomitant medications 
• 
Family weight management counseling 
• 
Dispensing and collecting study medication 
• 
Assessment of tolerability and compliance (via pill counts) 
• 
Assessment of adverse events 
 
The following study evaluations will be obtained at the Week 8 visit 
• 
Body weight, height, body mass index (BMI), vital signs 
• 
Collection of concomitant medications 
• 
Family weight management counseling 
• 
Collecting study medication 
• 
Assessment of tolerability and compliance (via pill counts) 
• 
Assessment of adverse events 
• 
Pregnancy test (if applicable) 
• 
Urinalysis 
• 
Labs including fasting insulin, Chemistry 26 and CBC (~7.5 mL or 1.5 teaspoons 
of blood) 
• 
DXA scan for body composition 
• 
Standard 12-lead EKG 
• 
Visual Analog Scale for hunger and satiety 
• 
Vanderbilt Assessment scale for ADHD  
 
The following study evaluation will be done at Week 9 over the phone 


Page 7 of 11 
 
• 
Follow up with the participant, or participant’s parent(s)/legal guardian if 
participant is a minor, to assess for any adverse events that may have occurred 
after stopping the intervention.   
 
Study Procedures 
Whole Body DXA scan (Baseline and Week 8) 
This scan measures the amount of bone, muscle, and fat in your body.  The scan will be 
performed using a whole-body scanner.  You will be required to wear a hospital gown, 
to remove all metal-containing objects from your body, and to lie down on the table.  A 
scanner emitting low energy X-rays and a detector will pass along your body.  The scan 
takes approximately 5 to 10 minutes.  You will be asked to remain completely still while 
the scan is in progress.  This scan is for research purposes only and not for diagnostic 
treatment. 
 
Study Schedule 
Procedures 
SV 
Baseline Week 2 
Week 4 Week 6 Week 8 Week 9 
Inclusion/Exclusion,  
Consent and Assent  
X 
 
 
 
 
 
 
History and Physical 
X 
 
 
 
 
 
 
Beck Depression Inventory 
X 
 
 
 
 
 
 
Weight, height, vital signs 
X 
X 
X 
X 
X 
X 
 
Collection of concomitant 
medications 
X 
X 
X 
X 
X 
X 
 
Family weight management 
counseling 
 
X 
X 
X 
X 
X 
 
DXA scan for body 
composition 
 
X 
 
 
 
X 
 
Screening labs: blood draw 
X 
 
 
X 
 
X 
 
Screening labs: urinalysis  
X 
X 
 
X 
 
X 
 
Urine pregnancy test (if 
applicable) 
 
X 
 
 
 
X 
 
EKG 
X 
 
 
X 
 
X 
 
Dispense and collect 
medications 
 
X 
X 
X 
X 
X 
 
Tolerability and compliance 
(pill counts) 
 
 
X 
X 
X 
X 
 
Adverse events 
 
 
X 
X 
X 
X 
 
Randomization to treatment 
sequence 
 
X 
 
 
 
 
 
Visual Analog Scale for 
hunger and satiety 
 
X 
 
X 
 
X 
 
Vanderbilt Assessment 
scale for ADHD 
 
X 
 
X 
 
X 
 
Follow up phone call 
 
 
 
 
 
 
X 
      # Each visit has a window of ± 2 days 
 


Page 8 of 11 
 
Power analysis 
This is a pilot study and no power analysis was calculated.  Data from this study may be 
used to power a larger study. 
 
Data and Specimen Management 
The data management will be conducted by the Pennington Biomedical Research 
Computing Group.  There will be limited access to all data including locked cabinets for 
paper files and password protected computers for electronic data.   
Each participant will be issued an ID number that will be utilized throughout the study. A 
secure master file linking names, addresses and ID numbers will be maintained in a 
confidential computer file accessible only to the investigators.  Access to data files can 
be made only with permission of the Principal Investigator.  Privacy in the context of this 
study includes confidentiality of data and personal information.  During interviews, 
measurements, and other study-related activities, the study staff will ensure full privacy 
of participants and will ensure that the data are stored in a secured area.  All study staff 
must be HIPAA certified. 
 
Statistics 
Comparisons will be made between the control and intervention groups using chi-
squared tests to compare categorical data and t-tests to compare means with 2 time 
points and analysis of variance for end points with repeated measures with a significant 
p value of ≤ 0.05. 
 
Provisions to Monitor the Data to Ensure the Safety of Subjects 
Participants and their parents will be instructed on symptoms that should prompt them 
to contact the site or seek medical attention, such as palpitations or lightheadedness.  
Adverse events will be monitored every 2 weeks at each study visit.  The PI and his co-
investigator will review lab data on a regular basis throughout the study (screening, 
Week 4, and Week 8) to ensure the safety of each subject.  Any significant health 
problems coming to our attention during the study will be referred to the participant’s 
usual source of medical care, with his/her parents’ permission. 
A formal IND application has been filed with the FDA and in addition to the reporting 
requirements set forth by the IRB, additional responsibilities include: 
-Reporting any unexpected fatal or life-threatening suspected adverse reactions to the 
FDA no later than 7 calendar days after initial receipt of the information 
-Reporting any (1) serious, unexpected suspected adverse reactions, (2) findings from 
other clinical, animal, or in-vitro studies that suggest significant human risk, and (3) a 
clinically important increase in the rate of a serious suspected adverse reaction to the 
FDA and to all investigators no later than 15 calendar days 
- Submitting annual progress reports within 60 days of the anniversary of the date that 
the IND became active (the date clinical studies were permitted to begin) 
 
Withdrawal of Subjects 
During the course of the study, participants may be withdrawn from the study for the 
following reasons: 
 Unwillingness to participate in the study or cooperate with study staff 


Page 9 of 11 
 
 Presentation of significant medical symptoms or abnormal findings such as a 
prolonged QTc that would warrant termination of study participation to protect the 
participant’s safety 
 
Data that have already been collected during the course of study participation from a 
withdrawn participant will be used, unless a specific request is otherwise received. 
Subjects will be notified of their withdrawal via telephone or mail. 
 
Risks to Subjects 
General: More serious risks and adverse effects may occur than those listed below.  
The combination of caffeine and albuterol has not been well studied and adverse effects 
may be additive or synergistic with the combination.  There may be more risks of 
ingesting additional caffeine products (such as coffee, tea, Coke® , or Red Bull®) and 
alcohol while in the study.  A list of caffeine-containing products to avoid during the 
study will be provided as a reference.  If a dose is missed, the participant should not 
double up on the dose because of the possible risk of adverse effects.  
 
Caffeine: This can be found in a variety of foods and beverages.  Caffeine is a stimulant 
and may cause increased heart rate, alertness, tremor, and difficulty sleeping.   
Albuterol: This is a prescription medication that is approved for use in treatment of 
asthma in children as young as 6 years of age.  The most common side effects include: 
nervousness, tremor, headache, tachycardia, and palpitations.   
 
Blood Draws:  There is the possibility of infection and/or pain and bruising at the vein on 
your arm where the needle is inserted.  Aseptic (sterile) technique and trained 
personnel minimize these risks.  
 
Electrocardiogram (EKG or ECG):  There are minimal risks associated with this test. 
There is a small possibility there may be some redness or irritation while cleaning the 
skin prior to applying the electrodes or if you happen to be allergic to the adhesive on 
the electrodes.   
 
Whole Body DXA scan: The amount of radiation used for this procedure is very small.  
The radiation dose for this scan is equivalent to the radiation you are naturally exposed 
to in the environment in less than one day.  Scans will not be performed on any subject 
who is pregnant, and all females will be provided with a urine pregnancy test prior to the 
scan.     
 
Visual Analog Scale for hunger and satiety: This is a well-validated scale to assess 
hunger and satiety and carries little to no risk. 
 
Vanderbilt Assessment scale for ADHD: There is little to no risk in filling out this 
questionnaire.  This is used by pediatricians and parents to identify a child’s risk for 
attention deficit/hyperactivity disorder.  This will serve as an additional safety measure 
and will be obtained at baseline and then every 4 weeks.  
 
Potential Benefits to Subjects 


Page 10 of 11 
 
While in the study, participants may demonstrate an improvement in body composition 
with decrease in fat mass and increase in lean body mass.  These changes may not be 
sustained once the participant stops the intervention.   
 
Vulnerable Populations 
Adolescents between the ages of 12 and 17 who are in the midst of puberty will be 
enrolled in the study.  All participants will be provided with an age appropriate assent 
form in additional to the parental consent form.  All questions and concerns will be 
addressed by the study staff.   
 
Sharing of Results with Subjects 
Individual participant results will not be provided; however, results from the study will be 
submitted for manuscripts in scholarly journals and presentations.  All study reports will 
present only aggregated data to minimize the risks that a participant can be identified 
form their participation in the study.  As required by the FDAAA this study will be 
registered on ClinicalTrials.gov. 
 
Setting 
Potential participants will be recruited utilizing the recruiting services (Recruitment Core) 
of the PBRC. All research procedures will be performed at the PBRC, namely, in the 
Outpatient Clinic. 
 
Resources Available 
Pennington Biomedical Research Center has had a good track record with recruiting a 
population of overweight/obese adolescents for a variety of intervention studies.  
Potential participants come from a variety of sources such as traditional advertising, 
physician referral, word of mouth, etc. 
The clinical research staff are well trained and have to undergo a variety of certifications 
before performing assessments on study participants.   
Medical personnel are available onsite to conduct study visits as well as oversee any 
adverse events.  Psychologists are available on call should the need arise for further 
evaluation based on screening inventories or other assessments.  
Prior to the opening of the study, a start up meeting is held with all of the relevant 
clinical cores to ensure that proper coordination is in place prior to the first screening 
visit. 
 
Compensation 
Each participant will receive $25 after the completion of the baseline visit, week 2, week 
4, and week 6 visits, and $50 after the week 8 visit for a total of $150.  
 
Provisions to Protect the Privacy Interests of Subjects 
All attempts will be made to maintain a subject’s privacy.  Safeguards such as password 
protected computers and networks have been put in place in order to limit access to 
subject data.  These data will be maintained until the results may be published and will 
only be accessible by study personnel.   
Subjects will be given ample time to read over the consent, ask questions, and agree to 
participate in the research study.  Subjects may decline answering questions they are 


Page 11 of 11 
 
not comfortable with.  Each procedure will be explained to the subject before it is 
performed. 
 
Compensation for Research-Related Injury 
No compensation will be provided for research-related injury. 
 
Economic Burden to Subjects 
All study-related tests and procedures will be at no cost to the subject.  The subject will 
incur transportation costs in getting to PBRC. 
 
Consent Process 
The P.I. or one of the designated clinic staff will obtain informed consent from the 
parent/guardian and separate assent from all of the minor participants in the outpatient 
clinic during the screening visit.  Ample opportunity will be given for the parent/guardian 
and the minor participant to review the consent/assent and to ask any questions prior to 
signing any forms. 
A parent/guardian is to provide his or her identification along with the child’s birth 
certificate in order to verify the relationship to the minor participant.   
As a greater than minimal risk study with the prospect of direct benefit, one parent may 
provide consent for the minor participant to engage in the study. 
 
Drugs or Devices 
Both interventions will be stored in the research pharmacy located in the outpatient 
clinic.  This is locked at all times and only accessible to limited personnel via a key card.  
The research pharmacist will maintain an inventory of study drugs.  Appropriate 
dispensation of study drug will be confirmed with a second clinic staff member to ensure 
correct drug is being provided to the correct participant.