Clinical trial NCT02192827
Conditions: Asthma
Keywords: Pediatric Asthma Exacerbation Dexamethasone
Sponsor: University at Buffalo
Start date: 2015-04-01; Completion date: 2018-03-01
Enrollment: 318 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
The purpose of this study is to compare the efficacy of a single dose of dexamethasone vs. two doses of dexamethasone in mild and moderate asthma exacerbations pediatric patients.

Detailed description:
The emergency department (ED) physician will follow the standard asthma care path to manage a pediatric asthma patient whether the patient meets study criteria or not. Patients' legal guardian will be approached for consent if patient meets the study criteria. Once the consent is obtained, the patient will be randomized into one of the two study groups according to a randomization table generated by a statistician. The first group will receive a single dose of dexamethasone sodium phosphate injection (0.6 mg/kg with max of 16 mg) mixed with equivalent volume of cherry syrup given orally in the ED. The second group will receive a first dose of dexamethasone with cherry syrup (also 0.6 mg/kg with max of 16 mg) in the ED, and a second dose will be prescribed to the patient at home on the day following the ED visit. This dose will be the same dosage of dexamethasone, but may be a pill or liquid form. Patients will continue their previously prescribed asthma regimen and albuterol as needed. Children who vomit the steroids in the ED will be re-dosed. Children who vomit the steroid a second time will be excluded from the study.

2\. Data collection. Demographic information such as age, race, duration of asthma symptoms, number of previous hospitalizations, and current medication will be collected on an ED data collection form. Pertinent exam findings such as patient's vital signs, pulse oximetry, Pediatric asthma severity score (PAS), patient asthma severity self-assessment sheets (PSAS), and ED treatment will also be collected on an ED data collection form.

After discharge from the ED, patients will be instructed to complete the PSAS on a daily basis for 5 days. The data will be documented on a home patient self-assessment sheet by the legal guardian. The investigators will train the legal guardians on how to complete the PSAS in the ED.

3\. Phone follow-up All patients will be contacted by phone by a research assistant 5 days after the ED visit. Information collected during the phone interview will include PSAS, unexpected visits to medical providers (ED, primary care or urgent care) for asthma symptoms, school days missed due to asthma exacerbation, length of time symptoms persisted, compliance with the recommended steroid regimen, vomiting, other side effects or medication administration problems caused by the steroids.

Arms: arm 1: 0.6 mg/kg of Dexamethasone Sodium Phosphate Injection 10mg/1ml given with equivalent volume of cherry syrup given orally once arm 2: 0.6 mg/kg of Dexamethasone Sodium Phosphate Injection 10mg/1ml given with equivalent volume of cherry syrup given orally once in the Emergency Department (ED), followed by another dose of dexamethasone at home, which will be prescribed from the ED. The second dose will be the same dosage, but will be prescribed and may be pill or liquid form.
Interventions: intervention 1: Dexamethasone Sodium Phosphate Injection
Intervention descriptions: intervention 1: None

Protocol documents:
Prot_SAP_000.pdf: 
 
 
Official Title: 
Single Dose Dexamethasone is Not Inferior to Two Doses 
in Mild to Moderate Pediatric Asthma Exacerbations in the 
Emergency Department 
NCT number: 
NCT02192827 
Document Type:  
Study Protocol and Statistical Analysis Plan 
Date of the 
Document: 
November 9, 2017 
 
 
 


Methods 
We performed a single-site, prospective, parallel group, un-blinded randomized clinical trial which 
enrolled a convenience sample of patients aged 2-20 years with previously diagnosed asthma presenting 
to a single emergency department with a mild to moderate exacerbation of asthma. The hospital’s 
institutional review board approved the study and informed consent was obtained from eligible 
patients’ legal guardians or adult patients (18-20 years age).  In addition, patients 7 to 17 years of age 
provided written ascent.  
 
Study Setting and Participants:  
Children aged 2 to 20 years with a known history of asthma who presented to the Emergency 
Department at Women and Children’s Hospital of Buffalo/Oishei Children’s Hospital between April 2015 
and March 2018 with an acute exacerbation of mild or moderate asthma were eligible for the study. 
Asthma severity was defined by Pediatric Asthma Scores[2, 12] (PAS). Mild asthma is defined as: PAS of 
5 to 7; Moderate asthma is defined as: PAS of 8 to 11; Severe asthma is defined as: PAS of 12 or more. 
History of asthma is defined by physician diagnosis of at least one prior episode of wheezing which 
responded to beta agonists.  Patients were excluded from the study if they had signs of severe asthma 
exacerbation (PAS of 12 or more), had used oral steroids in the last 2 weeks, had chronic lung disease 
(e.g., cystic fibrosis), had been given  parenteral steroids, or if they vomited two doses of oral steroids in 
emergency department.  
Research assistants screened asthma patients through the electronic medical record between the hours 
of 8 a.m. and 11 p.m. 7 days a week. Patients were approached sequentially based on registration time. 
If a patient appeared to meet study criteria, his/her ED treating physician was approached by the 
research assistant for identifying patient’s eligibility.  If a patient was fully eligible, the legal guardian 
and/or patient were approached for consent.  
 
Study Protocol: 
Block randomization was used to generate a list to be used for subject assignment with a 1:1 ratio of 
allocation to the single dose group and the two dose group. Demographic information such as age, 
gender, race, duration of asthma symptoms, number of previous hospitalizations, and current 
medication was collected.  Pertinent exam findings such as patient’s vital signs, pulse oximetry, and PAS 
were also collected along with medications given in the ED.   
Group 1 was given 0.6mg/kg (max 16mg) of dexamethasone orally in the emergency department. Group 
2 was given the same dose in the emergency department, and then a prescription for a second dose was 
sent to their pharmacy to be filled and administered at home 24 hours later.  We worked with a nearby 
24-hour pharmacy to ensure medication availability; however, pharmacy preference was left up to the 
family. Both groups received asthma treatment following a standardized asthma care path.     
All patients were contacted by phone by a research assistant on the 6th day following the emergency 
department visit. Research assistants were not completely blinded during the study. They were initially 
blinded to the study group at the start of the phone interviews; however, during the course of the 


interview they did ask if the prescription for the second dose of dexamethasone was filled and if the 
medication was given to the patient.  Information collected during the phone interview included 
additional visits to medical providers (ED, primary care or urgent care) for continued asthma symptoms, 
information was collected on the reason for visit, whether symptoms continued or worsened, and if 
additional treatment was needed (scheduled visits and unrelated visits were excluded). School days 
missed due to asthma exacerbation; length of time symptoms persisted; compliance with the 
recommended steroid regimen; and any vomiting, side effects or medication administration problems 
caused by the steroids was also collected.   
 
Analytic Plan: 
Descriptive characteristics were computed for all patients combined and separately by dexamethasone 
group assignment.  Categorical variables were reported as proportions in percentage, and continuous 
level variables as means and standard deviations.  Separate independent t-tests were used to assess 
differences by group for post-discharge outcomes of interest including days for symptoms to resolve.  
Separate binary logistic regression adjusting for age, gender and severity of exacerbation were 
conducted to assess group differences in post-discharge outcomes including patients who had a return 
visit for unresolved asthma symptoms (ED, primary care or urgent care), days to symptom resolution, 
any missed school days between discharge and follow-up, rates of vomiting, and side-effects (changes in 
appetite, insomnia, mood swings); odds ratios with 95% confidence intervals were reported.  Similar 
analyses were conducted to compare events and side effects between Groups 1 and 2 by asthma 
severity, using an intention-to-treat approach.  Based on our clinical experience, we based our a-priori 
sample size calculation on an estimated non-inferiority limit of 11% and a rate of 12% return for asthma 
for each group.  With a power of 80% and alpha of 0.05, a minimum sample size of 216 (108 subjects per 
group) was determined. All statistical tests were two-tailed, and analyses conducted with SYSTAT 13 
(SYSAT Software, 2004). 

ICF_001.pdf: 
Permission to Take Part in a Human Research Study
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IRB Approval Period
HRPP Revision Date: Aug 6, 2014
University at Buffalo Institutional Review Board (UBIRB)
Office of Research Compliance | Clinical and Translational Research Center Room 5018
875 Ellicott St. | Buffalo, NY 14203
UB Federalwide Assurance ID#: FWA00008824
Parental permission form 
 Title of research study: Efficacy of single vs. double dose dexamethasone treatment 
for mild to moderate asthma in a pediatric Emergency Department
Version Date:  November 7, 2017
Investigator: Dr. Heather Territo
Why is my child being invited to take part in a research study?
Your child is being invited to take part in this research study because he/she has mild to moderate 
asthma and will be treated with steroids in our emergency department.
What should I know about a research study?

Someone will explain this research study to you.

Whether or not your child takes part is up to you.

You can choose not to let your child take part.

You can agree to let your child take part and later change your mind.

Your decision will not be held against you or your child.

You can ask all the questions you want before you decide.
Who can I talk to?
If you have questions, concerns, or complaints, or think the research has hurt your child, talk to the 
research team at 716-323-0221.  You may also contact the research participant advocate at 716-888-
4845 or researchadvocate@buffalo.edu  
This research has been reviewed and approved by an Institutional Review Board (“IRB”). You may 
talk to them at (716) 888-4888 or email ub-irb@buffalo.edu.
 if:

Your questions, concerns, or complaints are not being answered by the research team.

You cannot reach the research team.

You want to talk to someone besides the research team.

You have questions about your child’s rights as a participant in this research.

You want to get information or provide input about this research.
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Why is this research being done?
Asthma affects millions of adults and children every year and accounts for one of the most common
emergency room visits during the winter months. One of the ways we treat asthma is with steroids. We 
usually use Prednisone or Prednisolone to treat asthma as an outpatient, but these medications do not 
taste good and can cause vomiting, trouble sleeping and behavior changes.  These medications also 
have to be taken twice a day for several days and it can be hard to remember to take them.
This study is designed to look at a different steroid medication called dexamethasone which has been
used in asthma treatment in other studies as well as in many pediatric clinics including this emergency 
department.  Dexamethasone is approved by the Food and Drug Administration to treat other breathing 
problems.  It does not taste as bad and may have fewer side effects when compared to prednisone.  
Dexamethasone does not need to be taken as much as the prednisone, which makes this medication 
easier to take.  By doing this study, we would like to know if one dose of dexamethasone works the 
same as two doses of dexamethasone in treating children with mild or moderate asthma symptoms.  If 
this is true, then one dose of dexamethasone would increase compliance and could potentially decrease 
the incidence of side effects. 
How long will the research last?
We expect that your child will be in this research study for 6 days, and on the 6th day post ED 
discharge, we will call you on the phone to follow-up with you about your child’s asthma control 
status.  Your child will not be staying longer in the ED due to his/her participation in this study.  We 
will ask you to document your child’s asthma status at home for five days after being discharged from 
our ED, so your child will be in the study for a total of 7 days including the ED visit day and the phone 
follow-up day. 
How many people will be studied?
We expect about 300 people will be enrolled in this research study.   
What happens if I say yes, I want my child to be in this research?
 If you decide to allow your child to take part in this research study, your child will receive the 
standard asthma treatment with albuterol or other medications as decided by the ED doctor.  In 
addition, your child will also be treated with dexamethasone for the study purpose.  We have two study 
groups; neither you nor the study doctor will choose which study group your child will be in. Your 
child will have an equal opportunity (like tossing a coin) to be enrolled into one of the two study 
groups.   
If your child is enrolled into the first study group, he/she will be given one dose of dexamethasone in 
the ED by our nurse.  If your child is enrolled in the second study group, we will give him/her two 
doses of dexamethasone.  The first dose will be given in the ED by our nurse, and the second dose will 
be given to your child by the care giver 24 hours after the first dose is being given.  We will prescribe 
the second dose of dexamethasone. You can fill the prescription either in our outpatient pharmacy or at 
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the Walgreens Pharmacy located at 650 Delaware Ave, Buffalo, NY 14202,  or a local pharmacy 
nearby your home. 
After ED discharge, your child will continue his/her routine asthma care at home including albuterol 
and controller medications if he/she has been prescribed these before. If your child is older than 6 years 
of age, we will also measure his/her peak flow in the emergency department, and our respiratory 
therapist will provide you a peak flow meter to use at home if your child does not have one. We will 
ask you to record your child’s peak flows every day for five days. We will also ask you to record the 
results in an asthma score sheet every day in order to monitor your child’s asthma symptoms.  The 
asthma score sheet will be provided to you in the ED.
A research assistant will call you on the 6th day after your child’s ED visit and get the asthma score 
information from you. In addition, we would also like to know if you have noticed that your child has 
experienced any side effects from the medication and if your child has to seek any further medical care 
after he/she is discharged from our ED. The phone call takes about 5 minutes of your time. 
We will not collect any specimen from your child by participating in this study. If your child vomits 
dexamethasone two times in the ED, he/she will be withdrawn from the study, and our ED doctor will 
administer IV solumedrol (another steroid) to your child, which is the standard asthma treatment in the 
ED for children who vomit the oral steroid medication.  The study procedure will be ended upon the 
completion of the phone follow up.  We will not contact you for any other study in the future. All data 
collected for this study will only be used for this research project, and will not be used for future 
research; the data forms will be kept in a locked file cabinet in our ED research office.  
What are my responsibilities if my child takes part in this research?
If your child takes part in this research, you as the legal guardian will be responsible to: (1) administer 
the second dose of dexamethasone to your child, or notify other care givers to give this medication to 
your child if your child is enrolled in the second study group; (2) document your child’s peak flow 
measurements and self-assessment scores for 5 days after the ED discharge; (3) we will call you on the 
6th day after the ED discharge and would like to get your help to answer some questions regarding your 
child’s asthma status. 
What happens if I do not want my child to be in this research?
 
If you decide not to let your child participate in this study, your child will receive the standard care for 
asthma treatment, i.e. receives prednisone or prednisolone (for children who cannot swallow pills) 
twice daily for 5 days or dexamethasone treatment.   The decision will be made by your child’s ED 
physician. 
One potential risk for your child to receive the 5 days prednisolone is poor compliance since the 
medication does not taste good, and it requires 5 days treatment.  We do not know if there is a 
significant benefit for children taking the 5 days prednisone or prednisolone treatment compared with 
taking one or two doses of dexamethasone.
 
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What happens if I say yes, but I change my mind later?
Your child can leave the research at any time and it will not be held against you or your child.
If you decide to withdraw your child from the study before your child is enrolled in the study, your 
child will be given the standard asthma treatment as described above.  No research data will be 
collected from your child’s medical record. 
If you decide to withdraw your child from the study after he/she gets enrolled in the study, but before 
we conduct phone follow up, we will not call you and collect the follow-up data. Data collected before 
your withdrawal will continue to be used for this study. 
If you withdraw your child from the study when we conduct the phone follow-up, we will collect no 
more data from your child, but data collected before your withdrawal will continue to be used for the 
study. 
Is there any way being in this study could be bad for my child?
This study involves no more than minimum risk to the participants; there is a risk that your child may 
not tolerate dexamethasone. If your child throws up two doses of the medication, he/she will be 
withdrawn from the study.  The other potential risk is that your child may continue to have asthma 
symptoms, but this could happen when your child receives the prednisone treatment as well. If your 
child’s condition is not becoming better or getting worse after ED discharge or finishing his/her second 
doses of dexamethasone at home, we suggest that you contact your child’s primary doctor immediately 
for further medical care.   
The medication used in the study is not a new medication and is used by many pediatric ED doctors to 
treat asthma in this country including this emergency department.  It also has been used to treat other 
breathing problems like croup. 
If your child is pregnant or becomes pregnant during this study, you should be aware that, the study 
drug is in the same pregnancy risk category (level of risk to the baby) as the drug that is typically used 
to treat mild to moderate asthma. However, there may be unforeseen risks to the fetus.
Will being in this study help my child in any way?
We cannot promise any benefits to your child or others by taking part in this research. However, 
possible benefits include that your child may experience fewer side effects from the dexamethasone 
compared with prednisone he/she would normally receive. In addition, your child will receive one or 
two doses of dexamethasone treatment, which may lead to a much higher compliance rate comparing 
the standard 5 days twice daily prednisolone/ prednisone treatment.  At the end,  his/her participation 
in this study will help our doctors to find out if we can treat asthma with one dose of dexamethasone 
with the same effectiveness as the two doses of dexamethasone in the future, which implies less 
medication and potentially fewer side effects.   
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What happens to the information collected for the research?
Efforts will be made to limit the use and disclosure of your child’s personal information, including 
research study and medical or education records, to people who have a need to review this information. 
We cannot promise complete secrecy. Organizations that may inspect and copy your child’s 
information include the IRB and other representatives of this organization.  
 
We may publish the results of this research.  However, we will keep your child’s name and other 
identifying information confidential, only group data will be presented. 
A description of this clinical trial will be available on http://www.ClinicalTrials.gov, as required by 
U.S. Law. This Web site will not include information that can identify your child. At most, the Web 
site will include a summary of the results. You can search this Web site at any time.
Federal law provides additional protections of your child’s medical records and related health 
information. These are described in the HIPAA section of this document.
 
Can my child be removed from the research without my OK?
The principal investigator of the study can remove your child from the research study without your 
approval.  Possible reasons for removal include if your child vomits dexamethasone two times in the 
ED. 
 
What else do I need to know?
 
You will not be informed of the results of the study. The study results may be presented at a national 
conference or published in a medical journal.  However, only group data will be presented.  No 
individual information or identity will be presented in the public.  
 
 Taking part in this research study will not add extra costs to you.   You and your insurance company 
will be charged for the health care services that you would ordinarily be responsible to pay. You will 
pay for the dexamethasone medication your child receives in the ED as well as the second dose of 
dexamethasone that he/she will receive if your child is enrolled in the second group of the study.  You 
will also pay for the flow meter that the respiratory therapist provides to you in the ED, since it is part 
of the standard care device for asthma patient.   You make the payment for the dexamethasone and the 
flow meter the same way as you would pay if your child is not taking part in the study. 
You or your child will not be paid by taking part in this study. 
  
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HIPAA:  Authorization for the Use and Disclosure of Identifiable Health 
Information for Research Purposes  
This section describes information about your child and about his/her health that will be obtained by 
the researchers when your child takes participate in the research study. Health information is 
considered "protected health information" when it may directly identify your child as an individual. By 
signing this form you are agreeing to permit the researchers and/or other parties (described in detail 
below) to have access to this information.  If there are any parts of this form that you do not 
understand, please be sure to ask us for further clarification.
A. What protected health information will be collected about your child as part of this 
research study?
 
___X__  Information from your child’s full medical records:
___X__  New Health information created from study related tests, procedures, visits, and/or   
questionnaires as described in this consent form.
Provide a general description of information that will be collected: Your child’s 
demographic information, current medications, physical exams findings, asthma severity score, 
patient self-assessment score, ED treatment etc. 
B.    Who is authorized to provide or collect this information?
  
___       Principal Investigator or designee
C.   With whom may your child’s protected health information be shared? 
Your child’s health information may be shared with others outside of the research group for 
purposes directly related to the conduct of this research study or as required by law, including 
but not limited to:
 
_____ Clinical staff not involved in this research study who may become involved in your 
child’s care if it is potentially relevant to his/her treatment
  
Your child’s information may also be shared with individuals or entities responsible for general 
administration, oversight and compliance of research activities.  Examples of this include the 
institution's Privacy and Security Officers or other internal oversight staff, Safety Monitoring 
Boards, an Institutional Review Board, The Research Foundation of the State University of 
New York, University at Buffalo Foundation Services, and accrediting bodies, or with certain 
government oversight agencies that have authority over the research including the Department 
of Health and Human Services (HHS), the Food and Drug Administration (FDA), the National 
Institutes of Health (NIH), and the Office of Human Research Protections (OHRP). Your 
child’s information may also be shared with other entities as permitted or required by law. All 
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reasonable efforts will be used to protect the confidentiality of your child’s individually 
identifiable health information that may be shared with others as described above.
All reasonable efforts will be used to protect the confidentiality of your child’s protected health 
information. There is the potential for individually identifiable information and the associated 
health information obtained with this authorization to be re-disclosed by the recipient(s). After 
such a disclosure, the information may no longer be protected by the terms of this authorization 
against further re-disclosure.
D.  How long will this information be kept by the Principal Investigator?
 
____  b.  This authorization will expire at the end of the research study. After that time, this 
authorization may not be used to acquire additional information about your child.
 
_____  d.  Your child’s protected health information will go into a database that will be 
maintained indefinitely. Any future study using this information that falls outside the 
scope of this current study will be required to follow guidelines designed to govern 
access to that information and to protect the privacy of that information.
E.  What are your rights after signing this authorization?
You have the right to revoke this authorization at any time. If you withdraw your authorization, 
no additional efforts to collect individually identifiable health information about your child will 
be made. You should know, however, that protected health information acquired using this 
authorization prior to its withdrawal may continue to be used to the extent that the 
investigator(s) have already relied on your permission to conduct the research. If you chose to 
withdraw this authorization, you must do so in writing to the following individual(s):
Dr.  Heather Territo
Division of Emergency Medicine
Conventus Building, 5th floor, Room 5272
1001 Main Street
Buffalo, NY 14203
If you send us a request to withdraw your authorization, we will forward that request to the 
institutions we have shared it with in order to collect your child’s individually identifiable 
health information. 
F.  What will happen if you decide not to sign this authorization?
Refusing to sign this authorization will not affect the present or future care your child receives 
at this institution and will not cause any penalty or loss of benefits to which your child is 
otherwise entitled. If you decide not to sign this authorization, your child will not be able to 
participate in the research study.  
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Signature Block for Parental Permission
Your signature documents your permission for the named child to take part in this 
research.  By signing this form you are not waiving any of your legal rights, including the 
right to seek compensation for injury related to negligence or misconduct of those 
involved in the research.
Printed name of child
Signature of parent or individual legally authorized to 
consent to the child’s general medical care
Date
Printed name of parent or individual legally authorized to 
consent to the child’s general medical care
Parent
Individual legally 
authorized to consent to 
the child’s general medical 
care (See note below)
Note: Investigators are to ensure that individuals who are not parents can demonstrate their legal 
authority to consent to the child’s general medical care. Contact legal counsel if any questions arise.


 
I certify that the nature and purpose, the potential benefits and possible risks associated with 
participation in this research study have been explained to the above individual and that any questions 
about this information have been answered. A copy of this document will be given to the subject.
Signature of person obtaining consent
Date
Printed name of person obtaining consent
Assen
t 

Child is birth-6 yrs. old - Assent is not required

Child is 7-17 yrs. old – A separate Assent Document is to be signed by the child

Assent will be obtained Verbally
IRB Approval Period:  
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