Adrenal Hyperplasia, CongenitalPhase 2NCT00621985
Dexamethasone Treatment of Congenital Adrenal Hyperplasia
A Phase 2 study testing dexamethasone and Hydrocortisone in people with Adrenal Hyperplasia, Congenital, run by Boston Children's Hospital at 1 site in United States.
Trial summary
The purpose of this study is to determine if dexamethasone given at night is a more effective treatment for congenital adrenal hyperplasia in young children than standard three times per day hydrocortisone. Read more about this study
- Who is it for?
- Ages 2 to 9 years
- Conditions the study is for
- Adrenal Hyperplasia, Congenital
- What is being tested?
- dexamethasone and Hydrocortisone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2008to Jun 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 9 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Classic salt-wasting 21-hydroxylase deficient congenital adrenal hyperplasia
- Pre-pubertal children with bone ages below 8 years
Who cannot join
- Age less than 2 years
- Patients with additional medical conditions necessitating glucocorticoid therapy.
- Patients on phenytoin, barbiturates, and rifampin as these medications accelerate the metabolism of glucocorticoids.
- Patients on ketoconazole as this medication increases the bioavailability of glucocorticoids.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital Boston
Boston, Massachusetts, 02115
About this study
The purpose of this study is to determine if dexamethasone given at night is a more effective treatment for congenital adrenal hyperplasia in young children than standard three times per day hydrocortisone. Our hypothesis is that nocturnal dexamethasone will lead to more efficient suppression of the hypothalamic-pituitary-adrenal axis. We performed a cross-over trial comparing hormonal control during two 24-hour hospitalizations, one on hydrocortisone and one on nocturnal dexamethasone.
Read the detailed description
This is a Phase II clinical trial, intended to estimate the effect of instituting Dexamethasone therapy in comparison to prior standard therapy. Each subject provides his own baseline data. There is no control group. Patients with CAH who meet inclusion criteria will be admitted to the clinical research center for two 24 hour hospitalizations. Adrenal hormone profiles will be measured during each hospitalization. The patient will take his or her baseline hydrocortisone regimen during one hospitalization and a new regimen consisting of a single daily nocturnal dose of Dexamethasone during the second hospitalization.
- Conditions
- Adrenal Hyperplasia, Congenital
- Being tested
- dexamethasone, Hydrocortisone
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
dexamethasoneDrug
Also called Decadron
Dexamethasone will be given at a dose that equals 1/50 of the total daily hydrocortisone dose of the patient. It will be given in solution form at 10 PM for 3 days.
HydrocortisoneDrug
Subjects were given their baseline hydrocortisone regimen which was three times daily for 4 of the subjects and twice daily for one subject. Doses were given at 8 AM, 2 PM, and 8 PM. The 2 PM time point was skipped for the subject who received hydrocortisone twice daily. Doses ranged from 6.9 to 18.5 milligrams per meter squared per day and were based on each individual's baseline regimen.
| Study group | Role | What they receive |
|---|---|---|
| Experimental | Experimental | Experimental therapy with nocturnal dexamethasone. Receives: Drug: dexamethasone; Drug: Hydrocortisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Difference in the Mean Log Transformed Area Under the Curve of 17-hydroxyprogesterone Between Regimens
Each subject was admitted for 2 24 hour hospitalizations, one on hydrocortisone and one on dexamethasone. Due to the timing of blood draws, the serum hormonal profile was only measured for 23 hours. The primary outcome was the Percent Difference in the Mean log transformed Area under the curve of 17-hydroxyprogesterone between the two regimens.
Measured: 23 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2008 (estimated)
- Study startApr 2008
- Primary completionJun 2009
- Study completionJun 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Adrenal Hyperplasia, CongenitalStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Investigators
- Joseph Majzoub, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00621985
- Sponsor's protocol ID
- 08-01-0025
- Status last verified
- Jan 2011
- First submitted
- 11 Feb 2008
- Last updated
- 23 Feb 2011
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Dauber A, Feldman HA, Majzoub JA. Nocturnal Dexamethasone versus Hydrocortisone for the Treatment of Children with Congenital Adrenal Hyperplasia. Int J Pediatr Endocrinol. 2010;2010:347636. doi: 10.1155/2010/347636. Epub 2010 Sep 14.
PubMed 20871859
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Dexamethasone Treatment of Congenital Adrenal Hyperplasia
- ClinicalTrials.gov ID
- NCT00621985
- Acronym / sponsor ID
- 08-01-0025
- Conditions
- Adrenal Hyperplasia, Congenital
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Study start
- Apr 2008
- Primary completion
- Jun 2009 (actual)
- Study completion
- Jun 2009 (actual)
- First posted
- 22 Feb 2008 (estimated)
- Results
- Posted 23 Feb 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine if dexamethasone given at night is a more effective treatment for congenital adrenal hyperplasia in young children than standard three times per day hydrocortisone. Our hypothesis is that nocturnal dexamethasone will lead to more efficient suppression of the hypothalamic-pituitary-adrenal axis. We performed a cross-over trial comparing hormonal control during two 24-hour hospitalizations, one on hydrocortisone and one on nocturnal dexamethasone.
Detailed description
This is a Phase II clinical trial, intended to estimate the effect of instituting Dexamethasone therapy in comparison to prior standard therapy. Each subject provides his own baseline data. There is no control group. Patients with CAH who meet inclusion criteria will be admitted to the clinical research center for two 24 hour hospitalizations. Adrenal hormone profiles will be measured during each hospitalization. The patient will take his or her baseline hydrocortisone regimen during one hospitalization and a new regimen consisting of a single daily nocturnal dose of Dexamethasone during the second hospitalization.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental | Experimental | Drug: dexamethasone, Drug: HydrocortisoneExperimental therapy with nocturnal dexamethasone. |
Who may qualify
- Ages
- 2 to 9 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Classic salt-wasting 21-hydroxylase deficient congenital adrenal hyperplasia
- Pre-pubertal children with bone ages below 8 years
Key exclusion criteria (4 listed)
- Age less than 2 years
- Patients with additional medical conditions necessitating glucocorticoid therapy.
- Patients on phenytoin, barbiturates, and rifampin as these medications accelerate the metabolism of glucocorticoids.
- Patients on ketoconazole as this medication increases the bioavailability of glucocorticoids.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| dexamethasoneAlso: DecadronDexamethasone will be given at a dose that equals 1/50 of the total daily hydrocortisone dose of the patient. It will be given in solution form at 10 PM for 3 days. | Drug | Experimental |
| HydrocortisoneSubjects were given their baseline hydrocortisone regimen which was three times daily for 4 of the subjects and twice daily for one subject. Doses were given at 8 AM, 2 PM, and 8 PM. The 2 PM time point was skipped for the subject who received hydrocortisone twice daily. Doses ranged from 6.9 to 18.5 milligrams per meter squared per day and were based on each individual's baseline regimen. | Drug | Experimental |
Primary outcome
Percent Difference in the Mean Log Transformed Area Under the Curve of 17-hydroxyprogesterone Between Regimens
Time frame: 23 hours
Each subject was admitted for 2 24 hour hospitalizations, one on hydrocortisone and one on dexamethasone. Due to the timing of blood draws, the serum hormonal profile was only measured for 23 hours. The primary outcome was the Percent Difference in the Mean log transformed Area under the curve of 17-hydroxyprogesterone between the two regimens.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 20871859Dauber A, Feldman HA, Majzoub JA. Nocturnal Dexamethasone versus Hydrocortisone for the Treatment of Children with Congenital Adrenal Hyperplasia. Int J Pediatr Endocrinol. 2010;2010:347636. doi: 10.1155/2010/347636. Epub 2010 Sep 14.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joseph Majzoub, MD principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing dexamethasone and Hydrocortisone in people with Adrenal Hyperplasia, Congenital, run by Boston Children's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Feb 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Feb 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Difference in the Mean Log Transformed Area Under the Curve of 17-hydroxyprogesterone Between Regimens (time frame: 23 hours). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00621985, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Feb 2011.