Adrenal Hyperplasia, CongenitalOPALE GHNCT03162172
Growth Hormone (GH) in Congenital Adrenal Hyperplasia
An observational study of Adrenal Hyperplasia, Congenital, run by Hospices Civils de Lyon at 1 site in France.
Trial summary
Congenital adrenal hyperplasia (CAH) is a genetic rare disease, which alters the adrenal production of gluco and mineralo corticoids. The treatment consists in supplementing children using hydrocortisone. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Adrenal Hyperplasia, Congenital
- How many people
- 25 participants
- Where
- 1 siteFrance
- Study dates
- 15 Sep 2015to 15 Mar 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Study population: Patients with genetically proven CAH, classical form, or simple virilizing, with 21 hydroxylase, or 11ᵝ hydroxylase, or 3βol-dehydrogenase deficit, born between 1970 and 1998, having received GH treatment for a minimal one year duration.
Who may be able to join
- Patients with CAH, born between 1970 and 1998, having received GH treatment for a minimal one year duration.
Who cannot join
- Patients with chronic any growth altering disease, Turner syndrome or other genetic anomaly; 8-year wrist Xray and adult height should be available to allow the use of the OPALE model prediction.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Evaluation of the Adult Height Gain With Growth Hormone Treatment in Children With Congenital Adrenal Hyperplasia (CDAH), Using the OPALE Prediction Model
Congenital adrenal hyperplasia (CAH) is a genetic rare disease, which alters the adrenal production of gluco and mineralo corticoids. The treatment consists in supplementing children using hydrocortisone. Despite care for these children has improve substantially across decades, short adult height still remains an important consequence of the disease. About 20 % of patients have an AH below 2 standard deviations compared to their expected height.
In the OPALE model study, the investigators have collected data from a cohort of 496 French patients, born between 1970 and 1991 and with a known genotype. Using their age, sex, growth, disease, bone maturation and pubertal data, they have built a model which allows to predict their AH using data available at 8 years of age. This model has shown that the currently used formula to calculate the predicted AH (Bayley Pineau's method) is not applicable to children with CAH.
Read more
In this project, the investigators plan to use the prediction model to compare the AH in patients who have received GH treatment to their predicted AH using the model.
The hypothesis is that GH improves the AH in such patients. Existing cohorts have shown improved growth celerity, and growth expectation using the Bayley-Pineau formula), but this has not been shown on the actual AH.
This study will allow to reinforce the investigators' hypothesis.
- Conditions
- Adrenal Hyperplasia, Congenital
- Keywords
- congenital adrenal hyperplasia, short stature, growth hormone treatment
- Medical subject headings
- Adrenal Hyperplasia, Congenital, Dwarfism
What is being measured
The primary outcome is the main question the study is designed to answer.
Adult height (AH) gain
Difference between AH predicted by the OPALE model, and observed AH defined as (i) the height recorded after age 20 in boys or 18 in girls; (ii) the height recorded when bone age (BA) is ≥ 18 years in boys and 16 years in girls (99.6% of AH) (10); or (iii) the height measured after growth velocity drop to ≤ 1 cm/year
Measured: up to 18 years
Secondary outcomes (1)
Number of treatment withdrawal due to adverse events
Safety
Measured: up to 6 years of GH treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2017
- Study start15 Sep 2015
- Primary completion15 Mar 2016
- Study completion15 Mar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Adrenal Hyperplasia, CongenitalStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Patricia Bretones, MD, Principal investigator, Hospices Civils de Lyon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03162172
- Sponsor's protocol ID
- CIC 2009-019
- Acronym
- OPALE GH
- Status last verified
- May 2017
- First submitted
- 16 May 2017
- Last updated
- 22 May 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Adult Height Gain With Growth Hormone Treatment in Children With Congenital Adrenal Hyperplasia (CDAH), Using the OPALE Prediction Model
- ClinicalTrials.gov ID
- NCT03162172
- Acronym / sponsor ID
- OPALE GH / CIC 2009-019
- Conditions
- Adrenal Hyperplasia, Congenital
- Keywords
- congenital adrenal hyperplasia, short stature, growth hormone treatment
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 15 Sep 2015 (actual)
- Primary completion
- 15 Mar 2016 (actual)
- Study completion
- 15 Mar 2016 (actual)
- First posted
- 22 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Congenital adrenal hyperplasia (CAH) is a genetic rare disease, which alters the adrenal production of gluco and mineralo corticoids. The treatment consists in supplementing children using hydrocortisone. Despite care for these children has improve substantially across decades, short adult height still remains an important consequence of the disease. About 20 % of patients have an AH below 2 standard deviations compared to their expected height.
In the OPALE model study, the investigators have collected data from a cohort of 496 French patients, born between 1970 and 1991 and with a known genotype. Using their age, sex, growth, disease, bone maturation and pubertal data, they have built a model which allows to predict their AH using data available at 8 years of age. This model has shown that the currently used formula to calculate the predicted AH (Bayley Pineau's method) is not applicable to children with CAH.
In this project, the investigators plan to use the prediction model to compare the AH in patients who have received GH treatment to their predicted AH using the model.
The hypothesis is that GH improves the AH in such patients. Existing cohorts have shown improved growth celerity, and growth expectation using the Bayley-Pineau formula), but this has not been shown on the actual AH.
This study will allow to reinforce the investigators' hypothesis.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Patients with genetically proven CAH, classical form, or simple virilizing, with 21 hydroxylase, or 11ᵝ hydroxylase, or 3βol-dehydrogenase deficit, born between 1970 and 1998, having received GH treatment for a minimal one year duration.
Key inclusion criteria (1 listed)
- Patients with CAH, born between 1970 and 1998, having received GH treatment for a minimal one year duration.
Key exclusion criteria (1 listed)
- Patients with chronic any growth altering disease, Turner syndrome or other genetic anomaly; 8-year wrist Xray and adult height should be available to allow the use of the OPALE model prediction.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Adult height (AH) gain
Time frame: up to 18 years
Difference between AH predicted by the OPALE model, and observed AH defined as (i) the height recorded after age 20 in boys or 18 in girls; (ii) the height recorded when bone age (BA) is ≥ 18 years in boys and 16 years in girls (99.6% of AH) (10); or (iii) the height measured after growth velocity drop to ≤ 1 cm/year
1 secondary outcome
Number of treatment withdrawal due to adverse events
Time frame: up to 6 years of GH treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2017.
- Individual participant dataSharing planned: No
Related trials for Adrenal Hyperplasia, Congenital
RecruitingPhase 2/3
A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Natural History Study of Patients With Excess Androgen
Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
RecruitingPhase 3
A Study to Evaluate Atumelnant in Adults With Congenital Adrenal Hyperplasia
Congenital Adrenal Hyperplasia, Classic Congenital Adrenal Hyperplasia
RecruitingObservational study
Adrenal Diseases in the Population of Modena: A Clinical-Epidemiological Database of Patients Referred to the Adrenal Disorders Outpatient Clinic of the Clinical Unit of Endocrinology at the Modena University Hospital (AOU Modena)
Cushing s Syndrome, Cushing Disease
RecruitingPhase 4
Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)
Mild Autonomous Cortisol Secretion (MACS)
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.
Patricia Bretones, MD principal investigator
Hospices Civils de Lyon
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Adrenal Hyperplasia, Congenital, run by Hospices Civils de Lyon at 1 site in France.
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 1 inclusion and 1 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 22 May 2017. 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 France. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Adult height (AH) gain (time frame: up to 18 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 22 May 2017.