Congenital Adrenal HyperplasiaNCT04087148
Linear Growth of Children With Congenital Adrenal Hyperplasia
An observational study of Congenital Adrenal Hyperplasia, run by Assiut University.
Trial summary
The congenital adrenal hyperplasias (CAHs) comprise a family of autosomal recessive disorders that disrupt adrenal steroidogenesis. Three specific enzyme deficiencies are associated with virilization of affected women. Read more about this study
- Who is it for?
- Ages 1 year to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Congenital Adrenal Hyperplasia
- How many people
- 60 participants
- Where
- No sites listed yet
- Study dates
- 24 Sep 2019to 24 Oct 2021
- 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 1 year to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: • pediatric patients with congenital adrenal hyperplasia who are taking glucocorticoid replacement therapy and attending Endocrine unit of Assiut University Children Hospital within one year.
Who may be able to join
- Age: 1-18 y.
- Both sexes.
- All patients who were diagnosed as having CAH of at least 1 y duration.
- On glucocorticoid replacement therapy .
Who cannot join
- Patients diagnosed as CAH for less than 1 year duration.
- Patients with deficient data at the time of diagnosis.
- Patients missed for follow up.
- Syndromatic patients.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Linear Growth of Children With Congenital Adrenal Hyperplasia.
The congenital adrenal hyperplasias (CAHs) comprise a family of autosomal recessive disorders that disrupt adrenal steroidogenesis. Three specific enzyme deficiencies are associated with virilization of affected women. The most common form is 21-hydroxylase deficiency (21-OHD) due to mutations in the 21-hydroxylase (CYP21A2) gene. Other virilizing forms include 3b-hydroxysteroid dehydrogenase type 2 (HSD3B2) and 11b-hydroxylase deficiencies associated with mutations in the HSD3B2 and 11b-hydroxylase (CYP11B1) genes, respectively.
Read the detailed description
It has been reported that approximately one child in every 18000 born in Great Britain has CAH. In North America, the incidence varies from 1:15000 to 1:16000. The reported rates of CAH have been as high as 1:280 among the Yupik people of Alaska and 1:2100 on the French island of Réunion in Indian ocean; both of these populations are geographically isolated. The reported incidence of CAH in the two Brazilian states that have routinely included CAH in their public newborn screening programs is 1:11655 in the South (Santa Catarina) and 1:10325 in Midwest (Goiás).
Salt-losing CAH accounts for about three quartes of cases reported and non-salt losing CAH for one quarter. Non-classic is more common ;Estimated as 1 in 1000-2000 in white populations. It is more frequent in certain ethnic groups, such as the Ashkenazi Jewish population. The mild non-classic form is a common cause of hyperandrogenism.
Treatment of classic 21-OHD consists of replacement doses of gluco- (GC) and mineralocorticoids aiming to reduce excess androgen, and to allow adequate linear growth. However, several series report that growth in these children is below expectation, as compared with both the reference population and the target height (TH).
The reasons for the inadequate growth and impairment of the final height (FH) are not completely understood. A major cause is the difficulty in accomplishing a fine balance between inhibition of excess androgen production which accelerates bone maturation and adequate GC replacement itself which even at slightly supraphysiologic doses can be deleterious to growth.
- Conditions
- Congenital Adrenal Hyperplasia
- 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.
| Study group | Role | What they receive |
|---|---|---|
| patients | patients who were diagnosed as having CAH of at least 1 y duration. and On glucocorticoid replacement therapy . | |
| controls | A comparable number of age and sex matched apparently normal children will be included as control. |
What is being measured
The primary outcome is the main question the study is designed to answer.
factors affecting linear growth in children with CAH.
by using appropriate growth charts and doing follow up wrist x-ray .
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2019
- Study start24 Sep 2019 (estimated)
- Primary completion24 Oct 2020 (estimated)
- Study completion24 Oct 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
- More trials run by Assiut UniversityThe 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
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assiut University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ali Hussin Mohamed, Assiut University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04087148
- Sponsor's protocol ID
- GCAH
- Status last verified
- Sep 2019
- First submitted
- 10 Sep 2019
- Last updated
- 12 Sep 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Delle Piane L, Rinaudo PF, Miller WL. 150 years of congenital adrenal hyperplasia: translation and commentary of De Crecchio's classic paper from 1865. Endocrinology. 2015 Apr;156(4):1210-7. doi: 10.1210/en.2014-1879. Epub 2015 Jan 30.
PubMed 25635623Silveira EL, dos Santos EP, Bachega TA, van der Linden Nader I, Gross JL, Elnecave RH. The actual incidence of congenital adrenal hyperplasia in Brazil may not be as high as inferred--an estimate based on a public neonatal screening program in the state of Goias. J Pediatr Endocrinol Metab. 2008 May;21(5):455-60. doi: 10.1515/jpem.2008.21.5.455.
PubMed 18655527Nunes AK, Wachholz RG, Rover MR, Souza LC. [Prevalence of disorders detected by newborn screening in Santa Catarina]. Arq Bras Endocrinol Metabol. 2013 Jul;57(5):360-7. doi: 10.1590/s0004-27302013000500005. Portuguese.
PubMed 23896802Trapp CM, Oberfield SE. Recommendations for treatment of nonclassic congenital adrenal hyperplasia (NCCAH): an update. Steroids. 2012 Mar 10;77(4):342-6. doi: 10.1016/j.steroids.2011.12.009. Epub 2011 Dec 13.
PubMed 22186144Nebesio TD, Eugster EA. Growth and reproductive outcomes in congenital adrenal hyperplasia. Int J Pediatr Endocrinol. 2010;2010:298937. doi: 10.1155/2010/298937. Epub 2010 Feb 1.
PubMed 20148087
Study at a glance
From the registry record, last updated by the sponsor on 12 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Linear Growth of Children With Congenital Adrenal Hyperplasia.
- ClinicalTrials.gov ID
- NCT04087148
- Acronym / sponsor ID
- GCAH
- Conditions
- Congenital Adrenal Hyperplasia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Assiut UniversityUniversity, hospital or other
- Study start
- 24 Sep 2019 (estimated)
- Primary completion
- 24 Oct 2020 (estimated)
- Study completion
- 24 Oct 2021 (estimated)
- First posted
- 12 Sep 2019 (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.
The congenital adrenal hyperplasias (CAHs) comprise a family of autosomal recessive disorders that disrupt adrenal steroidogenesis. Three specific enzyme deficiencies are associated with virilization of affected women. The most common form is 21-hydroxylase deficiency (21-OHD) due to mutations in the 21-hydroxylase (CYP21A2) gene. Other virilizing forms include 3b-hydroxysteroid dehydrogenase type 2 (HSD3B2) and 11b-hydroxylase deficiencies associated with mutations in the HSD3B2 and 11b-hydroxylase (CYP11B1) genes, respectively.
Detailed description
It has been reported that approximately one child in every 18000 born in Great Britain has CAH. In North America, the incidence varies from 1:15000 to 1:16000. The reported rates of CAH have been as high as 1:280 among the Yupik people of Alaska and 1:2100 on the French island of Réunion in Indian ocean; both of these populations are geographically isolated. The reported incidence of CAH in the two Brazilian states that have routinely included CAH in their public newborn screening programs is 1:11655 in the South (Santa Catarina) and 1:10325 in Midwest (Goiás).
Salt-losing CAH accounts for about three quartes of cases reported and non-salt losing CAH for one quarter. Non-classic is more common ;Estimated as 1 in 1000-2000 in white populations. It is more frequent in certain ethnic groups, such as the Ashkenazi Jewish population. The mild non-classic form is a common cause of hyperandrogenism.
Treatment of classic 21-OHD consists of replacement doses of gluco- (GC) and mineralocorticoids aiming to reduce excess androgen, and to allow adequate linear growth. However, several series report that growth in these children is below expectation, as compared with both the reference population and the target height (TH).
The reasons for the inadequate growth and impairment of the final height (FH) are not completely understood. A major cause is the difficulty in accomplishing a fine balance between inhibition of excess androgen production which accelerates bone maturation and adequate GC replacement itself which even at slightly supraphysiologic doses can be deleterious to growth.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| patients | – | –patients who were diagnosed as having CAH of at least 1 y duration. and On glucocorticoid replacement therapy . |
| controls | – | –A comparable number of age and sex matched apparently normal children will be included as control. |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- • pediatric patients with congenital adrenal hyperplasia who are taking glucocorticoid replacement therapy and attending Endocrine unit of Assiut University Children Hospital within one year.
Key inclusion criteria (4 listed)
- Age: 1-18 y.
- Both sexes.
- All patients who were diagnosed as having CAH of at least 1 y duration.
- On glucocorticoid replacement therapy .
Key exclusion criteria (4 listed)
- Patients diagnosed as CAH for less than 1 year duration.
- Patients with deficient data at the time of diagnosis.
- Patients missed for follow up.
- Syndromatic patients.
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
factors affecting linear growth in children with CAH.
Time frame: 6 months
by using appropriate growth charts and doing follow up wrist x-ray .
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Sep 2019.
- Background: PubMed 25635623Delle Piane L, Rinaudo PF, Miller WL. 150 years of congenital adrenal hyperplasia: translation and commentary of De Crecchio's classic paper from 1865. Endocrinology. 2015 Apr;156(4):1210-7. doi: 10.1210/en.2014-1879. Epub 2015 Jan 30.
- Background: PubMed 18655527Silveira EL, dos Santos EP, Bachega TA, van der Linden Nader I, Gross JL, Elnecave RH. The actual incidence of congenital adrenal hyperplasia in Brazil may not be as high as inferred--an estimate based on a public neonatal screening program in the state of Goias. J Pediatr Endocrinol Metab. 2008 May;21(5):455-60. doi: 10.1515/jpem.2008.21.5.455.
- Background: PubMed 23896802Nunes AK, Wachholz RG, Rover MR, Souza LC. [Prevalence of disorders detected by newborn screening in Santa Catarina]. Arq Bras Endocrinol Metabol. 2013 Jul;57(5):360-7. doi: 10.1590/s0004-27302013000500005. Portuguese.
- Background: PubMed 22186144Trapp CM, Oberfield SE. Recommendations for treatment of nonclassic congenital adrenal hyperplasia (NCCAH): an update. Steroids. 2012 Mar 10;77(4):342-6. doi: 10.1016/j.steroids.2011.12.009. Epub 2011 Dec 13.
- Background: PubMed 20148087Nebesio TD, Eugster EA. Growth and reproductive outcomes in congenital adrenal hyperplasia. Int J Pediatr Endocrinol. 2010;2010:298937. doi: 10.1155/2010/298937. Epub 2010 Feb 1.
- Individual participant dataSharing planned: Undecided
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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.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Adrenal Hyperplasia, run by Assiut University.
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 4 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: status not confirmed, as of the sponsor's last update on 12 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Hanaa Abdellatef Mohammad, Professor of pediatrics (01064747613, hae50@aun.edu.eg), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
factors affecting linear growth in children with CAH. (time frame: 6 months). 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 NCT04087148, which also lists 5 linked publications.
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 12 Sep 2019.