Congenital Adrenal HyperplasiaNCT04293133
Final Height in Patients With CAH
An observational study of Congenital Adrenal Hyperplasia, run by Ain Shams University.
Trial summary
Congenital adrenal hyperplasia (CAH) is the most common inherited disorder in the adrenal gland in children. Growth is usually affected in CAH patients either due to the disease itself or treatment consequences. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Congenital Adrenal Hyperplasia
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- 11 Mar 2020to 11 Sep 2020
- 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 Up to 18 years
- All sexes
- Child, Adult
Study population: Patients having a documented history of classical CAH.
Who may be able to join
- Patients having a documented history of classical CAH.
Who cannot join
- Patients with non classical CAH.
- Patients treated with growth hormone.
- Chronic use of medications unrelated to CAH which may affect growth like immunosuppressive drugs like azathioprine and drugs that affect growth hormone release like octreotide, pegvisomant, bromocriptine and cabergoline.
- Other chronic diseases that may affect growth like heart disease, inflammatory bowel disease and renal disease.
- Other causes of adrenal insufficiency.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Final Height in Patients With Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) is the most common inherited disorder in the adrenal gland in children. Growth is usually affected in CAH patients either due to the disease itself or treatment consequences.
Read the detailed description
CAH comprises a group of autosomal recessive disorders caused by a deficiency of one of five enzymes needed for the synthesis of cortisol leading to defect in cortisol synthesis with or without aldosterone deficiency and an increase in the production of adrenocorticotropic hormone through negative feedback.
The most common form is 21-hydroxylase deficiency (21OHD), which forms more than 90 % of the cases.
In classic CAH, 75% of the patients have the salt wasting (SW) and 25% have the non salt-wasting phenotype (NSW).There are no clinical signs at birth in male infants and in female patients, CAH is suspected shortly after birth if there is genital ambiguity, ranging from slight clitromegaly to complete masculinization with acceleration of growth and pubertal development.
The non-classic (late onset) form of CAH is a less severe form of 21OHD, and is diagnosed later in life.
Final height in early and late onset patients has been reported as diminished (Hauffa et al, 1997).This could be attributed to androgen excess or treatment with steroids. Androgen excess can occur at any age leading to accelerated growth, early epiphyseal closure and compromised final adult height.
Despite that all forms of CAH differ in their degree of enzymatic deficiency, they all represent a therapeutic challenge to pediatric endocrinologists attempting to optimize growth.
- Conditions
- Congenital Adrenal Hyperplasia
- Medical subject headings
- Adrenal Hyperplasia, Congenital
What is being measured
The primary outcome is the main question the study is designed to answer.
Final height in patients with congenital adrenal hyperplasia
Target height(TH) will be calculated using the formula: [maternal height + paternal height - 13 cm for girls and + 13 cm for boys]/2
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2020
- Study start11 Mar 2020 (estimated)
- Primary completion11 Aug 2020 (estimated)
- Study completion11 Sep 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Congenital Adrenal HyperplasiaStudies of the same condition, open and under way.
- 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
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ain Shams University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Mohammed Kamel El-Desouky Ashour, Ain Shams University
- Investigators
- Rana Ahmed, Principal investigator, Ain Shams University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04293133
- Sponsor's protocol ID
- CAH
- Status last verified
- Mar 2020
- First submitted
- 2 Mar 2020
- Last updated
- 3 Mar 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Cole TJ, Freeman JV, Preece MA. Body mass index reference curves for the UK, 1990. Arch Dis Child. 1995 Jul;73(1):25-9. doi: 10.1136/adc.73.1.25.
PubMed 7639544Charmandari E, Brook CG, Hindmarsh PC. Classic congenital adrenal hyperplasia and puberty. Eur J Endocrinol. 2004 Nov;151 Suppl 3:U77-82. doi: 10.1530/eje.0.151u077.
PubMed 15554890
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Final Height in Patients With Congenital Adrenal Hyperplasia
- ClinicalTrials.gov ID
- NCT04293133
- Acronym / sponsor ID
- CAH
- Conditions
- Congenital Adrenal Hyperplasia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Ain Shams UniversityUniversity, hospital or other
- Study start
- 11 Mar 2020 (estimated)
- Primary completion
- 11 Aug 2020 (estimated)
- Study completion
- 11 Sep 2020 (estimated)
- First posted
- 3 Mar 2020 (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 the most common inherited disorder in the adrenal gland in children. Growth is usually affected in CAH patients either due to the disease itself or treatment consequences.
Detailed description
CAH comprises a group of autosomal recessive disorders caused by a deficiency of one of five enzymes needed for the synthesis of cortisol leading to defect in cortisol synthesis with or without aldosterone deficiency and an increase in the production of adrenocorticotropic hormone through negative feedback.
The most common form is 21-hydroxylase deficiency (21OHD), which forms more than 90 % of the cases.
In classic CAH, 75% of the patients have the salt wasting (SW) and 25% have the non salt-wasting phenotype (NSW).There are no clinical signs at birth in male infants and in female patients, CAH is suspected shortly after birth if there is genital ambiguity, ranging from slight clitromegaly to complete masculinization with acceleration of growth and pubertal development.
The non-classic (late onset) form of CAH is a less severe form of 21OHD, and is diagnosed later in life.
Final height in early and late onset patients has been reported as diminished (Hauffa et al, 1997).This could be attributed to androgen excess or treatment with steroids. Androgen excess can occur at any age leading to accelerated growth, early epiphyseal closure and compromised final adult height.
Despite that all forms of CAH differ in their degree of enzymatic deficiency, they all represent a therapeutic challenge to pediatric endocrinologists attempting to optimize growth.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- Patients having a documented history of classical CAH.
Key inclusion criteria (1 listed)
- Patients having a documented history of classical CAH.
Key exclusion criteria (5 listed)
- Patients with non classical CAH.
- Patients treated with growth hormone.
- Chronic use of medications unrelated to CAH which may affect growth like immunosuppressive drugs like azathioprine and drugs that affect growth hormone release like octreotide, pegvisomant, bromocriptine and cabergoline.
- Other chronic diseases that may affect growth like heart disease, inflammatory bowel disease and renal disease.
- Other causes of adrenal insufficiency.
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
Final height in patients with congenital adrenal hyperplasia
Time frame: Baseline
Target height(TH) will be calculated using the formula: [maternal height + paternal height - 13 cm for girls and + 13 cm for boys]/2
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2020.
- Background: PubMed 7639544Cole TJ, Freeman JV, Preece MA. Body mass index reference curves for the UK, 1990. Arch Dis Child. 1995 Jul;73(1):25-9. doi: 10.1136/adc.73.1.25.
- Background: PubMed 15554890Charmandari E, Brook CG, Hindmarsh PC. Classic congenital adrenal hyperplasia and puberty. Eur J Endocrinol. 2004 Nov;151 Suppl 3:U77-82. doi: 10.1530/eje.0.151u077.
Related trials for Congenital Adrenal Hyperplasia
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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.
Rana Ahmed principal investigator
Ain Shams University
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Adrenal Hyperplasia, run by Ain Shams 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 1 inclusion and 5 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 3 Mar 2020. 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 Mohammed Kamel (+2001285819500, kamelasbour@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Final height in patients with congenital adrenal hyperplasia (time frame: Baseline). 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 NCT04293133, which also lists 2 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 3 Mar 2020.