Adult Height Prediction in Congenital Adrenal Hyperplasia

Completed

• Conditions: Congenital Adrenal Hyperplasia
• Interventions: Other: Collection of growth and bone maturation data

• Registration Number: NCT03162159
• Lead Sponsor: Hospices Civils de Lyon

Brief Summary

Congenital Adrenal Hyperplasia (CAH) is a genetic rare disease, which alters the adrenal production of gluco and mineralo corticoïds. The treatment consists in supplementing children with hydrocortisone. Despite care for these children has improved substantially across decades, short adult height (AH) still remains an important consequence of the disease. About 20% of patients have an AH below 2 standard deviations compared to their expected AH.

In the OPALE-Model study, the investigators want to collect data from a cohort of 496 CAH French patients, born between 1970 and 1991 with a known genotype. Using their age, sex, growth, disease, bone maturation and pubertal data, the investigators will build a model which allows to predict their AH using data available at 8 years of age. The growth charts built from this cohort have shown that currently used formula to calculate the predicted AH (Bayley-Pineau's formula) is not applicable to children with CAH.

In this project, the investigators plan to compute an AH prediction model using data from children born between 1970 and 1993, and to validate the model using data from a different cohort (i.e. children born between 1994 and 1998). this choice was due to availability of data for computing the model first, and in a second stage, data from more recently born patients.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-09
• Study First Submitted: 2017-05-11
• Study First Submitted QC: 2017-05-19
• Study First Posted: 2017-05-22
• Primary Completion: 2018-12-31
• Study Completion: 2018-12-31
• Status Verified: 2020-08
• Last Update Submitted: 2020-08-10
• Last Update Posted: 2020-08-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 496

Inclusion Criteria

• children with CAH, genetically proven, classical form, virilizing form, with deficit of 21 hydroxylase, 11 beta hydroxylase, or 3 beta ol dehydrogenase, born between 1972-1993 (cohort 1) and 1994-1998 (cohort 2).
• medical charts should be available.

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Exclusion Criteria

• Patients with chronic growth altering disease, Turner syndrome, or other genetic anomaly
• Patients having received any treatment with Growth Hormone (GH)

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
cohort for model computing	Collection of growth and bone maturation data	patients with CAH, born between 1970 and 1993, with genetically proven CAH, available growth and bone maturation data.
cohort for model validation	Collection of growth and bone maturation data	patients with CAH, born between 1994 and 1998, with genetically proven CAH, available growth and bone maturation data.

Primary Outcome Measures

Name	Time	Method
Adult height (AH)	up to 18 years	AH is defined as i) the height recorded after age 20 in boys or 18 in girls; ii) Or the height recorded when bone age is \>= 18 years in boys and 16 years in girls; Or iii) the height measured after growth velocity dropped to \<= 1 cm/year.

Trial Locations

Locations (1)

Hospices Civils de Lyon; 🇫🇷 Lyon, France