Adult Height Prediction in Congenital Adrenal Hyperplasia

Congenital Adrenal Hyperplasia Clinical Trial

— OPALE Model  

Official title:

An Adult Height Prediction Model for Congenital Adrenal Hyperplasia From a National Cohort (OPALE Model Study)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03162159
• Other study ID #: CE-CIC-GREN-09-19
• Status: Completed
• Start date: September 2010
• Est. completion date: December 31, 2018

Study information

• Verified date: August 2020
• Source: Hospices Civils de Lyon
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 496
• Est. completion date: December 31, 2018
• Est. primary completion date: December 31, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 40 Years

Eligibility

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.

Exclusion Criteria:

• Patients with chronic growth altering disease, Turner syndrome, or other genetic anomaly

• Patients having received any treatment with Growth Hormone (GH)

Related Conditions & MeSH terms

• Adrenal Hyperplasia, Congenital
• Adrenocortical Hyperfunction
• Adrenogenital Syndrome
• Congenital Adrenal Hyperplasia
• Hyperplasia

Intervention

Other:
• Collection of growth and bone maturation data

Locations

Country	Name	City	State
France	Hospices Civils de Lyon	Lyon

Sponsors (1)

Lead Sponsor	Collaborator
Hospices Civils de Lyon

Country where clinical trial is conducted

France, 

References & Publications (1)

Bretones P, Riche B, Pichot E, David M, Roy P, Tardy V, Kassai B, Gaillard S, Bernoux D, Morel Y, Chatelain P, Nicolino M, Cornu C; French Collaborative CAH Growth Study Group. Growth curves for congenital adrenal hyperplasia from a national retrospective cohort. J Pediatr Endocrinol Metab. 2016 Dec 1;29(12):1379-1388. doi: 10.1515/jpem-2016-0156. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Adult height (AH)	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.	up to 18 years