Growth Hormone (GH) in Congenital Adrenal Hyperplasia

Adrenal Hyperplasia, Congenital Clinical Trial

— OPALE GH  

Official title:

Evaluation of the Adult Height Gain With Growth Hormone Treatment in Children With Congenital Adrenal Hyperplasia (CDAH), Using the OPALE Prediction Model

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03162172
• Other study ID #: CIC 2009-019
• Status: Completed
• Phase: N/A
• First received: May 16, 2017
• Last updated: May 19, 2017
• Start date: September 15, 2015
• Est. completion date: March 15, 2016

Study information

• Verified date: May 2017
• 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 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 25
• Est. completion date: March 15, 2016
• Est. primary completion date: March 15, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 50 Years

Eligibility

Inclusion Criteria:

Patients with CAH, born between 1970 and 1998, having received GH treatment for a minimal one year duration.

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

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

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, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	up to 18 years
Secondary	Number of treatment withdrawal due to adverse events	Safety	up to 6 years of GH treatment