Growth Hormone (GH) in Congenital Adrenal Hyperplasia (OPALE GH)

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

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.

Study Overview

• Status: Completed
• Conditions: Adrenal Hyperplasia, Congenital

• Study Type: Observational
• Enrollment (Actual): 25

Contacts and Locations

Study Locations

• France
  • Lyon, France, 69500
    • Hospices Civils de Lyon

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 50 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

Patients with genetically proven CAH, classical form, or simple virilizing, with 21 hydroxylase, or 11ᵝ hydroxylase, or 3βol-dehydrogenase deficit, born between 1970 and 1998, having received GH treatment for a minimal one year duration.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of treatment withdrawal due to adverse events	Safety	up to 6 years of GH treatment

Collaborators and Investigators

• Sponsor: Hospices Civils de Lyon
• Investigators: Principal Investigator: Patricia Bretones, MD, Hospices Civils de Lyon

Study record dates

Study Major Dates

• Study Start (Actual): September 15, 2015
• Primary Completion (Actual): March 15, 2016
• Study Completion (Actual): March 15, 2016

Study Registration Dates

• First Submitted: May 16, 2017
• First Submitted That Met QC Criteria: May 19, 2017
• First Posted (Actual): May 22, 2017

Study Record Updates

• Last Update Posted (Actual): May 22, 2017
• Last Update Submitted That Met QC Criteria: May 19, 2017
• Last Verified: May 1, 2017

More Information

Terms related to this study

• Keywords: short stature; growth hormone treatment; congenital adrenal hyperplasia
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Endocrine System Diseases; Gonadal Disorders; Disorders of Sex Development; Urogenital Abnormalities; Congenital Abnormalities; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Adrenal Gland Diseases; Steroid Metabolism, Inborn Errors; Hyperplasia; Adrenal Hyperplasia, Congenital; Adrenogenital Syndrome; Adrenocortical Hyperfunction
• Other Study ID Numbers: CIC 2009-019

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No