Growth Hormone (GH) in Congenital Adrenal Hyperplasia

NCT03162172 | Completed

• 1 other identifier: CIC 2009-019
• Study Type: observational
• Target: 25
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Adrenal Hyperplasia, Congenital

Keywords

congenital adrenal hyperplasia; short stature; growth hormone treatment

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Number of treatment withdrawal due to adverse events

  up to 6 years of GH treatment

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)
• 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.

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Hospices Civils de Lyon: lead

Study Sites (1)

Hospices Civils de Lyon

Lyon, 69500, France

Location

MeSH Terms

Conditions

Adrenal Hyperplasia, Congenital; Dwarfism

Condition Hierarchy (Ancestors)

Adrenogenital Syndrome; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Steroid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Adrenal Gland Diseases; Endocrine System Diseases; Gonadal Disorders; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases

Study Officials

• Patricia Bretones, MD

  Hospices Civils de Lyon

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2017
• First Posted: May 22, 2017
• Study Start: September 15, 2015
• Primary Completion: March 15, 2016
• Study Completion: March 15, 2016
• Last Updated: May 22, 2017

Record last verified: 2017-05

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)