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CompletedNCT03162159OPALE ModelUpdated Aug 11, 2020

Adult Height Prediction in Congenital Adrenal Hyperplasia

An observational study in Congenital Adrenal Hyperplasia, sponsored by Hospices Civils de Lyon. Completed at 1 site in France. Open to participants aged 18 Years to 40 Years. Per ClinicalTrials.gov, last updated 2020-08-11.

Sponsored by Hospices Civils de Lyon · Observational

Study type
Observational
Model
Cohort
Time perspective
Retrospective
Enrollment
496
Ages
18 Years to 40 Years
Sex
All
01

Study 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.

02

Conditions studied

03

Who can participate

Ages eligible
18 Years to 40 Years
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Adullts with Congenital Adrenal Hyperplasia

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

Exclusion Criteria:

  • Patients with chronic growth altering disease, Turner syndrome, or other genetic anomaly
  • Patients having received any treatment with Growth Hormone (GH)
04

Study design

Observational model
Cohort
Time perspective
Retrospective
Enrollment
496 participants (actual)
Patient registry
No

Groups and cohorts

  • cohort for model computing

    patients with CAH, born between 1970 and 1993, with genetically proven CAH, available growth and bone maturation data.

    Other: Collection of growth and bone maturation data

  • cohort for model validation

    patients with CAH, born between 1994 and 1998, with genetically proven CAH, available growth and bone maturation data.

    Other: Collection of growth and bone maturation data

Interventions

  • OtherCollection of growth and bone maturation data
05

What researchers measure

Primary outcomes

  1. 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.

    Time frame: up to 18 years

06

Study locations

1 site
  • Hospices Civils de Lyon
    Lyon, 69500, France
07

References and documents

Publications

  • 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. PubMed 27852974 ↗

Individual participant data

Plan to share: No

08

Registry details

Key details

Study ID
NCT03162159
Lead sponsor
Hospices Civils de Lyon
Responsible party
Sponsor
First posted
May 22, 2017
Start date
Sep 2010
Primary completion
Dec 31, 2018
Completion
Dec 31, 2018
Last update
Aug 11, 2020

Study contacts

Catherine Cornu, MD
study chair · Hospices Civils de Lyon

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Aug 2020. You cannot join it, but the record below documents what was studied.

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