Adult Height Prediction in Congenital Adrenal Hyperplasia

NCT ID: NCT03162159

Last Updated: 2020-08-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

496 participants

Study Classification

OBSERVATIONAL

Study Start Date

2010-09-30

Study Completion Date

2018-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Congenital Adrenal Hyperplasia

Study Design

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Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

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cohort for model computing

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

Collection of growth and bone maturation data

Intervention Type OTHER

cohort for model validation

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

Collection of growth and bone maturation data

Intervention Type OTHER

Interventions

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Collection of growth and bone maturation data

Intervention Type OTHER

Eligibility Criteria

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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)
Minimum Eligible Age

18 Years

Maximum Eligible Age

40 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Hospices Civils de Lyon

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Catherine Cornu, MD

Role: STUDY_CHAIR

Hospices Civils de Lyon

Locations

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Hospices Civils de Lyon

Lyon, , France

Site Status

Countries

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France

References

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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.

Reference Type BACKGROUND
PMID: 27852974 (View on PubMed)

Other Identifiers

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CE-CIC-GREN-09-19

Identifier Type: -

Identifier Source: org_study_id

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