Adult Height Prediction in Congenital Adrenal Hyperplasia (OPALE Model)
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| ClinicalTrials.gov Identifier: NCT03162159 |
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Recruitment Status :
Completed
First Posted : May 22, 2017
Last Update Posted : August 11, 2020
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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.
| Condition or disease | Intervention/treatment |
|---|---|
| Congenital Adrenal Hyperplasia | Other: Collection of growth and bone maturation data |
| Study Type : | Observational |
| Actual Enrollment : | 496 participants |
| Observational Model: | Cohort |
| Time Perspective: | Retrospective |
| Official Title: | An Adult Height Prediction Model for Congenital Adrenal Hyperplasia From a National Cohort (OPALE Model Study) |
| Actual Study Start Date : | September 2010 |
| Actual Primary Completion Date : | December 31, 2018 |
| Actual Study Completion Date : | December 31, 2018 |
| Group/Cohort | Intervention/treatment |
|---|---|
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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.
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Other: Collection of growth and bone maturation data |
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cohort for model validation
patients with CAH, born between 1994 and 1998, with genetically proven CAH, available growth and bone maturation data.
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Other: Collection of growth and bone maturation data |
- Adult height (AH) [ Time Frame: up to 18 years ]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.
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| Ages Eligible for Study: | 18 Years to 40 Years (Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03162159
| France | |
| Hospices Civils de Lyon | |
| Lyon, France, 69500 | |
| Study Chair: | Catherine Cornu, MD | Hospices Civils de Lyon |
| Responsible Party: | Hospices Civils de Lyon |
| ClinicalTrials.gov Identifier: | NCT03162159 |
| Other Study ID Numbers: |
CE-CIC-GREN-09-19 |
| First Posted: | May 22, 2017 Key Record Dates |
| Last Update Posted: | August 11, 2020 |
| Last Verified: | August 2020 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Adrenal Hyperplasia, Congenital Adrenogenital Syndrome Adrenocortical Hyperfunction Hyperplasia Pathologic Processes Disorders of Sex Development Urogenital Abnormalities Congenital Abnormalities |
Genetic Diseases, Inborn Steroid Metabolism, Inborn Errors Metabolism, Inborn Errors Metabolic Diseases Adrenal Gland Diseases Endocrine System Diseases Gonadal Disorders |