An observational study in Adrenal Hyperplasia, Congenital, sponsored by Hospices Civils de Lyon. Completed at 1 site in France. Open to participants aged 18 Years to 50 Years. Per ClinicalTrials.gov, last updated 2017-05-22.
Sponsored by Hospices Civils de Lyon · Observational
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.
101 studies on the registry are indexed under Adrenal Hyperplasia, Congenital; 28 are open to participants now.
This study's enrollment of 25 is below the median of 61 across 44 observational studies indexed under Adrenal Hyperplasia, Congenital.
Browse Adrenal Hyperplasia, Congenital studies →Hospices Civils de Lyon is the lead sponsor of 1,866 studies on the registry; 448 are open to participants now.
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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.
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Patients with CAH, born between 1970 and 1998, having received GH treatment for a minimal one year duration.
Exclusion Criteria:
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
Time frame: up to 18 years
Number of treatment withdrawal due to adverse events
Safety
Time frame: up to 6 years of GH treatment
Plan to share: No
No publications or documents are linked to this record.
Ran from Sep 2015 to primary completion in Mar 2016, 6 months.
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This study is completed, as verified in May 2017. You cannot join it, but the record below documents what was studied.
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Adrenal Hyperplasia, Congenital →