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Trial record 18 of 108 for:    "21-hydroxylase deficiency"

Adult Height Prediction in Congenital Adrenal Hyperplasia (OPALE Model)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03162159
Recruitment Status : Unknown
Verified May 2017 by Hospices Civils de Lyon.
Recruitment status was:  Recruiting
First Posted : May 22, 2017
Last Update Posted : May 22, 2017
Sponsor:
Information provided by (Responsible Party):
Hospices Civils de Lyon

Tracking Information
First Submitted Date May 11, 2017
First Posted Date May 22, 2017
Last Update Posted Date May 22, 2017
Actual Study Start Date September 2010
Actual Primary Completion Date December 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 19, 2017)
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.
Original Primary Outcome Measures Same as current
Change History No Changes Posted
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Adult Height Prediction in Congenital Adrenal Hyperplasia
Official Title An Adult Height Prediction Model for Congenital Adrenal Hyperplasia From a National Cohort (OPALE Model Study)
Brief 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.

Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Retrospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Adullts with Congenital Adrenal Hyperplasia
Condition Congenital Adrenal Hyperplasia
Intervention Other: Collection of growth and bone maturation data
Study Groups/Cohorts
  • cohort for model computing
    patients with CAH, born between 1970 and 1993, with genetically proven CAH, available growth and bone maturation data.
    Intervention: 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.
    Intervention: Other: Collection of growth and bone maturation data
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Unknown status
Estimated Enrollment
 (submitted: May 19, 2017)
600
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 2018
Actual Primary Completion Date December 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria

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)
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years to 40 Years   (Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries France
Removed Location Countries  
 
Administrative Information
NCT Number NCT03162159
Other Study ID Numbers CE-CIC-GREN-09-19
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Hospices Civils de Lyon
Study Sponsor Hospices Civils de Lyon
Collaborators Not Provided
Investigators Not Provided
PRS Account Hospices Civils de Lyon
Verification Date May 2017