Recruiting

Observational Study

Sponsor:

BioMarin Pharmaceutical

Code:

NCT06212947

Conditions

Hypochondroplasia

Eligibility Criteria

Sex: All

Age: 0 - 15

Healthy Volunteers: Not accepted

Study Details

Brief summary:

This study will assess growth over time and the clinical course of HCH in children by collecting growth measurements and other variables of interest.

Conditions

Hypochondroplasia

Study ID

NCT06212947

Start date

Nov 27, 2023

Status verified date

Apr, 2025

Completion date

Dec 31, 2043

Anticipated

Primary completion date

Dec 31, 2043

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 15

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Participants must be ≤ 15 years old at the time of signing the informed consent
  • Participants must have genetic confirmation of Hypochondroplasia diagnosis

Exclusion Criteria:

  • Have a diagnosis of another genetic short stature condition other than Hypochondroplasia or a genetic variant known to cause another genetic syndrome associated with short stature
  • Received an investigational product or medical device within 6 months before the Screening visit

Study Design

Enrollment

400 participants

Anticipated

Interventions and Outcome Measures

Arms

Children with Hypochondroplasia

Children with Hypochondroplasia

Primary outcome measure

  • Change in annualized growth velocity (AGV) [ Time Frame: Every 6 months through end of study, up to 15 years ]
  • Change in Height Z-score [ Time Frame: Every 6 months through end of study, up to 15 years ]
  • Change in Height [ Time Frame: Every 6 months through end of study, up to 15 years ]
  • Change in BMI [ Time Frame: Every 6 months through end of study, up to 15 years ]
  • Change in BMI Z-score [ Time Frame: Every 6 months through end of study, up to 15 years ]
  • Change in ratios of upper to lower body segments [ Time Frame: Every 6 months through end of study, up to 15 years ]
  • Change in ratios of upper and lower leg length ratio [ Time Frame: Every 6 months through end of study, up to 15 years ]
  • Change in ratio of arms span to standing height ratio [ Time Frame: Every 6 months through end of study, up to 15 years ]

Central Contacts and Locations

Central contacts

Trial Specialist

1-800-983-4587medinfo@bmrn.com

Locations

Phoenix Children's Hospital

Recruiting

Phoenix, Arizona, United States, 85016

UCSF Benioff Children's Hospital

Recruiting

Oakland, California, United States, 94609

Nemours Alfred I. Dupont Hospital for Children

Recruiting

Wilmington, Delaware, United States, 19803

Children's National Health System

Recruiting

Washington, District of Columbia, United States, 20010

Ann & Robert H. Lurie Children's Hospital of Chicago

Recruiting

Chicago, Illinois, United States, 60611

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

University of Minnesota Masonic Children's Hospital

Recruiting

Minneapolis, Minnesota, United States, 55454

University of Missouri

Recruiting

Columbia, Missouri, United States, 65212

Washington University School of Medicine in St. Louis

Recruiting

Saint Louis, Missouri, United States, 63110

Mount Sinai Kravis Children's Hospital

Recruiting

New York, New York, United States, 10029

Cook Children's Endocrinology

Recruiting

Fort Worth, Texas, United States, 76104

Medical College of Wisconsin, Children's Hospital

Recruiting

Milwaukee, Wisconsin, United States, 53226

Stollery Children's Hospital

Recruiting

Edmonton, Alberta, Canada

SickKids - The Hospital for Sick Children

Recruiting

Toronto, Ontario, Canada

More Information

Sponsor

BioMarin Pharmaceutical

Last update posted

Apr 3, 2025

Last verified

Apr, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by BioMarin Pharmaceutical on 2025-04-03.