RecruitingNot Applicable

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

United States400 participantsStarted 2023-11-27

Plain-language 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.

Who can participate

Age range15 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Change in annualized growth velocity (AGV)

Timeframe: Every 6 months through end of study, up to 15 years

Change in Height Z-score

Timeframe: Every 6 months through end of study, up to 15 years

Change in Height

Timeframe: Every 6 months through end of study, up to 15 years

Change in BMI

Timeframe: Every 6 months through end of study, up to 15 years

Change in BMI Z-score

Timeframe: Every 6 months through end of study, up to 15 years

Change in ratios of upper to lower body segments

Timeframe: Every 6 months through end of study, up to 15 years

Change in ratios of upper and lower leg length ratio

Timeframe: Every 6 months through end of study, up to 15 years

Change in ratio of arms span to standing height ratio

Timeframe: Every 6 months through end of study, up to 15 years

Trial details

NCT IDNCT06212947

SponsorBioMarin Pharmaceutical

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2043-12-31

Contact for this trial

Trial Specialist

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

View on ClinicalTrials.gov More Hypochondroplasia trials

Who can participate

Age range15 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Change in annualized growth velocity (AGV)

Timeframe: Every 6 months through end of study, up to 15 years

Change in Height Z-score

Timeframe: Every 6 months through end of study, up to 15 years

Change in Height

Timeframe: Every 6 months through end of study, up to 15 years

Change in BMI

Timeframe: Every 6 months through end of study, up to 15 years

Change in BMI Z-score

Timeframe: Every 6 months through end of study, up to 15 years

Change in ratios of upper to lower body segments

Timeframe: Every 6 months through end of study, up to 15 years

Change in ratios of upper and lower leg length ratio

Timeframe: Every 6 months through end of study, up to 15 years

Change in ratio of arms span to standing height ratio

Timeframe: Every 6 months through end of study, up to 15 years