RecruitingNot Applicable

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

United States, Argentina, Australia400 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 range

15 Years

Sex

ALL

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

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

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

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 range

15 Years

Sex

ALL

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