NCT06212947 - A Multicenter Multinational Observational Study of Children With Hypochondroplasia | Crick | Crick

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

N/ARecruitingNCT06212947

BioMarin Pharmaceutical400 enrolled

Overview

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

Study Type

OBSERVATIONAL

Enrollment

400

Conditions

Hypochondroplasia

Eligibility

Sex: ALLMax age: 15 Years

Medical Language ↔ Plain English

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

Locations (43)

Outcomes

Primary Outcomes

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

Secondary Outcomes

Change in Quality of Life in Short Statured Youth (QoLISSY) physical domain

Time frame: Every 52 weeks through end of study, up to 15 years

Change in Quality of Life in Short Statured Youth (QoLISSY) total score

Time frame: Every 52 weeks through end of study, up to 15 years

Central Contacts

Trial Specialist

CONTACT

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

Data from ClinicalTrials.gov

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