Health TrialsbyHealthPanel

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

ConditionHypochondroplasia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeUp to 15
SponsorBioMarin Pharmaceutical

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Participants must be ≤ 15 years old at the time of signing the informed consent

Participants must have genetic confirmation of Hypochondroplasia diagnosis

Disqualifiers

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

Trial population

The study population will include approximately 400 pediatric individuals with hypochondroplasia.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

400 Participants
are grouped into 1 trial group
Group A: Children with Hypochondroplasia

Trial outcomes

Primary outcomes

1

Change in annualized growth velocity (AGV)

Time frame
Every 6 months through end of study, up to 15 years
2

Change in Height Z-score

Time frame
Every 6 months through end of study, up to 15 years
3

Change in Height

Time frame
Every 6 months through end of study, up to 15 years
4

Change in BMI

Time frame
Every 6 months through end of study, up to 15 years

Secondary outcomes

1

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
2

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
3

Change in patient global impression of severity (PGI-S)

Time frame
Every 52 weeks through end of study, up to 15 years
4

Change in caregiver global impression of severity (CaGI-S)

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

BioMarin Pharmaceutical

Lead sponsor

Source ClinicalTrials.gov
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