Clinical Research Directory

A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.

Prospective Longitudinal Monocentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia

The study aims to identify which Syde®-derived digital outcomes are reliable in FGFR3-related Skeletal Dysplasia. This requires to set-up a natural history study to measure limb movements in patients with ACH or HCH.

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).

An Interventional Study of Infigratinib in Children With Hypochondroplasia

ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety, of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib, at one of two doses, of who have completed at least 26 weeks of participation in QED-sponsored ACCEL (QBGJ398-004).

Prospective Clinical Assessment Study in Children With Hypochondroplasia

This is a long-term, multicenter, non-interventional study of children ages 2.5 to \<17 years with hypochondroplasia (HCH).

Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

A Multicenter Multinational Observational Study of Children With Hypochondroplasia

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

Registry for Patients With Achondroplasia / Hypochondroplasia (OMPR-Ach/Hy)

This registry is a observational, single-center study designed to collect clinical data on patients with achondroplasia and hypochondroplasia.