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

Part of paid clinical trials in Wilmington, Delaware.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT07073014
Phase
PHASE3
Status
Enrolling By Invitation

Conditions

  • Hypochondroplasia

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Vosoritide — DRUG
    Open-label administration of vosoritide using weight-band dosing

Study Details

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

Key Dates

Start date
Jun 20, 2025
Status verified
Jul 2025
Primary completion
Dec 31, 2040
Completion
Dec 31, 2040

Study Design

Enrollment
140 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Vosoritide
    Open-label active drug

Primary Outcome Measure

Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH) [ Time Frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males ]

Locations (4)

FacilityCityStateZIPSite coordinators
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)WilmingtonDelaware19803-
Children's National Medical CenterWashington D.C.District of Columbia20010-
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-
Medical College of WisconsinMilwaukeeWisconsin53226-

Find similar trials in Wilmington, DE

By research site
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)· Wilmington, DEChildren's National Medical Center· Washington D.C., DCAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILMedical College of Wisconsin· Milwaukee, WI

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