Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

Part of paid clinical trials in Wilmington, Delaware.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT06455059
Phase
PHASE3
Status
Active Not Recruiting

Conditions

  • Hypochondroplasia

Eligibility Criteria

Sex
ALL
Age
3 Years - 17 Years
Healthy Volunteers
Not accepted

Interventions

  • Vosoritide — DRUG
    Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
  • Placebo — DRUG
    Subcutaneous injection of recommended dose of placebo

Study Details

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).

Key Dates

Start date
Jun 17, 2024
Status verified
Jan 2026
Primary completion
Aug 1, 2026
Completion
Aug 1, 2026

Study Design

Enrollment
80 participants (estimated)
Allocation
RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: vosoritide injection with vial and syringe
  • Placebo Comparator: Placebo injection with vial and syringe

Primary Outcome Measure

Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo [ Time Frame: At week 52 ]

Locations (4)

FacilityCityStateZIPSite coordinators
Nemours Alfred I. DuPont Hospital for ChildrenWilmingtonDelaware19803-
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 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

Related Studies