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

Part of paid clinical trials in Phoenix, Arizona.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT07126262
Phase
PHASE2
Status
Recruiting
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Conditions

  • Hypochondroplasia

Eligibility Criteria

Sex
ALL
Age
0 Months - 36 Months
Healthy Volunteers
Not accepted

Interventions

  • Vosoritide — DRUG
    The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH
  • Placebo — DRUG
    Subcutaneous injection of recommended dose of placebo

Study Details

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.

Key Dates

Start date
Jul 30, 2025
Status verified
Mar 2026
Primary completion
Jun 30, 2028
Completion
Jun 30, 2028

Study Design

Enrollment
60 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Vosoritide injection with vial and syringe
    Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
  • Placebo Comparator: Placebo injection with vial and syringe
    Subcutaneous injection of recommended dose of placebo

Primary Outcome Measure

Incidence of treatment-emergent adverse events [ Time Frame: From baseline to end of treatment at 52 weeks ]

Central Contacts

Locations (10)

FacilityCityStateZIPSite coordinators
Phoenix Children's Hospital - Thomas Campus (Main)PhoenixArizona85016-
Cedars-Sinai Medical CenterLos AngelesCalifornia90048-
Benioff Children's Hospital - OaklandOaklandCalifornia94609-
Children's National Medical CenterWashington D.C.District of Columbia20010-
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-
The Johns Hopkins University School of MedicineBaltimoreMaryland21205-
University of Minneasota Masonic Children's HospitalMinneapolisMinnesota55454-
University of MissouriColumbiaMissouri65211-
Cook Children's EndocrinologyFort WorthTexas76104-
Children's Wisconsin - Fox Valley HospitalNeenahWisconsin54956-

Find similar trials in Phoenix, AZ

By research site
Phoenix Children's Hospital - Thomas Campus (Main)· Phoenix, AZCedars-Sinai Medical Center· Los Angeles, CABenioff Children's Hospital - Oakland· Oakland, CAChildren's National Medical Center· Washington D.C., DCAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, ILThe Johns Hopkins University School of Medicine· Baltimore, MD

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