Active — Not RecruitingPhase 3

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

United States, Australia80 participantsStarted 2024-06-17

Plain-language summary

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).

Who can participate

Age range3 Years – 17 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Participants must be ≥ 3 to \< 18 years of age at enrollment
✓. A confirmed genetic diagnosis of HCH
✓. A height Z score of ≤ - 2.0 standard deviations (SDs) in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts
✓. Males and females are eligible to participate in this clinical study.
✓. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study.
✓. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.

Exclusion criteria

✕. Short stature condition other than HCH
✕. Have an unstable condition likely to require surgical intervention during the study.
✕. Evidence of decreased growth velocity and/or growth plate closure
✕. Taking any of the prohibited medications
✕. Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids
✕. Planned or expected to have limb-lengthening surgery during the study period.
✕. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period
✕. Require any investigational agent prior to completion of study period.

What they're measuring

Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo

Timeframe: At week 52

Trial details

NCT IDNCT06455059

SponsorBioMarin Pharmaceutical

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-08-01

View on ClinicalTrials.gov More Hypochondroplasia trials