A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months (NCT07126262) | Clinical Trial Compass
RecruitingPhase 2
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
United States60 participantsStarted 2025-07-30
Plain-language summary
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.
Who can participate
Age range0 Months – 36 Months
SexALL
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Inclusion criteria
✓. Participants must be 0 to \< 36 months of age at randomization.
✓. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
✓. Participants aged 0 to \< 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to \< 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts.
✓. Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.
Exclusion criteria
✕. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
✕. Have an unstable medical condition likely to require surgical intervention during the study period.
✕. Taking any of the prohibited medications.
✕. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (\> 3 months) at any time.
✕. Require any investigational agent prior to completion of study period.
✕. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.
✕. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time.
✕. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
What they're measuring
1
Incidence of treatment-emergent adverse events
Timeframe: From baseline to end of treatment at 52 weeks
2
Incidence of serious adverse events versus placebo over the course of the study
Timeframe: From baseline to end of treatment at 52 weeks
3
Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)
Timeframe: At week 26, at week 52
4
Changes from baseline in heart rate
Timeframe: At week 13, at week 26, at week 39, at week 52
5
Change from baseline in height Z-score
Timeframe: At week 52
6
Changes from baseline in respiratory rate
Timeframe: At week 13, at week 26, at week 39, at week 52
7
Changes from baseline in temperature
Timeframe: At week 13, at week 26, at week 39, at week 52