NIPH Clinical Trials Search

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

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	Hypochondroplasia
Date of first enrollment	05/11/2025
Target sample size	8
Countries of recruitment	Australia,Japan,UK,Japan,France,Japan,Germany,Japan,Italy,Japan,United States,Japan
Study type	Interventional
Intervention(s)	Drug: Vosoritide Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily for 52 weeks. Other Names: Modified recombinant human C-type natriuretic peptide Drug: Placebo Subcutaneous injection of recommended dose of placebo once daily for 52 weeks.

Outcome(s)

Primary Outcome	- Incidence of TEAEs versus placebo over the course of the study - Incidence of SAEs versus placebo over the course of the study - Changes in standard clinical laboratory values (urinalysis, chemistry, hematology) versus placebo over the course of the study - Changes in vital signs versus placebo over the course of the study - Change from baseline at Week 52 versus placebo in height Z-score
Secondary Outcome	- Change from baseline at Week 52 versus placebo in height - Cumulative AGV at Week 52 versus placebo - 6-month interval AGV at Week 26 and Week 52, respectively (non-comparative)

Key inclusion & exclusion criteria

Age minimum	>=
Age maximum	< 36month old
Gender	Both
Include criteria	1 Participants must be 0 to < 36 months of age at randomization. 2 Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH). 3 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. 4 Participant's weight at the Day 1 visit (pre-treatment) must be >= 3 kg.
Exclude criteria	1.Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia). 2.Have an unstable medical condition likely to require surgical intervention during the study period. 3.Taking any of the prohibited medications. 4.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. 5.Require any investigational agent prior to completion of study period. 6.Have received another investigational product or investigational medical device within 30 days prior to the Screening visit. 7.Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time. 8.Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy. 9.Have known hypersensitivity to vosoritide or its excipients. 10.Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study. 11.Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.