Vosoritide: The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH
Placebo: Subcutaneous injection of recommended dose of placebo
Study 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.
Eligibility
Sex
ALL
Min age
0 Months
Max age
36 Months
Healthy volunteers
No
Key Inclusion 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.
Key Exclusion 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.
Primary outcome measure(s)
Incidence of treatment-emergent adverse events — From baseline to end of treatment at 52 weeks
Incidence of serious adverse events versus placebo over the course of the study — From baseline to end of treatment at 52 weeks
Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry) — At week 26, at week 52
Changes from baseline in heart rate — At week 13, at week 26, at week 39, at week 52 Units of measure: bpm
Change from baseline in height Z-score — At week 52
Changes from baseline in respiratory rate — At week 13, at week 26, at week 39, at week 52 Units of measure: breaths/min
Changes from baseline in temperature — At week 13, at week 26, at week 39, at week 52 Units of measure: celsius
Changes from baseline in blood pressure — At week 13, at week 26, at week 39, at week 52 Units of measure: mmHg
Trial sites (26)
Facility
City
Region
Status
Phoenix Children's Hospital - Thomas Campus (Main)
Phoenix
Arizona
Recruiting
Cedars-Sinai Medical Center
Los Angeles
California
Not Yet Recruiting
Benioff Children's Hospital - Oakland
Oakland
California
Recruiting
Children's National Medical Center
Washington D.C.
District of Columbia
Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago
Illinois
Recruiting
The Johns Hopkins University School of Medicine
Baltimore
Maryland
Recruiting
University of Minneasota Masonic Children's Hospital
Minneapolis
Minnesota
Recruiting
University of Missouri
Columbia
Missouri
Recruiting
Cook Children's Endocrinology
Fort Worth
Texas
Not Yet Recruiting
Children's Wisconsin - Fox Valley Hospital
Neenah
Wisconsin
Not Yet Recruiting
Children's Health Queensland Hospital and Health Service
South Brisbane
Queensland
Not Yet Recruiting
Royal Children's Hospital Melbourne
Parkville
Victoria
Recruiting
Hôpital Bicêtre
Le Kremlin-Bicêtre
Paris
Recruiting
Hospices Civils de Lyon - Hôpital Femme Mère Enfant
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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