DRUG
Vosoritide
Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
Status
Active, not recruiting
Phase
Phase 3
Enrollment
80
Locations
23
Results
Not posted
Publications
0
Study 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).
This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH. Eligible participants with documented HCH confirmed by genetic testing will roll over from Study 111-902 and enter the 111-303 study. Participants will be randomly assigned to one of two treatment groups: Placebo or Vosoritide. The route of administration is subcutaneous injection, and the frequency of administration is daily.
Interventions
DRUG
Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
DRUG
Subcutaneous injection of recommended dose of placebo
Timeline
First posted
Jun 12, 2024
Study start
Jun 17, 2024
Primary completion
Aug 1, 2026
Study completion
Aug 1, 2026
Results posted
Not reported
Registry updated
Jan 20, 2026
Outcomes
Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo
Time frame · At week 52
Change from baseline in standing height at Week 52 versus placebo
Time frame · At week 52
Change from baseline in height Z-score at Week 52 versus placebo
Time frame · At week 52
Eligibility
Inclusion Criteria: 1. Participants must be ≥ 3 to \< 18 years of age at enrollment 2. A confirmed genetic diagnosis of HCH 3. 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 4. Males and females are eligible to participate in this clinical study. 5. 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. 6. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study. Exclusion Criteria: 1. Short stature condition other than HCH 2. Have an unstable condition likely to require surgical intervention during the study. 3. Evidence of decreased growth velocity and/or growth plate closure 4. Taking any of the prohibited medications 5. Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids 6. Planned or expected to have limb-lengthening surgery during the study period. 7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period 8. Require any investigational agent prior to completion of study period. 9. Received vosoritide or another investigational product or investigational medical device in the past 10. Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide 11. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy. 12. Have known hypersensitivity to vosoritide or its excipients. 13. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
Study locations
Australia · Canada · France · Germany · Italy · Japan · Spain · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
Nemours Alfred I. DuPont Hospital for Children
Wilmington, Delaware, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
University of Alberta Stollery Children's Hospital
Edmonton, Alberta, Canada
SickKids - The Hospital for Sick Children
Toronto, Ontario, Canada
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
Hospices Civils de Lyon - Hopital Femme Mere Enfant
Lyon, France
Hopital de la Timone
Marseille, France
Hopital Necker-Enfants Malade
Paris, France
CHU de Toulouse
Toulouse, France
Uniklinik Koln
Cologne, Germany
Univeristatskinderklinik Magdeburg
Magdeburg, Germany
Instituto Giannina Gaslini
Genoa, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, Italy
Ospedale Pediatrico Bambino Gesu
Rome, Italy
Osaka University Hosptial
Osaka, Japan
Tokushima University Hospital
Tokushima, Japan
Institute of Science Tokyo Hospital
Tokyo, Japan
Tottori University Hospital
Tottori, Japan
Vithas Hospital San Jose
Vitoria-Gasteiz, Spain
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom
Publications
No PMID-linked publications were present in this registry snapshot.
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