Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Hypochondroplasia

In brief

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).

Key facts

Study ID

NCT06455059

Run by

BioMarin Pharmaceutical

People needed

80

Starts

2024-06-17

Expected to finish

2026-08-01

Last updated by the study team

2026-01-20

Who can join

Age: 3 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.
• Received vosoritide or another investigational product or investigational medical device in the past
• Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide
• Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
• Have known hypersensitivity to vosoritide or its excipients.
• Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.

Where it is running

• 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

Full record on ClinicalTrials.gov

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