Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

Enrolling by invitation · Phase 3

Conditions studied: Hypochondroplasia

In brief

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

Key facts

Study ID: NCT07073014
Run by: BioMarin Pharmaceutical
People needed: 140
Starts: 2025-06-20
Expected to finish: 2040-12-01
Last updated by the study team: 2025-07-18

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays
Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure
Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline 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
Participants are willing and able to perform all study procedures as physically possible
Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training

You may not qualify if…

Permanently discontinued study treatment in the studies 111-303 or 111-212
Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care
Taking or planning to take any prohibited medications
Planned or expected to have limb-lengthening surgery during the study period
Planned or expected bone-related surgery during the study period
Require any investigational agent prior to completion of study period
Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy
Have known hypersensitivity to vosoritide or its excipients
Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study
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 Children's Hospital, Delaware (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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