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

NCT07073014

Enrolling By InvitationPhase 3

Sponsor: BioMarin Pharmaceutical

Conditions: Hypochondroplasia

Interventions: Vosoritide

Countries: United States, Australia, Canada, France, Germany, Italy, Japan, Spain

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

Eligibility overview

Sex: ALL

Healthy volunteers: No

Study type: INTERVENTIONAL

Eligibility criteria
Inclusion Criteria:

* 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

Exclusion Criteria:

* 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
Locations (30)
  • Phoenix, Arizona, United States
  • Oakland, California, United States
  • Wilmington, Delaware, United States
  • Washington D.C., District of Columbia, United States
  • Chicago, Illinois, United States
  • Baltimore, Maryland, United States
  • Minneapolis, Minnesota, United States
  • Columbia, Missouri, United States
  • Fort Worth, Texas, United States
  • Milwaukee, Wisconsin, United States
  • Parkville, Victoria, Australia
  • Edmonton, Alberta, Canada
  • Toronto, Ontario, Canada
  • Montreal, Quebec, Canada
  • Lyon, France
  • Marseille, France
  • Paris, France
  • Toulouse, France
  • Homburg, Saarland, Germany
  • Cologne, Germany
  • Magdeburg, Germany
  • Genoa, Italy
  • Rome, Italy
  • Rome, Italy
  • Osaka, Japan
  • Tokushima, Japan
  • Tokyo, Japan
  • Tottori, Japan
  • Vitoria-Gasteiz, Spain
  • London, United Kingdom