Vosoritide: Open-label administration of vosoritide using weight-band dosing
Study summary
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
Eligibility
Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
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
Primary outcome measure(s)
Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH) — Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males As measured by change from baseline in height Z-score
Trial sites (30)
Facility
City
Region
Status
Phoenix Children's Hospital - Thomas Campus
Phoenix
Arizona
Benioff Children's Hospital - Oakland
Oakland
California
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)
Wilmington
Delaware
Children's National Medical Center
Washington D.C.
District of Columbia
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago
Illinois
The Johns Hopkins University School of Medicine
Baltimore
Maryland
University of Minnesota Masonic Children's Hospital
Minneapolis
Minnesota
University of Missouri
Columbia
Missouri
Cook Children's Endocrinology
Fort Worth
Texas
Medical College of Wisconsin
Milwaukee
Wisconsin
Murdoch Children's Research Institute of The Royal Children's Hospital
Parkville
Victoria
University of Alberta Stollery Children's Hospital
Edmonton
Alberta
SickKids - The Hospital for Sick Children
Toronto
Ontario
Centre Hospitalier Universitaire Sainte-Justine
Montreal
Quebec
Hospices Civils de Lyon - Hopital Femme Mere Enfant
Great Ormond Street Hospital for Children NHS Foundation Trust
London
United Kingdom
Official registry record
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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