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Clinical Trials in the UK / NCT07073014
Enrolling by invitation Phase 3

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

NCT07073014 · tracked via the Priya Life Science UK tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 3
Started
2025-06-20
Last updated
2026-09-09

Condition(s) studied

Hypochondroplasia

Investigational drug(s) / intervention(s)

Vosoritide

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)

Trial sites (30)

FacilityCityRegionStatus
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 Lyon France
Hopital de la Timone Marseille France
Hopital Necker-Enfants Malade Paris France
CHU de Toulouse Toulouse France
Universitätsklinikum des Saarlandes Homburg Saarland
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
Hospital MiKS - Unidad de Cirugía Artroscópica Vitoria-Gasteiz Spain
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.

View NCT07073014 on ClinicalTrials.gov ↗ ← All trials in the UK