Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
NCT ID: NCT07073014
Last Updated: 2025-07-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ENROLLING_BY_INVITATION
PHASE3
140 participants
INTERVENTIONAL
2025-06-20
2040-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Vosoritide
Open-label active drug
Vosoritide
Open-label administration of vosoritide using weight-band dosing
Interventions
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Vosoritide
Open-label administration of vosoritide using weight-band dosing
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Locations
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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
Countries
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Other Identifiers
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111-308
Identifier Type: -
Identifier Source: org_study_id
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