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

Not Applicable

• Conditions: Hypochondroplasia
• Interventions: Drug: Vosoritide

• Registration Number: NCT07073014
• Lead Sponsor: BioMarin Pharmaceutical

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2025-06-20
• Study First Submitted: 2025-06-27
• Status Verified: 2025-07
• Study First Submitted QC: 2025-07-09
• Last Update Submitted: 2025-07-09
• Study First Posted: 2025-07-18
• Last Update Posted: 2025-07-18
• Primary Completion: 2040-12
• Study Completion: 2040-12-01

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 140

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Vosoritide	Vosoritide	Open-label active drug

Primary Outcome Measures

Name	Time	Method
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

Secondary Outcome Measures

Name	Time	Method
Evaluate long-term effect of vosoritide treatment on bone age versus chronological age	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 left-hand and wrist x-ray
Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers	Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males	Change from baseline in serum collagen X marker
Evaluate long-term effect of vosoritide treatment on bone quality	Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males	Change from baseline in bone mineral density (BMD) as measured by Dual X-Ray Absorptiometry
Evaluate the maintenance effect of vosoritide treatment on standing height	Baseline to week 52	change from baseline in height at Week 52
Evaluate long-term effect of vosoritide treatment on growth velocity	Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males	Absolute 6- or 12-month interval AGV
Evaluate the maintenance effect of vosoritide treatment on standing height Z-score	Baseline to week 52	Change from baseline in height Z-score at Week 52
Evaluate long-term effect of vosoritide treatment on body proportionality	Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males	Change from baseline in upper to lower body segment ratio
Evaluate long-term changes in Health-Related Quality of Life (HRQoL)	Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males	Change from baseline as measured by Quality of Life In Short-Statured Youth (QoLISSY) total score
To monitor the frequency of seizures	Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging	Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
To monitor the incidence of otitis media	Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

Trial Locations

Locations (23)

Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children); 🇺🇸 Wilmington, Delaware, United States

Children's National Medical Center; 🇺🇸 Washington, 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; 🇨🇦 Montréal, Quebec, Canada

Hospices Civils de Lyon - Hopital Femme Mere Enfant; 🇫🇷 Lyon, France

Hopital de la Timone; 🇫🇷 Marseille, France

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