DRUG
Vosoritide
Open-label administration of vosoritide using weight-band dosing
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
Status
Enrolling by invitation
Phase
Phase 3
Enrollment
140
Locations
23
Results
Not posted
Publications
0
Study summary
What the protocol is testing.
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
Interventions
Treatment arms and agents.
Timeline
From registration to results.
First posted
Jul 18, 2025
Study start
Jun 20, 2025
Primary completion
Dec 2040
Study completion
Dec 2040
Results posted
Not reported
Registry updated
Jul 18, 2025
Outcomes
What the study measures.
Primary outcomes
Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)
Time frame · 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 outcomes
Evaluate the maintenance effect of vosoritide treatment on standing height
Time frame · Baseline to week 52
change from baseline in height at Week 52
Evaluate the maintenance effect of vosoritide treatment on standing height Z-score
Time frame · Baseline to week 52
Change from baseline in height Z-score at Week 52
Evaluate long-term effect of vosoritide treatment on growth velocity
Time frame · 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 long-term effect of vosoritide treatment on body proportionality
Time frame · 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 effect of vosoritide treatment on bone age versus chronological age
Time frame · 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
Time frame · 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 changes in Health-Related Quality of Life (HRQoL)
Time frame · 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
Evaluate long-term effect of vosoritide treatment on bone quality
Time frame · 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 long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging
Time frame · 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
Time frame · Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Eligibility
Who can take part.
- Minimum age
- Not reported
- Maximum age
- Not reported
- Sex
- ALL
- 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
Study locations
23 registered sites.
Australia · Canada · France · Germany · Italy · Japan · Spain · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
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
Publications
Results and literature.
No PMID-linked publications were present in this registry snapshot.
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NCT IDStudyPhase / statusEnrollmentUpdated
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Recruiting
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A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
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