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Clinical Trials in the USA / NCT06455059
Active, not recruiting Phase 3

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

NCT06455059 · tracked via the Priya Life Science USA tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 3
Started
2024-06-17
Last updated
2026-01-20

Condition(s) studied

Hypochondroplasia

Investigational drug(s) / intervention(s)

VosoritidePlacebo

Vosoritide: Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.

Placebo: Subcutaneous injection of recommended dose of placebo

Study summary

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).

Eligibility

Sex
ALL
Min age
3 Years
Max age
17 Years
Healthy volunteers
No
Inclusion Criteria: 1. Participants must be ≥ 3 to \< 18 years of age at enrollment 2. A confirmed genetic diagnosis of HCH 3. A height Z score of ≤ - 2.0 standard deviations (SDs) in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts 4. Males and females are eligible to participate in this clinical study. 5. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study. 6. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study. Exclusion Criteria: 1. Short stature condition other than HCH 2. Have an unstable condition likely to require surgical intervention during the study. 3. Evidence of decreased growth velocity and/or growth plate closure 4. Taking any of the prohibited medications 5. Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids 6. Planned or expected to have limb-lengthening surgery during the study period. 7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period 8. Require any investigational agent prior to completion of study period. 9. Received vosoritide or another investigational product or investigational medical device in the past 10. Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide 11. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy. 12. Have known hypersensitivity to vosoritide or its excipients. 13. 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 (23)

FacilityCityRegionStatus
Nemours 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
Medical College of Wisconsin Milwaukee Wisconsin
Murdoch Children's Research Institute 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
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

More BioMarin Pharmaceutical trials in the USA

Other trials for the same condition

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 NCT06455059 on ClinicalTrials.gov ↗ ← All trials in the USA