Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

NCT06455059 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: 111-303
• Study Type: interventional
• Target: 80
• Locations: 9 countries
• Sites: 23

Brief 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).

Conditions

Hypochondroplasia

Keywords

Hypochondroplasia; Dwarfism; Bone Diseases; Bone Disease, Developmental; HCH; Natriuretic Peptide, C-Type; Musculoskeletal Diseases; Natriuretic Agents; Physiological Effects of Drugs; Skeletal Dysplasias; Genetic Diseases, Inborn; Osteochondrodysplasias; Vosoritide; Voxzogo

Outcome Measures

Primary Outcomes (1)

• Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo

  At week 52

Secondary Outcomes (2)

• Change from baseline in standing height at Week 52 versus placebo

  At week 52

• Change from baseline in height Z-score at Week 52 versus placebo

  At week 52

Study Arms (2)

vosoritide injection with vial and syringe

EXPERIMENTAL

Drug: Vosoritide

Placebo injection with vial and syringe

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Vosoritide DRUG

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

Also known as: Modified recombinant human C-type natriuretic peptide

vosoritide injection with vial and syringe

Placebo DRUG

Subcutaneous injection of recommended dose of placebo

Placebo injection with vial and syringe

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participants must be ≥ 3 to \< 18 years of age at enrollment
• A confirmed genetic diagnosis of HCH
• 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
• Males and females are eligible to participate in this clinical study.
• 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.
• If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.

You may not qualify if:

• Short stature condition other than HCH
• Have an unstable condition likely to require surgical intervention during the study.
• Evidence of decreased growth velocity and/or growth plate closure
• Taking any of the prohibited medications
• Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids
• Planned or expected to have limb-lengthening surgery during the study period.
• Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period
• Require any investigational agent prior to completion of study period.
• Received vosoritide or another investigational product or investigational medical device in the past
• Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide
• Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
• Have known hypersensitivity to vosoritide or its excipients.
• Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (23)

Nemours Alfred I. DuPont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

Location

University of Alberta Stollery Children's Hospital

Edmonton, Alberta, Canada

Location

SickKids - The Hospital for Sick Children

Toronto, Ontario, Canada

Location

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, Canada

Location

Hospices Civils de Lyon - Hopital Femme Mere Enfant

Lyon, France

Location

Hopital de la Timone

Marseille, France

Location

Hopital Necker-Enfants Malade

Paris, France

Location

CHU de Toulouse

Toulouse, France

Location

Uniklinik Koln

Cologne, Germany

Location

Univeristatskinderklinik Magdeburg

Magdeburg, Germany

Location

Instituto Giannina Gaslini

Genoa, Italy

Location

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, Italy

Location

Ospedale Pediatrico Bambino Gesu

Rome, Italy

Location

Osaka University Hosptial

Osaka, Japan

Location

Tokushima University Hospital

Tokushima, Japan

Location

Institute of Science Tokyo Hospital

Tokyo, Japan

Location

Tottori University Hospital

Tottori, Japan

Location

Vithas Hospital San Jose

Vitoria-Gasteiz, Spain

Location

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

Location

MeSH Terms

Conditions

Hypochondroplasia; Dwarfism; Bone Diseases; Bone Diseases, Developmental; Musculoskeletal Diseases; Genetic Diseases, Inborn; Osteochondrodysplasias

Interventions

vosoritide

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Endocrine System Diseases

Study Officials

• Medical Director, MD

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 7, 2024
• First Posted: June 12, 2024
• Study Start: June 17, 2024
• Primary Completion (Estimated): August 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

CA (3); AU (1); DE (2); IT (3); US (4); ES (1); JP (4); GB (1); FR (4)