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

NCT07073014 | Enrolling By Invitation Phase 3 DMC FDA Drug

• 1 other identifier: 111-308
• Study Type: interventional
• Target: 140
• Locations: 9 countries
• Sites: 23

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

Conditions

Hypochondroplasia

Outcome Measures

Primary Outcomes (1)

• Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)

  As measured by change from baseline in height Z-score

  Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

Secondary Outcomes (11)

• Evaluate the maintenance effect of vosoritide treatment on standing height

  Baseline to week 52

• Evaluate the maintenance effect of vosoritide treatment on standing height Z-score

  Baseline to 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

• 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

• 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

Study Arms (1)

Vosoritide

EXPERIMENTAL

Open-label active drug

Drug: Vosoritide

Interventions

Vosoritide DRUG

Open-label administration of vosoritide using weight-band dosing

Vosoritide

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (23)

Nemours Children's Hospital, Delaware (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

Interventions

vosoritide

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 27, 2025
• First Posted: July 18, 2025
• Study Start: June 20, 2025
• Primary Completion (Estimated): December 1, 2040
• Study Completion (Estimated): December 1, 2040
• Last Updated: July 18, 2025

Record last verified: 2025-07

Locations

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