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

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Trial Status Enrolling By Invitation

Trial Identifier

NCT07073014

Condition

Hypochondroplasia

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

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

Eligibility Criteria

Birth Sex

All

Age

Any

Healthy Volunteers

No

Drug/Treatment:

Vosoritide

Phase:

Phase 3

Study Type:

Interventional

Number of Participants:

140

Study Started:

2025-06-20

Study Updated:

2026-09-09

Trial Locations

  • Phoenix Children's Hospital - Thomas Campus

    Phoenix, Arizona, United States

  • Benioff Children's Hospital - Oakland

    Oakland, California, United States

  • 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

  • The Johns Hopkins University School of Medicine

    Baltimore, Maryland, United States

  • University of Minnesota Masonic Children's Hospital

    Minneapolis, Minnesota, United States

  • University of Missouri

    Columbia, Missouri, United States

  • Cook Children's Endocrinology

    Fort Worth, Texas, United States

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, United States

  • Murdoch Children's Research Institute of The Royal Children's Hospital

    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

  • Universitätsklinikum des Saarlandes

    Homburg, Saarland, Germany

  • 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

  • Hospital MiKS - Unidad de Cirugía Artroscópica

    Vitoria-Gasteiz, Spain

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, United Kingdom

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

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