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New drug aims to help short-statured children grow taller

NCT ID NCT06455059

Ongoing Disease control Sponsor: BioMarin Pharmaceutical Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called vosoritide to see if it can safely help children with hypochondroplasia grow faster. About 80 children aged 3 to 17 with a confirmed genetic diagnosis and very short stature will receive either the drug or a placebo for one year. The main goal is to measure how much their growth rate improves compared to those on placebo.

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Conditions

The condition(s) this trial relates to.

Bone Diseases, Developmental bone disorder Dwarfism hereditary disease hypochondroplasia musculoskeletal system disorder osteochondrodysplasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HYPOCHONDROPLASIA

Contacts and locations

Locations

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

    Chicago, Illinois, 60611, United States

  • CHU de Toulouse

    Toulouse, France

  • Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Quebec, Canada

  • Children's National Medical Center

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

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Rome, Italy

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, United Kingdom

  • Hopital Necker-Enfants Malade

    Paris, France

  • Hopital de la Timone

    Marseille, France

  • Hospices Civils de Lyon - Hopital Femme Mere Enfant

    Lyon, France

  • Institute of Science Tokyo Hospital

    Tokyo, Japan

  • Instituto Giannina Gaslini

    Genoa, Italy

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Murdoch Children's Research Institute

    Parkville, Victoria, 3052, Australia

  • Nemours Alfred I. DuPont Hospital for Children

    Wilmington, Delaware, 19803, United States

  • Osaka University Hosptial

    Osaka, Japan

  • Ospedale Pediatrico Bambino Gesu

    Rome, Italy

  • SickKids - The Hospital for Sick Children

    Toronto, Ontario, Canada

  • Tokushima University Hospital

    Tokushima, Japan

  • Tottori University Hospital

    Tottori, Japan

  • Uniklinik Koln

    Cologne, Germany

  • Univeristatskinderklinik Magdeburg

    Magdeburg, Germany

  • University of Alberta Stollery Children's Hospital

    Edmonton, Alberta, Canada

  • Vithas Hospital San Jose

    Vitoria-Gasteiz, Spain

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