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A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months

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Trial Status Recruiting

Trial Identifier

NCT07126262

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 safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to < 36 months over a 52-week period.

Eligibility Criteria

Birth Sex

All

Age

0 Months to 36 Months

Healthy Volunteers

No

Drug/Treatment:

Vosoritide, Placebo

Phase:

Phase 2

Study Type:

Interventional

Number of Participants:

60

Study Started:

2025-07-30

Study Updated:

2025-08-17

Trial Locations

  • Phoenix Children's Hospital - Thomas Campus (Main)

    Phoenix, Arizona, United States

    Trial Status Not yet recruiting

  • Cedars-Sinai Medical Center

    Los Angeles, California, United States

    Trial Status Not yet recruiting

  • Benioff Children's Hospital - Oakland

    Oakland, California, United States

    Trial Status Not yet recruiting

  • Children's National Medical Center

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

    Trial Status Recruiting

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

    Chicago, Illinois, United States

    Trial Status Not yet recruiting

  • The Johns Hopkins University School of Medicine

    Baltimore, Maryland, United States

    Trial Status Not yet recruiting

  • University of Minneasota Masonic Children's Hospital

    Minneapolis, Minnesota, United States

    Trial Status Not yet recruiting

  • University of Missouri

    Columbia, Missouri, United States

    Trial Status Not yet recruiting

  • Children's Wisconsin - Fox Valley Hospital

    Neenah, Wisconsin, United States

    Trial Status Not yet recruiting

  • Royal Children's Hospital Melbourne

    Parkville, Victoria, Australia

    Trial Status Not yet recruiting

  • Hôpital Bicêtre

    Le Kremlin-Bicêtre, Paris, France

    Trial Status Not yet recruiting

  • Hospices Civils de Lyon - Hôpital Femme Mère Enfant

    Bron, Rhone-Alps, France

    Trial Status Not yet recruiting

  • Uniklinik Köln

    Cologne, North Rhine-Westphalia, Germany

    Trial Status Not yet recruiting

  • Universitätsklinikum des Saarlandes

    Homburg, Saarland, Germany

    Trial Status Not yet recruiting

  • Universitätskinderklinik Magdeburg

    Magdeburg, Saxony-Anhalt, Germany

    Trial Status Not yet recruiting

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, Rome, Italy

    Trial Status Not yet recruiting

  • IRCCS Istituto Giannina Gaslini

    Genova, Italy

    Trial Status Not yet recruiting

  • Myriad Trials

    London, England, United Kingdom

    Trial Status Not yet recruiting

  • Great Ormond Street Hospital

    London, England, United Kingdom

    Trial Status Not yet recruiting

Inclusion Criteria

  1. Participants must be 0 to < 36 months of age at randomization.
  2. Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
  3. Participants aged 0 to < 12 months must have a height Z-score of ≤ -1.0 SDS andparticipants aged ≥ 12 to < 36 months must have a height Z-score of ≤ -2.0 SDS in reference to the average stature of the same sex and age, as calculated using the Center for Disease Control and Prevention (CDC) growth charts.
  4. Participant’s weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg

Exclusion Criteria

  1. Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
  2. Have an unstable medical condition likely to require surgical intervention during the study period.
  3. Taking any of the prohibited medications.
  4. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the 6 months prior to Screening, or long-term treatment (> 3 months) at any time.
  5. Require any investigational agent prior to completion of study period.
  6. Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.
  7. Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time.
  8. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
  9. Have known hypersensitivity to vosoritide or its excipients.
  10. Have a condition or circumstance that, in the view of the investigator, places the participant at high risk for poor treatment compliance or for not completing the study.
  11. Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.

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