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NCT07126262PHASE2Recruiting

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

BioMarin Pharmaceutical

Start Date

7/30/2025

Completion Date

6/30/2028

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.

Detailed Description

Study 111-212 is a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to assess the safety and efficacy of vosoritide versus placebo in infants and young children with HCH. Eligible participants with documented HCH confirmed by genetic testing will be randomized in a 1:1 ratio to receive vosoritide or placebo. Participants will receive study treatment daily for 52 weeks by subcutaneous (SC) injection, followed by a 2-week safety follow-up visit. Vosoritide dosing will follow a weight-band regimen.

Eligibility Criteria

Age Range: No minimum to No maximum

Key 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. Key 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.

Interventions

DRUG

Vosoritide

DRUG

Placebo

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Conditions

Hypochondroplasia

Locations

Phoenix Children's Hospital - Thomas Campus (Main)

Phoenix, Arizona 85016

United States

Cedars-Sinai Medical Center

Los Angeles, California 90048

United States

Benioff Children's Hospital - Oakland

Oakland, California 94609

United States

Children's National Medical Center

Washington D.C., District of Columbia 20010

United States

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

Chicago, Illinois 60611

United States

The Johns Hopkins University School of Medicine

Baltimore, Maryland 21205

United States

University of Minneasota Masonic Children's Hospital

Minneapolis, Minnesota 55454

United States

University of Missouri

Columbia, Missouri 65211

United States

Cook Children's Endocrinology

Fort Worth, Texas 76104

United States

Children's Wisconsin - Fox Valley Hospital

Neenah, Wisconsin 54956

United States

Children's Health Queensland Hospital and Health Service

South Brisbane, Queensland 4101

Australia

Royal Children's Hospital Melbourne

Parkville, Victoria 3052

Australia

Hôpital Bicêtre

Le Kremlin-Bicêtre, Paris 94270

France

Hospices Civils de Lyon - Hôpital Femme Mère Enfant

Bron, Rhone-Alps 69500

France

Uniklinik Köln

Cologne, North Rhine-Westphalia 50937

Germany

Universitätsklinikum des Saarlandes

Homburg, Saarland 66421

Germany

Universitätskinderklinik Magdeburg

Magdeburg, Saxony-Anhalt 39120

Germany

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Rome 00168

Italy

IRCCS Istituto Giannina Gaslini

Genova, 16147

Italy

Kumamoto University Hospital

Kumamoto, 860-8556

Japan

Osaka Women's and Children's Hospital

Osaka, 94-1101

Japan

Institute of Science Tokyo Hospital

Tokyo, 113-8519

Japan

Nihon University Itabashi Hospital

Tokyo, 173-8610

Japan

Tottori University Hospital

Tottori, 683-0854

Japan

Myriad Trials

London, England W1W 5DT

United Kingdom

Great Ormond Street Hospital

London, England WC1N3BH

United Kingdom