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

NCT07126262 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 111-212
• Study Type: interventional
• Target: 60
• Locations: 7 countries
• Sites: 26

Brief 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.

Conditions

Hypochondroplasia

Outcome Measures

Primary Outcomes (8)

• Incidence of treatment-emergent adverse events

  From baseline to end of treatment at 52 weeks

• Incidence of serious adverse events versus placebo over the course of the study

  From baseline to end of treatment at 52 weeks

• Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)

  At week 26, at week 52

• Changes from baseline in heart rate

  Units of measure: bpm

  At week 13, at week 26, at week 39, at week 52

• Change from baseline in height Z-score

  At week 52

• Changes from baseline in respiratory rate

  Units of measure: breaths/min

  At week 13, at week 26, at week 39, at week 52

• Changes from baseline in temperature

  Units of measure: celsius

  At week 13, at week 26, at week 39, at week 52

• Changes from baseline in blood pressure

  Units of measure: mmHg

  At week 13, at week 26, at week 39, at week 52

Secondary Outcomes (19)

• Change in height

  At week 52

• Cumulative annualized growth velocity (AGV)

  At week 52

• 6-month interval AGV

  At week 26, at week 52

• Change from baseline in upper to lower body segment ratio

  At week 52

• Change from baseline in arm span

  At week 52

Study Arms (2)

Vosoritide injection with vial and syringe

EXPERIMENTAL

Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.

Drug: Vosoritide

Placebo injection with vial and syringe

PLACEBO COMPARATOR

Subcutaneous injection of recommended dose of placebo

Drug: Placebo

Interventions

Vosoritide DRUG

The vosoritide dose administered will be based on the participant's weight and will follow the weight-band dosing regimen approved for ACH

Vosoritide injection with vial and syringe

Placebo DRUG

Subcutaneous injection of recommended dose of placebo

Placebo injection with vial and syringe

Eligibility Criteria

• Age: 0 Months - 36 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participants must be 0 to \< 36 months of age at randomization.
• Participants must have a confirmed genetic diagnosis of HCH (obtained via whole genome sequencing; presence of a FGFR3 pathogenic variant associated with HCH).
• 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.
• Participant's weight at the Day 1 visit (pre-treatment) must be ≥ 3 kg.

You may not qualify if:

• Short stature condition other than HCH (eg, ACH, trisomy 21, pseudoachondroplasia).
• Have an unstable medical condition likely to require surgical intervention during the study period.
• Taking any of the prohibited medications.
• 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.
• Require any investigational agent prior to completion of study period.
• Have received another investigational product or investigational medical device within 30 days prior to the Screening visit.
• Have used any other investigational product or investigational medical device for the treatment of HCH or short stature at any time.
• Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
• Have known hypersensitivity to vosoritide or its excipients.
• 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.
• Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason.

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead
• ICON Clinical Research: collaborator

Study Sites (26)

Phoenix Children's Hospital - Thomas Campus (Main)

Phoenix, Arizona, 85016, United States

RECRUITING

Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States

NOT YET RECRUITING

Benioff Children's Hospital - Oakland

Oakland, California, 94609, United States

RECRUITING

Children's National Medical Center

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

RECRUITING

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

Chicago, Illinois, 60611, United States

RECRUITING

The Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States

RECRUITING

University of Minneasota Masonic Children's Hospital

Minneapolis, Minnesota, 55454, United States

RECRUITING

University of Missouri

Columbia, Missouri, 65211, United States

RECRUITING

Cook Children's Endocrinology

Fort Worth, Texas, 76104, United States

NOT YET RECRUITING

Children's Wisconsin - Fox Valley Hospital

Neenah, Wisconsin, 54956, United States

NOT YET RECRUITING

Children's Health Queensland Hospital and Health Service

South Brisbane, Queensland, 4101, Australia

NOT YET RECRUITING

Royal Children's Hospital Melbourne

Parkville, Victoria, 3052, Australia

RECRUITING

Hôpital Bicêtre

Le Kremlin-Bicêtre, Paris, 94270, France

RECRUITING

Hospices Civils de Lyon - Hôpital Femme Mère Enfant

Bron, Rhone-Alps, 69500, France

NOT YET RECRUITING

Uniklinik Köln

Cologne, North Rhine-Westphalia, 50937, Germany

RECRUITING

Universitätsklinikum des Saarlandes

Homburg, Saarland, 66421, Germany

RECRUITING

Universitätskinderklinik Magdeburg

Magdeburg, Saxony-Anhalt, 39120, Germany

NOT YET RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Roma, Rome, 00168, Italy

NOT YET RECRUITING

IRCCS Istituto Giannina Gaslini

Genova, 16147, Italy

RECRUITING

Kumamoto University Hospital

Kumamoto, 860-8556, Japan

RECRUITING

Osaka Women's and Children's Hospital

Osaka, 94-1101, Japan

RECRUITING

Institute of Science Tokyo Hospital

Tokyo, 113-8519, Japan

RECRUITING

Nihon University Itabashi Hospital

Tokyo, 173-8610, Japan

RECRUITING

Tottori University Hospital

Tottori, 683-0854, Japan

RECRUITING

Myriad Trials

London, England, W1W 5DT, United Kingdom

NOT YET RECRUITING

Great Ormond Street Hospital

London, England, WC1N3BH, United Kingdom

NOT YET RECRUITING

MeSH Terms

Conditions

Hypochondroplasia

Interventions

vosoritide

Central Study Contacts

Trial Specialist

CONTACT

1-800-983-4587; medinfo@bmrn.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 29, 2025
• First Posted: August 17, 2025
• Study Start: July 30, 2025
• Primary Completion (Estimated): June 30, 2028
• Study Completion (Estimated): June 30, 2028
• Last Updated: March 31, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (2); FR (2); JP (5); DE (3); IT (2); AU (2); US (10)