RECRUITING

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months

Quick Facts

Study Start:2025-07-30

Study Completion:2028-06-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT07126262

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:0 Months to 36 Months

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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.	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.

Inclusion Criteria

Exclusion 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.

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.

Contacts and Locations

Study Contact

Trial Specialist

CONTACT

1-800-983-4587

medinfo@bmrn.com

Study Locations (Sites)

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, 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

Children's Wisconsin - Fox Valley Hospital

Neenah, Wisconsin, 54956

United States

Collaborators and Investigators

Sponsor: BioMarin Pharmaceutical

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-07-30

Study Completion Date2028-06-30

Study Record Updates

Study Start Date2025-07-30

Study Completion Date2028-06-30

Terms related to this study

Additional Relevant MeSH Terms

Hypochondroplasia