Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

Study Purpose

The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	3 Years - 17 Years
Gender	All

More Inclusion & Exclusion Criteria

Inclusion Criteria:

1. Participants must be ≥ 3 to < 18 years of age at enrollment. 2. A confirmed genetic diagnosis of HCH. 3. A height Z score of ≤

- 2.0 standard deviations (SDs) in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts.

4. Males and females are eligible to participate in this clinical study. 5. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study. 6. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.

Exclusion Criteria:

1. Short stature condition other than HCH. 2. Have an unstable condition likely to require surgical intervention during the study. 3. Evidence of decreased growth velocity and/or growth plate closure. 4. Taking any of the prohibited medications. 5. Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids. 6. Planned or expected to have limb-lengthening surgery during the study period. 7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period. 8. Require any investigational agent prior to completion of study period. 9. Received vosoritide or another investigational product or investigational medical device in the past. 10. Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide. 11. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy. 12. Have known hypersensitivity to vosoritide or its excipients. 13. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT06455059
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 3
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	BioMarin Pharmaceutical
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	Medical Director, MD
Principal Investigator Affiliation	BioMarin Pharmaceutical
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Enrolling by invitation
Countries	Australia, United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Hypochondroplasia

Additional Details

This is a Phase 3 randomized, stratified, placebo-controlled, double-blind multicenter study to evaluate the effect of 52 weeks of daily vosoritide administration on annualized growth velocity (AGV) in participants with HCH. Eligible participants with documented HCH confirmed by genetic testing will roll over from Study 111-902 and enter the 111-303 study. Participants will be randomly assigned to one of two treatment groups: Placebo or Vosoritide. The route of administration is subcutaneous injection, and the frequency of administration is daily.

Arms & Interventions

Arms

Experimental: vosoritide injection with vial and syringe

Placebo Comparator: Placebo injection with vial and syringe

Interventions

Drug: - Vosoritide

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

Drug: - Placebo

Subcutaneous injection of recommended dose of placebo

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Children's National Medical Center, Washington, District of Columbia

Status

Address

Children's National Medical Center

Washington, District of Columbia, 20010

International Sites

Murdoch Children's Research Institute, Parkville, Victoria, Australia

Status

Address

Murdoch Children's Research Institute

Parkville, Victoria, 3052

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