Vosoritide for Hypochondroplasia

Recruiting at 18 trial locations

Overseen By Trial Specialist

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: BioMarin Pharmaceutical

Must not be taking: Growth hormone, IGF-1, Anabolic steroids

Disqualifiers: Short stature, Unstable condition, Malignancy, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

Duration: 52 weeks

60 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and effectiveness of vosoritide, an injectable medication, for children with hypochondroplasia (HCH), a genetic condition that causes short stature. The trial will include two groups: one receiving vosoritide and the other a placebo (a non-active treatment) to compare results over a year. Children under 36 months with confirmed HCH and specific growth challenges are suitable candidates for this trial. As a Phase 2 trial, the research focuses on assessing the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to early understanding of its impact.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does mention that certain medications are prohibited. It's best to discuss your current medications with the trial team to see if they are allowed.

Is there any evidence suggesting that vosoritide is likely to be safe for humans?

A previous study found that vosoritide was generally well-tolerated by children with hypochondroplasia, meaning it didn't cause unexpected problems. Researchers identified no new major safety issues. Another report indicated that the safety profile of vosoritide matched what is already known from its use in treating achondroplasia, a related condition. This means the side effects were expected and manageable. This information suggests that vosoritide is safe for use in humans, particularly in children with certain growth conditions. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for hypochondroplasia?

Vosoritide is unique because it directly targets the root cause of hypochondroplasia by acting as a C-type natriuretic peptide (CNP) analog. Unlike standard treatments that primarily focus on managing symptoms, vosoritide works by promoting bone growth at the growth plates, potentially addressing the underlying growth deficiency. Researchers are excited about vosoritide because it offers a new approach that could improve growth outcomes and overall quality of life for individuals with hypochondroplasia.

What evidence suggests that vosoritide might be an effective treatment for hypochondroplasia?

Research has shown that vosoritide, which participants in this trial may receive, can help children with hypochondroplasia grow faster. In studies, children who received vosoritide grew more than those who did not receive the treatment. Vosoritide also reduced the outward curve of the shin bone, a common issue in some bone conditions. Real-world data supports that children treated with vosoritide for 24 months experienced significant height increases. These findings suggest that vosoritide can positively impact growth and bone development in children with hypochondroplasia. ^{1 2 6 7 8}

Are You a Good Fit for This Trial?

This trial is for very young children, from birth up to less than 3 years old, who have been diagnosed with Hypochondroplasia (HCH), a genetic bone growth disorder. Specific eligibility details are not provided, but typically participants must meet certain health criteria and not be involved in other studies.

Inclusion Criteria

I have a confirmed genetic diagnosis of HCH.

I am under 3 years old.

My weight is at least 3 kg on the first day of treatment.

Exclusion Criteria

Have received another investigational product or investigational medical device within 30 days prior to the Screening visit

Have known hypersensitivity to vosoritide or its excipients

Have any concurrent disease or condition that, in the view of the investigator, will interfere with study participation or safety evaluations, for any reason

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive daily subcutaneous injections of vosoritide or placebo for 52 weeks

52 weeks

Weekly visits for monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 weeks

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Vosoritide

Trial Overview

The study is testing the safety and effectiveness of vosoritide, administered daily over a year. Vosoritide is compared against a placebo to see if it can improve conditions related to HCH. The trial randomly assigns participants to either the treatment or placebo group.

How Is the Trial Designed?

Group I: Vosoritide injection with vial and syringe Experimental Treatment 1 Intervention

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

Group II: Placebo injection with vial and syringe Placebo Group 1 Intervention

Subcutaneous injection of recommended dose of placebo

Who Is Running the Clinical Trial?

BioMarin Pharmaceutical

Lead Sponsor

Trials

162

Recruited

115,000+

Alexander Hardy

BioMarin Pharmaceutical

Chief Executive Officer since 2023

MBA from INSEAD

Greg Friberg

BioMarin Pharmaceutical

Chief Medical Officer

MD from New York Medical College

ICON Clinical Research

Industry Sponsor

Trials

52

Recruited

15,100+

Citations

1.

thelancet.com

thelancet.com/journals/eclinm/article/PIIS2589-5370(24)00170-6/fulltext

Vosoritide treatment for children with hypochondroplasia

Vosoritide was safe and effective in increasing growth velocity in children with hypochondroplasia. Efficacy was similar to what has been ...

2.

investors.biomarin.com

investors.biomarin.com/news/news-details/2025/BioMarin-Presents-New-Data-for-VOXZOGO-vosoritide-in-Children-with-Achondroplasia-and-Other-Skeletal-Conditions-at-Two-International-Endocrinology-Meetings/default.aspx

BioMarin Presents New Data for VOXZOGO® (vosoritide) ...

Children who received VOXZOGO had a significant reduction in the magnitude of tibial bowing compared to children who received placebo.

3.

sciencedirect.com

sciencedirect.com/science/article/pii/S2949774424001584

Vosoritide increases growth velocity in hypochondroplasia ...

The primary outcomes are rate of adverse events and change in AGV and height SD from baseline. Secondary outcomes included measurement of body proportions and ...

4.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07126262

NCT07126262 | A Study of Vosoritide Versus Placebo in ...

Study Overview. The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH ...

5.

rarediseaseadvisor.com

rarediseaseadvisor.com/news/real-world-studies-demonstrated-vosoritides-growth-benefits-achondroplasia/

Real-World Data Support Efficacy of Vosoritide in Children ...

Those treated for 24 months exhibited even greater growth, with an average height gain of 11.86 cm and a Z-score improvement of 1.15. These ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04219007

Vosoritide for Selected Genetic Causes of Short Stature

To evaluate immunogenicity and assess impact on safety and efficacy measures after 12 months of daily SC injections of vosoritide as evidenced by rates of ...

7.

childrensnational.org

childrensnational.org/about-us/newsroom/2024/first-clinical-trial-of-vosoritide-for-children-with-hypochondroplasia-shows-increased-growth

First clinical trial of vosoritide for children with ...

Vosoritide's first global phase 2 study showed an average increased growth rate of 1.8 cm per year in children with hypochondroplasia, a genetic cause of short ...

8.

clinicaltrials.ucsf.edu

clinicaltrials.ucsf.edu/trial/NCT07126262

A Study of Vosoritide Versus Placebo in Children With ...

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 ...