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Compensation: Varies

Number of Visits: Unknown

Duration: 1 year

73 Participants Needed

BMN 111 for Achondroplasia

Recruiting at 15 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: BioMarin Pharmaceutical

Must not be taking: Antihypertensives, ACE inhibitors, Diuretics, others

Disqualifiers: Abnormal cardiac function, Pregnancy, others

No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, including antihypertensive drugs, ACE inhibitors, and others that affect heart or kidney function. If you're on any of these, you may need to stop them to participate.

How does the drug BMN 111 (vosoritide) differ from other treatments for achondroplasia?

BMN 111, also known as vosoritide, is unique because it is a modified version of a natural protein called C-type natriuretic peptide (CNP) that helps promote bone growth. Unlike CNP, which is quickly broken down in the body, vosoritide has a longer half-life, making it more effective in increasing growth velocity in children with achondroplasia.¹ ² ³ ⁴ ⁵

What is the purpose of this trial?

This trial is testing BMN111, an injection, in children with Achondroplasia to help them grow taller. BMN111 has shown promise in promoting growth in children with achondroplasia.

Research Team

Medical Director MD

Principal Investigator

BioMarin Pharmaceutical

Eligibility Criteria

This trial is for children with Achondroplasia who have completed a year in the BMN 111-206 study. They must be able to perform all study procedures and their parents or guardians agree to consent. Children can't join if they have serious heart issues, need other investigational drugs, have conditions affecting study participation or compliance, stopped BMN 111 early in the previous study, are on certain heart or kidney medications, or are pregnant.

Inclusion Criteria

Must have completed Study 111-206 on investigational treatment (BMN 111 or placebo)

I am willing and able to follow all study requirements.

My guardian or I have agreed to participate in the study and signed the consent form.

Exclusion Criteria

Pregnant or planning to become pregnant (self or partner) at any time during the study

I am currently taking medication for high blood pressure or heart issues.

Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason

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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a daily dose of BMN111 by subcutaneous injection according to their age

1 year

Long-term Extension

Participants continue to receive BMN111 until they reach near-adult final height

Up to 5 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

BMN 111

Trial Overview The trial tests long-term safety and effectiveness of BMN 111 given as a daily subcutaneous injection based on weight until near-adult height is reached. It's an open-label Phase 2 extension where around 70 subjects from a prior study continue with BMN 111 treatment.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Active BMN 111Experimental Treatment1 Intervention

Once daily subcutaneous injections of recommended dose of BMN 111 based on weight-band dosing.

Find a Clinic Near You

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

Findings from Research

Achondroplasia, the most common form of dwarfism, is caused by a mutation in the FGFR3 gene that leads to increased inhibition of cartilage formation, resulting in characteristic phenotypic features.

Vosoritide, a modified version of C-type natriuretic peptide (CNP) with a longer half-life, has been shown in clinical trials to significantly increase growth velocity in children with achondroplasia before their growth plates fuse.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of vosoritide in the treatment of achondroplasia.Paton, DM.[2022]

Vosoritide is the first approved treatment for achondroplasia, and parents of children using it reported a strong motivation to manage the daily injectable treatment for potential long-term benefits, such as improved height and independence.

The study highlighted the need for better support and consistent training for families, as experiences with treatment initiation varied significantly, impacting their ability to manage the treatment effectively at home.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Parents' Experience of Administering Vosoritide: A Daily Injectable for Children with Achondroplasia.NiMhurchadha, S., Butler, K., Argent, R., et al.[2023]