Apply to Trial

Compensation: Varies

Number of Visits: 12

Duration: 12 months

119 Participants Needed

BMN 111 for Achondroplasia

Recruiting at 23 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: BioMarin Pharmaceutical

Must not be taking: Renal-altering medications

Disqualifiers: Cardiac arrhythmia, Decreased growth, others

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

Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval

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 does not specify if you need to stop taking your current medications, but it does exclude those on medications that alter kidney function. It's best to discuss your specific medications with the study team.

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 increase bone growth. Unlike CNP, which breaks down quickly in the body, vosoritide has a longer-lasting effect, making it effective in increasing growth rates in children with achondroplasia.¹ ² ³ ⁴ ⁵

What is the purpose of this trial?

The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia

Eligibility Criteria

This trial is for children with Achondroplasia who completed Study 111-301. They must be willing to use contraception if sexually active, and girls over 10 or menstruating need a negative pregnancy test. Participants should commit to study procedures, and minors require consent from guardians.

Inclusion Criteria

Must have completed Study 111-301

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

If sexually active, willing to use a highly effective method of contraception while participating in the study

See 2 more

Exclusion Criteria

I am taking medication that affects my kidney function.

I stopped taking BMN 111 or placebo before finishing the 111-301 study.

Require any investigational agent prior to completion of study period

See 5 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive BMN 111 to assess its efficacy and safety in treating children with Achondroplasia

12 months

Visits every 12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive BMN 111 to evaluate long-term safety and efficacy

Up to at least 16 years for females and 18 years for males

Treatment Details

Interventions

BMN 111

Trial Overview The Phase 3 study tests BMN 111's effectiveness and safety in treating Achondroplasia in children. It aims to provide an additional therapeutic option by extending previous research findings from an earlier phase of the study.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: BMN 111Experimental Treatment1 Intervention

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]