BMN 111 for Achondroplasia

(ACH Trial)

Recruiting at 8 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: BioMarin Pharmaceutical

Must be taking: BMN 111

Must not be taking: Investigational agents

Disqualifiers: Pregnancy, Poor compliance, Others

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

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

Compensation: Varies

Number of Visits: Unknown

Duration: Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.

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 mentions that you cannot be on chronic therapy with restricted medications. 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 substance called C-type natriuretic peptide (CNP), which helps promote bone growth. Unlike CNP, vosoritide has a longer half-life, making it more effective in increasing growth velocity in children with achondroplasia. ^{1 2 3 4 5}

Who Is on the Research Team?

Medical Director, MD

Principal Investigator

BioMarin Pharmaceutical

Are You a Good Fit for This Trial?

This trial is for children with Achondroplasia who have already completed 24 months of BMN 111 treatment in a previous study. They must be able to perform study procedures, use contraception if sexually active, and not be pregnant. Those reaching legal adult age during the study will need to consent again.

Inclusion Criteria

I am willing and able to follow all study requirements.

Parent(s) or guardian(s) are willing and able to provide written, signed informed consent. Subjects under the age of majority are willing and able to provide written assent (if required). Subjects who reach the age of majority in their country will be asked to provide their own written consent upon reaching the legal age of majority

I am a female over 10 or have started my periods and can take pregnancy tests during the study.

Exclusion Criteria

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

Subject is pregnant at Baseline visit or planning to become pregnant (self or partner) at any time during the study

I am on long-term medication that is restricted.

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 BMN 111

Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

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

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• BMN 111

Trial Overview The trial tests the long-term safety and effectiveness of daily injections of BMN 111 in children with Achondroplasia over an extended period after they've been treated for two years in a prior study (111-202).

How Is the Trial Designed?

Group I: BMN 111 - Subcutaneous Injection Experimental Treatment 1 Intervention

111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen.

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

Published Research Related to This Trial

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]

Citations

1. United States pubmed.ncbi.nlm.nih.gov

Whole-body and segmental analysis of body composition in adult males with achondroplasia using dual X-ray absorptiometry. [2020]

2. Spain pubmed.ncbi.nlm.nih.gov

Efficacy of vosoritide in the treatment of achondroplasia. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Parents' Experience of Administering Vosoritide: A Daily Injectable for Children with Achondroplasia. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Whole body bone mass and body composition in a girl with achondroplasia, at ages 9 through 12. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Achondroplasia: a comprehensive clinical review. [2023]