BMN 111 for Achondroplasia

Not currently recruiting at 24 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 Group All 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 Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 12

Duration: 12 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests BMN 111, an experimental treatment for children with achondroplasia, a condition affecting bone growth. The goal is to determine if BMN 111 can improve growth in children who participated in a previous study on this treatment. Children who completed the earlier study and can perform study tasks are suitable candidates for this trial. As a Phase 3 trial, it represents the final step before FDA approval, providing an opportunity to contribute to the potential approval of a promising treatment.

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.

Is there any evidence suggesting that BMN 111 is likely to be safe for humans?

Research has shown that BMN 111, also known as vosoritide, has been tested for safety in children with Achondroplasia. Earlier studies examined how well children tolerate this treatment. For instance, one study assessed the safety of daily BMN 111 injections over two years in children with Achondroplasia, and the results indicated that the treatment was generally well-tolerated.

Ongoing research continues to monitor the long-term safety of BMN 111 by observing patients already using the treatment to evaluate their responses over time.

In summary, studies suggest that BMN 111 is generally well-tolerated in children, with ongoing research to ensure its continued safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

BMN 111 is unique because it targets the underlying cause of achondroplasia by using a C-type natriuretic peptide (CNP) analog. Unlike traditional treatments like surgery or growth hormone therapy, which manage symptoms, BMN 111 aims to address the root of the condition by promoting bone growth directly. Researchers are excited about its potential to significantly improve growth outcomes and quality of life for those affected by achondroplasia, offering a more targeted approach than existing options.

What evidence suggests that BMN 111 might be an effective treatment for Achondroplasia?

Research has shown that BMN 111, also known as vosoritide, holds promise for treating children with achondroplasia. Studies have found that vosoritide can accelerate growth in children. For instance, one study revealed that children taking vosoritide grew an average of 0.45 standard height scores more over about a year. Children treated with vosoritide demonstrated better growth compared to those who did not receive the treatment. These findings suggest that BMN 111 could effectively aid growth in children with achondroplasia. ^{5 6 7 8 9}

Are You a Good Fit for This Trial?

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

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

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

Group I: BMN 111 Experimental Treatment 1 Intervention

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

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]

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]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03197766

A Study to Evaluate the Efficacy and Safety of BMN 111 in ... This is a Phase 3 randomized, placebo-controlled, double-blind multicenter study with approximately 110 subjects, aged 5 to < 18 years old.

2. medical.biomarin.com medical.biomarin.com/en-us/wp-content/uploads/sites/2/2024/03/ACMG-2024-208-Results_Approved-for-use.pdf?v=1.0

Results from a phase II extension study with vosoritide ... trial (BMN 111-202) and its extension study. (BMN 111-205) in children with ACH aged ≥ 5 years showed that vosoritide treatment resulted in ...

3. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2021/214938Orig1s000IntegratedR.pdf

214938Orig1s000 - accessdata.fda.gov Tolerability, and Efficacy of BMN-111 in Children with Achondroplasia (who completed. Study 111-202).” • On February 29, 2016, the Office of Pharmaceutical ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT02055157

NCT02055157 | A Phase 2 Study of BMN 111 to Evaluate ... This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10796712/

Vosoritide Therapy in Children with Achondroplasia Data from CrescNet show that an increase of 0.45 H-SDS was observed in 85 patients treated with vosoritide after 1.23 years (SD 0.75) [31].

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03424018

NCT03424018 | An Extension Study to Evaluate the ... 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. Official Title.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT02724228

NCT02724228 | A Study to Evaluate Long-Term Safety, ... 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 ...

8. clinicaltrials.biomarin.com clinicaltrials.biomarin.com/clinical-trial/a-clinical-trial-to-evaluate-safety-of-vosoritide-in-at-risk-infants-with-achondroplasia/

A Clinical Trial to Evaluate Safety of Vosoritide in At-risk ... A Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk.

9. catalogues.ema.europa.eu catalogues.ema.europa.eu/node/3537/administrative-details

A multicentre, non-interventional study to evaluate long-term ... This post-authorisation safety study (PASS, BMN 111-603) will evaluate the long-term safety of treatment with Voxzogo in subjects with achondroplasia (ACH) in ...