BMN 111 for Achondroplasia

Not currently 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 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: 1 year

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of BMN 111, a potential new drug for children with Achondroplasia, a condition affecting bone growth. The goal is to determine if BMN 111 helps children grow closer to an average adult height. Children who participated in a previous study with BMN 111 or a placebo and completed it can join this trial, receiving daily injections of BMN 111. Participants should have finished the earlier study without major heart issues or the need for certain medications. As a Phase 2 trial, this research measures how well the treatment works in an initial, smaller group.

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.

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

Research has shown that BMN 111, a treatment for achondroplasia in children, has been studied for safety and tolerability. Studies have found that daily injections of BMN 111 under the skin are generally well-tolerated by children. Some studies have monitored children for over two years to assess long-term safety.

While side effects can occur, they are usually mild. Common side effects include reactions at the injection site, headaches, and vomiting, which often resolve on their own. Overall, evidence suggests that BMN 111 is safe for children with achondroplasia, but each child's experience may vary. It is important to consult a healthcare provider about any concerns. ^{1 2 3 4 5}

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

BMN 111 is unique because it targets achondroplasia at its core by being a C-type natriuretic peptide (CNP) analog. Unlike traditional treatments, which often focus on managing symptoms or complications, BMN 111 aims to address the underlying cause of the condition by promoting bone growth. This new mechanism of action brings hope for more effective treatment outcomes, which is why researchers are excited about its potential. Additionally, BMN 111 is administered through a once-daily subcutaneous injection tailored to the patient's weight, providing a more personalized approach.

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

Research has shown that BMN 111, also known as vosoritide, may benefit children with achondroplasia, a condition affecting bone growth. In one study, children treated with vosoritide grew faster, with their height increasing by 0.45 in height standard deviation scores (H-SDS) after about a year. This suggests the drug could help children with achondroplasia grow taller more quickly than usual. These findings indicate that BMN 111 might effectively improve growth in children with this condition. ^{1 5 6 7 8}

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 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

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

Group I: Active BMN 111 Experimental Treatment 1 Intervention

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

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. 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. clinicaltrials.gov clinicaltrials.gov/ct2/show/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 ...

4. 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].

5. 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 ...

6. 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 ...

7. clinicaltrials.biomarin.com clinicaltrials.biomarin.com/clinical-trial/a-study-to-evaluate-long-term-safety-tolerability-efficacy-of-bmn-111-in-children-with-achondroplasia-ach/

A Study to Evaluate Long-Term Safety, Tolerability, & ... 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.

8. 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 ...