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Peptide

BMN 111 for Achondroplasia

Phase 2
Waitlist Available
Research Sponsored by BioMarin Pharmaceutical
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up "through study completion, an average of 5 years"
Awards & highlights

Study Summary

This trial is testing a drug to see if it's safe and effective for children with achondroplasia, a form of dwarfism, until they reach their full adult height.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While specific side effects aren't listed here, potential risks may include reactions at the injection site, general discomforts like headaches or nausea, and any unforeseen complications related to growth patterns due to drug action.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~"through study completion, an average of 5 years"
This trial's timeline: 3 weeks for screening, Varies for treatment, and "through study completion, an average of 5 years" for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Evaluate change in height/length z-score in children with ACH treated with BMN 111
Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Secondary outcome measures
Assess effect on sleep disordered breathing by polysomnography in patients up to 5 years old.
BMN 111 activity will be assessed by measuring bone and collagen metabolism
Characterize maximum concentration (Cmax) of BMN 111 in plasma
+12 more

Trial Design

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.

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Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor
157 Previous Clinical Trials
189,938 Total Patients Enrolled
13 Trials studying Achondroplasia
3,042 Patients Enrolled for Achondroplasia
Medical Director MDStudy DirectorBioMarin Pharmaceutical

Media Library

BMN 111 (Peptide) Clinical Trial Eligibility Overview. Trial Name: NCT03989947 — Phase 2
Achondroplasia Research Study Groups: Active BMN 111
Achondroplasia Clinical Trial 2023: BMN 111 Highlights & Side Effects. Trial Name: NCT03989947 — Phase 2
BMN 111 (Peptide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03989947 — Phase 2
~22 spots leftby Sep 2026
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