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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ "through study completion, an average of 5 years"

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.

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalLead Sponsor

156 Previous Clinical Trials

190,652 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is the FDA currently approving of Active BMN 111: Daily subcutaneous injection of 15 ug/kg of BMN 111?

"Active BMN 111: Daily subcutaneous injection of 15 ug/kg of BMN 111 has some safety data backing it, but none for efficacy. Therefore, it received a 2."

Answered by AI

Are new patients being accepted into the research program at this time?

"As of right now, this particular trial is not looking for more patients. The posting date was June 12th, 2019 and the last update was on June 30th, 2022. There are currently 12 other studies involving achondroplasia that are actively recruiting participants as well as 5 trials for Active BMN 111: Daily subcutaneous injection of 15 ug/kg of BMN 111."

Answered by AI

Are there other ongoing or previous research studies that have used Active BMN 111 in a similar way?

"BMN 111, a daily subcutaneous injection of 15 ug/kg, was first studied in 2016 at Murdoch Children's Research Institute. Since the completion of 4 trials, there are now 5 active clinical trials being conducted with many of them based in Torrance, California."

Answered by AI

Does this experiment differ significantly from others like it?

"BMN 111: Daily subcutaneous injection of 15 ug/kg has been an active area of research since 2016, when the first study was sponsored by BioMarin Pharmaceutical. After the initial Phase 1 trial with 30 patients in 2016, Phase 2 approval was given. Currently, there are 5 ongoing trials across 16 cities and 8 different countries."

Answered by AI

How many patients are being allowed to enroll in this clinical trial?

"This study is no longer recruiting patients. Per the posting date and most recent update on clinicaltrials.gov, this trial was active from June 12th, 2019 to June 30th, 2022. If you are seeking other studies, there are presently 17 trials actively enrolling patients with achondroplasia or related conditions."

Answered by AI

At how many locations is this experiment being conducted?

"To make things more convenient for study participants, the 9 sites are all located near major cities. These include Torrance, Decatur, Oakland and 6 other locations."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

2019. "An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03989947.

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