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An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
NCT03989947 · View on ClinicalTrials.gov ↗
Study Summary
This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.
Conditions Studied
Interventions
- DRUG Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Study Locations (16)
Other
- Osaka University — Osaka
- Saitama Children's Medical Center — Saitama
- Tokushima University Hospital — Tokushima
- Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital — London
- Sheffield Children's NHS Foundation Trust — Sheffield
California
- Children's Hospital & Research Center Oakland — Oakland
- Harbor - UCLA Medical Center — Torrance
Delaware
- Alfred I. duPont Hospital for Children — Wilmington
Georgia
- Emory University — Decatur
Illinois
- Ann Robert and H. Lurie Children's Hospital of Chicago — Chicago
Ohio
- Cincinnati Childrens Hospital — Cincinnati
Tennessee
- Vanderbilt University Medical Center — Nashville
Texas
- Baylor College of Medicine — Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 73 participants |
| Start Date | 2019-06-12 |
| Est. Completion | 2038-05 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03989947
The ClinicalTrials.gov registry entry for NCT03989947 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 73 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is BioMarin Pharmaceutical, which has 31 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Achondroplasia appearing as the primary indexed condition, and to 1 intervention — of which Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03989947 reports 16 study locations spanning 11 distinct geographic areas — top geographies include Other, California, Delaware. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03989947 about?
NCT03989947 is a clinical study titled "An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia". This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatmen...
What is the current status of trial NCT03989947?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 73 participants. The study started on 2019-06-12. Estimated completion is 2038-05.
What conditions does trial NCT03989947 study?
This clinical trial studies the following conditions: Achondroplasia. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03989947?
The interventions under investigation include: Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03989947?
This trial is sponsored by BioMarin Pharmaceutical, which has 31 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03989947 being conducted?
This trial has 16 study locations across California, Delaware, Georgia, Illinois, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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