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NCT03989947 · ClinicalTrials.gov registry record · Phase 2
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
A Phase 2 study of Achondroplasia, sponsored by BioMarin Pharmaceutical.
- Active
- Registry status
- Phase 2
- Development phase
- 73
- Enrollment target
- 16
- Study locations
NCT03989947: Active Phase 2 study of Achondroplasia, sponsored by BioMarin Pharmaceutical.
NCT03989947 is a Phase 2 study of Achondroplasia that is active but no longer recruiting, run by BioMarin Pharmaceutical. The registered enrollment target is 73 participants, below the 143-participant average among 9 other Achondroplasia trials with a reported enrollment target (49% lower). The trial reports 16 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03989947, a Phase 2 study of Achondroplasia, is active but no longer recruiting, sponsored by BioMarin Pharmaceutical.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 73 participants
- Enrollment target
- 16
- Study locations
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.
Primary Outcome
Number of study participants with treatment-emergent adverse events or serious adverse events
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 |
What the registry record for NCT03989947 still lists
NCT03989947 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 73 participants, a relatively small participant target, below the 143-participant average among 9 other Achondroplasia trials with a reported enrollment target (49% lower).
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.
NCT03989947 lists 16 locations in 11 states (Other, California, Delaware).
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.
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).
Who is sponsoring clinical trial NCT03989947?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for Achondroplasia
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03989947's enrollment target sits among peer trials
73 8th of 9 higher than 2 of 9 other Achondroplasia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Achondroplasia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
RECRUITING · Phase 2
-
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
RECRUITING · Phase 2
-
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
RECRUITING · Phase 2
-
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
ACTIVE NOT RECRUITING · Phase 2
-
VIrtual STudy in Achondroplasia for the US (VISTA)
RECRUITING
-
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
ACTIVE NOT RECRUITING
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