An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia (NCT03989947)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (3 items) and avoid the listed exclusions (8 items).
Location: Children's Hospital & Research Center Oakland — Oakland, California
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria:
- Permanently discontinued BMN 111 or placebo prior to completion of Study 111-206
- Have a clinically significant finding or arrhythmia on ECG that indicates abnormal cardiac function or conduction or QTc-F \> 450 msec
- Require any investigational agent (except BMN 111) prior to completion of study period
- Current therapy with antihypertensive medications, angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, GnRH agonists, any medication that may impair or enhance compensatory tachycardia, diuretics, or other drugs known to alter renal or tubular function
- Pregnant or planning to become pregnant (self or partner) at any time during the study
- Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason
- Have a condition or circumstance that, in the view of the investigator, places the subject at high risk for poor treatment compliance
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (16 Sites)
- Children's Hospital & Research Center Oakland — Oakland, California
- Harbor - UCLA Medical Center — Torrance, California
- Alfred I. duPont Hospital for Children — Wilmington, Delaware
- Emory University — Decatur, Georgia
- Ann Robert and H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- Cincinnati Childrens Hospital — Cincinnati, Ohio
- Vanderbilt University Medical Center — Nashville, Tennessee
- Baylor College of Medicine — Houston, Texas
- Medical College of Wisconsin, Children's Hospital — Milwaukee, Wisconsin
- The Children's Hospital at Westmead — Westmead, New South Wales
- Murdoch Children's Research Institute — Parkville, Victoria
- 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,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.