Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.
Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.: Modified recombinant human C-type natriuretic peptide (subject to adjustment per protocol)
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.
Eligibility
Sex
ALL
Min age
15 Months
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Must have completed Study 111-206 on investigational treatment (BMN 111 or placebo).
2. Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research related procedure. Also, subjects under the age of majority are willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure. Subjects who reach the age of majority in their country while the study is ongoing will be asked to provide their own written consent again upon reaching the legal age of majority.
3. Are willing and able to perform all study procedures
Exclusion Criteria:
1. Permanently discontinued BMN 111 or placebo prior to completion of Study 111-206
2. Have a clinically significant finding or arrhythmia on ECG that indicates abnormal cardiac function or conduction or QTc-F \> 450 msec
3. Require any investigational agent (except BMN 111) prior to completion of study period
4. 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
5. Pregnant or planning to become pregnant (self or partner) at any time during the study
6. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason
7. Have a condition or circumstance that, in the view of the investigator, places the subject at high risk for poor treatment compliance
Primary outcome measure(s)
Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] — "Through study completion, an average of 5 years" Number of study participants with treatment-emergent adverse events or serious adverse events
Evaluate change in height/length z-score in children with ACH treated with BMN 111 — "Through study completion, an average of 5 years"
Trial sites (16)
Facility
City
Region
Status
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
Japan
Saitama Children's Medical Center
Saitama
Japan
Tokushima University Hospital
Tokushima
Japan
Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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