DRUG
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)
Status
Active, not recruiting
Phase
Phase 2
Enrollment
73
Locations
16
Results
Not posted
Publications
0
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.
Interventions
DRUG
Modified recombinant human C-type natriuretic peptide (subject to adjustment per protocol)
Timeline
First posted
Jun 18, 2019
Study start
Jun 12, 2019
Primary completion
May 2038
Study completion
May 2038
Results posted
Not reported
Registry updated
Mar 13, 2026
Outcomes
Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame · "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
Time frame · "Through study completion, an average of 5 years"
Evaluate the change from baseline of mean annualized growth velocity (AGV)
Time frame · "Through study completion, an average of 5 years"
Characterize maximum concentration (Cmax) of BMN 111 in plasma
Time frame · "Through study completion, an average of 5 years"
Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞)
Time frame · "Through study completion, an average of 5 years"
Characterize the elimination half-life of BMN 111 (t½)
Time frame · "Through study completion, an average of 5 years"
Characterize the apparent clearance of drug
Time frame · "Through study completion, an average of 5 years"
Characterize the apparent volume of distribution based upon the terminal phase (Vz/F)
Time frame · "Through study completion, an average of 5 years"
Characterize the amount of time BMN 111 is present at maximum concentration (Tmax)
Time frame · "Through study completion, an average of 5 years"
Evaluate the change from baseline on body proportion ratios of the extremities
Time frame · "Through study completion, an average of 5 years"
Effect of BMN 111 on bone morphology and quality by XRay
Time frame · "Through study completion, an average of 5 years"
The effect of BMN 111 on bone morphology/quality will be assessed by measuring bone mineral density via Dual X-ray Absorptiometry
Time frame · "Through study completion, an average of 5 years"
Eligibility
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
Study locations
Australia · Japan · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.
Children's Hospital & Research Center Oakland
Oakland, California, United States
Harbor - UCLA Medical Center
Torrance, California, United States
Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States
Emory University
Decatur, Georgia, United States
Ann Robert and H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Cincinnati Childrens Hospital
Cincinnati, Ohio, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Baylor College of Medicine
Houston, Texas, United States
Medical College of Wisconsin, Children's Hospital
Milwaukee, Wisconsin, United States
The Children's Hospital at Westmead
Westmead, New South Wales, Australia
Murdoch Children's Research Institute
Parkville, Victoria, Australia
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
London, United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield, United Kingdom
Publications
No PMID-linked publications were present in this registry snapshot.
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