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Clinical Trials in the USA / NCT03989947
Active, not recruiting Phase 2

An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia

NCT03989947 · tracked via the Priya Life Science USA tracker
Sponsor
BioMarin Pharmaceutical
Phase
Phase 2
Started
2019-06-12
Last updated
2026-03-13

Condition(s) studied

Achondroplasia

Investigational drug(s) / intervention(s)

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)

Trial sites (16)

FacilityCityRegionStatus
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 London United Kingdom
Sheffield Children's NHS Foundation Trust Sheffield United Kingdom

More BioMarin Pharmaceutical trials in the USA

Other trials for the same condition

Official registry record

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.

View NCT03989947 on ClinicalTrials.gov ↗ ← All trials in the USA