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ACTIVE NOT RECRUITING
NCT03989947
PHASE2

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

Sponsor: BioMarin Pharmaceutical

View on ClinicalTrials.gov

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.

Official title: A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia

Key Details

Gender

All

Age Range

15 Months - Any

Study Type

INTERVENTIONAL

Enrollment

73

Start Date

2019-06-12

Completion Date

2038-05

Last Updated

2026-03-13

Healthy Volunteers

No

Conditions

Interventions

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)

Locations (16)

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