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

NCT ID: NCT03989947

Last Updated: 2025-01-09

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 73 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-06-12
• Study Completion Date: 2038-05-31

Brief 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.

Conditions

Achondroplasia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Active BMN 111

Once daily subcutaneous injections of recommended dose of BMN 111 based on weight-band dosing.

Group Type: EXPERIMENTAL

Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.

Intervention Type: DRUG

Modified recombinant human C-type natriuretic peptide (subject to adjustment per protocol)

Interventions

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)

Intervention Type: DRUG

Other Intervention Names

Vosoritide

Eligibility Criteria

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

• Minimum Eligible Age: 15 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

BioMarin Pharmaceutical

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director MD

Role: STUDY_DIRECTOR

BioMarin Pharmaceutical

Locations

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

Countries

United States; Australia; Japan; United Kingdom

Other Identifiers

111-208

Identifier Type: -

Identifier Source: org_study_id