A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

NCT ID: NCT02724228

Last Updated: 2025-01-09

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-01-26
• Study Completion Date: 2028-02-29

Brief Summary

This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.

Conditions

Achondroplasia

Study Design

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

Study Groups

BMN 111 - Subcutaneous Injection

111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen.

Group Type: EXPERIMENTAL

BMN 111

Intervention Type: DRUG

BMN 111 will be administered subcutaneously daily.

Interventions

BMN 111

BMN 111 will be administered subcutaneously daily.

Intervention Type: DRUG

Other Intervention Names

Modified recombinant human C-type natriuretic peptide; Vosoritide

Eligibility Criteria

Inclusion Criteria

• Have completed 24 months of BMN 111 treatment in Study 111-202.
• Parent(s) or guardian(s) are willing and able to provide written, signed informed consent. Subjects under the age of majority are willing and able to provide written assent (if required). Subjects who reach the age of majority in their country will be asked to provide their own written consent upon reaching the legal age of majority.
• If sexually active, willing to use a highly effective method of contraception while participating in the study.
• Females >= 10 years old or who have started menses must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests during the study
• Willing and able to perform all study procedures as physically possible
• Parents/caregivers willing to administer daily injections to the subjects and complete the required training.

Exclusion Criteria

• Requires any investigational agent prior to completion of study period.
• Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study.
• Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations for any reason.
• Permanently discontinued BMN 111 during the 111-202 study.
• Subject is pregnant at Baseline visit or planning to become pregnant (self or partner) at any time during the study.
• Current chronic therapy with restricted medications.

• Minimum Eligible Age: 7 Years
• 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

Ann and Robert H. Lurie Childrens Hospital of Chicago

Chicago, Illinois, United States

Johns Hopkins McKusick - Institute of Genetic Medicine

Baltimore, Maryland, United States

Vanderbilt University

Nashville, Tennessee, United States

Baylor College of Medicine

Houston, Texas, United States

Murdoch Children's Research Institute

Parkville, Victoria, Australia

Institut Necker

Paris, , France

Guys & St. Thomas NHS Foundation Trust Evelina Hospital

London, , United Kingdom

Countries

United States; Australia; France; United Kingdom

References

Qi Y, Chan ML, Mould DR, Larimore K, Fisheleva E, Cherukuri A, Day J, Savarirayan R, Irving M, Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Bober MB, Henshaw J. Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model. Clin Pharmacokinet. 2024 May;63(5):707-719. doi: 10.1007/s40262-024-01371-6. Epub 2024 Apr 23.

Reference Type: DERIVED

PMID: 38649657 (View on PubMed)

Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019 Jul 4;381(1):25-35. doi: 10.1056/NEJMoa1813446. Epub 2019 Jun 18.

Reference Type: DERIVED

PMID: 31269546 (View on PubMed)

Other Identifiers

2015-004004-30

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

111-205

Identifier Type: -

Identifier Source: org_study_id