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

NCT03989947 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: 111-208
• Study Type: interventional
• Target: 73
• Locations: 4 countries
• Sites: 16

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

Keywords

Dwarfism; Bone Disease; ACH; Natriuretic Peptide C-Type; Musculoskeletal Diseases; Skeletal Dysplasias

Outcome Measures

Primary Outcomes (2)

• Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

  Number of study participants with treatment-emergent adverse events or serious adverse events

  "Through study completion, an average of 5 years"

• Evaluate change in height/length z-score in children with ACH treated with BMN 111

  "Through study completion, an average of 5 years"

Secondary Outcomes (15)

• Evaluate the change from baseline of mean annualized growth velocity (AGV)

  "Through study completion, an average of 5 years"

• Characterize maximum concentration (Cmax) of BMN 111 in plasma

  "Through study completion, an average of 5 years"

• Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞)

  "Through study completion, an average of 5 years"

• Characterize the elimination half-life of BMN 111 (t½)

  "Through study completion, an average of 5 years"

• Characterize the apparent clearance of drug

  "Through study completion, an average of 5 years"

Study Arms (1)

Active BMN 111

EXPERIMENTAL

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

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

Interventions

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

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

Also known as: Vosoritide

Active BMN 111

Eligibility Criteria

• Age: 15 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Must have completed Study 111-206 on investigational treatment (BMN 111 or placebo).
• 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.
• Are willing and able to perform all study procedures

You may not qualify if:

• Permanently discontinued BMN 111 or placebo prior to completion of Study 111-206
• Have a clinically significant finding or arrhythmia on ECG that indicates abnormal cardiac function or conduction or QTc-F \> 450 msec
• Require any investigational agent (except BMN 111) prior to completion of study period
• 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
• Pregnant or planning to become pregnant (self or partner) at any time during the study
• Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason
• Have a condition or circumstance that, in the view of the investigator, places the subject at high risk for poor treatment compliance

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (16)

Children's Hospital & Research Center Oakland

Oakland, California, 94609, United States

Location

Harbor - UCLA Medical Center

Torrance, California, 90509, United States

Location

Alfred I. duPont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Emory University

Decatur, Georgia, 30033, United States

Location

Ann Robert and H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Cincinnati Childrens Hospital

Cincinnati, Ohio, 45229, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-2578, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Medical College of Wisconsin, Children's Hospital

Milwaukee, Wisconsin, 53226, United States

Location

The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

Location

Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

Location

Osaka University

Osaka, 565-0871, Japan

Location

Saitama Children's Medical Center

Saitama, 330-8777, Japan

Location

Tokushima University Hospital

Tokushima, 77-8503, Japan

Location

Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital

London, SE19RT, United Kingdom

Location

Sheffield Children's NHS Foundation Trust

Sheffield, S102TH, United Kingdom

Location

MeSH Terms

Conditions

Achondroplasia; Dwarfism; Bone Diseases; Musculoskeletal Diseases

Interventions

vosoritide

Condition Hierarchy (Ancestors)

Bone Diseases, Developmental; Osteochondrodysplasias; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Endocrine System Diseases

Study Officials

• Medical Director MD

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 22, 2019
• First Posted: June 18, 2019
• Study Start: June 12, 2019
• Primary Completion (Estimated): May 1, 2038
• Study Completion (Estimated): May 1, 2038
• Last Updated: March 13, 2026

Record last verified: 2026-03

Locations

US (9); GB (2); AU (2); JP (3)