NCT02724228

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.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
21mo left

Started Jan 2016

Longer than P75 for phase_2

Geographic Reach
4 countries

9 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress86%
Jan 2016Feb 2028

Study Start

First participant enrolled

January 26, 2016

Completed
17 days until next milestone

First Submitted

Initial submission to the registry

February 12, 2016

Completed
2 months until next milestone

First Posted

Study publicly available on registry

March 31, 2016

Completed
11.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2027

Expected
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2028

Last Updated

March 13, 2026

Status Verified

March 1, 2026

Enrollment Period

11.9 years

First QC Date

February 12, 2016

Last Update Submit

March 12, 2026

Conditions

Achondroplasia

Keywords

AchondroplasiaDwarfismBone Diseases, DevelopmentalBone Diseases

Outcome Measures

Primary Outcomes (1)

  • Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

    * Number of study participants with treatment-emergent adverse events. * Number of study participants with treatment-emergent serious adverse events

    Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later

Secondary Outcomes (4)

  • Growth Velocity [Efficacy]

    Until near final adult height is reached

  • Growth Parameters (Efficacy)

    Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later

  • Body Proportions (Efficacy)

    Until near final adult height is reached, and up to at least 16 years of age for females and 18 years of age for males, whichever occurs later

  • Final Adult Height

    Up to at least 16 years of age for females and 18 years of age for males

Study Arms (1)

BMN 111 - Subcutaneous Injection

EXPERIMENTAL

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.

Drug: BMN 111

Interventions

BMN 111DRUG

BMN 111 will be administered subcutaneously daily.

Also known as: Modified recombinant human C-type natriuretic peptide, Vosoritide
BMN 111 - Subcutaneous Injection

Eligibility Criteria

Age7 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Children's Hospital & Research Center Oakland

Oakland, California, 94609, United States

Location

Harbor - UCLA Medical Center

Torrance, California, 90509, United States

Location

Ann and Robert H. Lurie Childrens Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Johns Hopkins McKusick - Institute of Genetic Medicine

Baltimore, Maryland, 21287, United States

Location

Vanderbilt University

Nashville, Tennessee, 37232-2578, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

Location

Institut Necker

Paris, 75015, France

Location

Guys & St. Thomas NHS Foundation Trust Evelina Hospital

London, SE1 9RT, United Kingdom

Location

Related Publications (2)

  • 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.

    PMID: 38649657DERIVED

  • 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.

    PMID: 31269546DERIVED

MeSH Terms

Conditions

AchondroplasiaDwarfismBone Diseases, DevelopmentalBone Diseases

Interventions

vosoritide

Condition Hierarchy (Ancestors)

Musculoskeletal DiseasesOsteochondrodysplasiasGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesEndocrine 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

February 12, 2016

First Posted

March 31, 2016

Study Start

January 26, 2016

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

February 1, 2028

Last Updated

March 13, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)US(6)AU(1)GB(1)