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NCT02724228·Phase 2·INTERVENTIONAL

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

Status

Active, not recruiting

Phase

Phase 2

Enrollment

30

Locations

9

Results

Not posted

Publications

2

Study summary

What the protocol is testing.

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.

Interventions

Treatment arms and agents.

DRUG

BMN 111

BMN 111 will be administered subcutaneously daily.

Timeline

From registration to results.

  1. First posted

    Mar 31, 2016

  2. Study start

    Jan 26, 2016

  3. Primary completion

    Dec 2027

  4. Study completion

    Feb 2028

  5. Results posted

    Not reported

  6. Registry updated

    Mar 13, 2026

Outcomes

What the study measures.

Primary outcomes

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

Time frame · 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

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

Secondary outcomes

Growth Velocity [Efficacy]

Time frame · Until near final adult height is reached

Annualized growth velocity (cm/yr)

Growth Parameters (Efficacy)

Time frame · 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

Height standard score (Z-score)

Body Proportions (Efficacy)

Time frame · 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

Upper-to-lower body segment ratio

Final Adult Height

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

Height at the age of 16 years for females and 18 years for males

Eligibility

Who can take part.

Minimum age
7 Years
Maximum age
Not reported
Sex
ALL
Healthy volunteers
No

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.

Study locations

9 registered sites.

Australia · France · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.

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

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