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Active, Not Recruiting

NCT Number: NCT02724228

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Age range

7 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Murdoch Children's Research Institute, Parkville, Victoria, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

BMN 111

Drug

BMN 111 will be administered subcutaneously daily.

Other names: Modified recombinant human C-type natriuretic peptide, Vosoritide

Primary outcomes

  1. 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

  1. Growth Velocity [Efficacy]

    Time frame: Until near final adult height is reached

    Annualized growth velocity (cm/yr)

  2. 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)

  3. 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

  4. 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

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study title

A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia

Acronym: ACH

Important dates

Study start
2016
Primary completion
2027
Study completion
2028
First posted
Mar 31, 2016
Registry last updated
Mar 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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