Skip to content

Trial · Interventional

Active, not recruitingPhase 3Registry data

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

NCTNCT03424018 (opens ClinicalTrials.gov in a new tab)Other id111-302Other id2017-002404-28Other id2017
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Active, not recruiting
Phase
Phase 3
Enrollment
119 participants (actual)
Sponsor
BioMarin Pharmaceutical · Industry
Design
Allocation not applicable · Open label (no masking)
Countries
7 countries

United States, Australia, Germany, Japan, Spain, Turkey (Türkiye), United Kingdom

Start
12 Dec 2017
Primary completion
Jun 2031 (estimated)
Completion
Jun 2031 (estimated)
First posted
6 Feb 2018
Last update posted
13 Mar 2026
Conditions
Achondroplasia

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
202020252030
  1. Ph 3

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

    NCT03424018

    12 Dec 2017 to est. Jun 2031

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia

Interventions

  • BMN 111Drug

Arms1

  • Experimental

    BMN 111

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Change from baselines in mean annualized growth velocity

    Time frame · Through study completion, an average of 1 year

    Registry description

    Long term efficacy as measured by change in annualized growth velocity

Secondary outcomes(13)

  1. Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire

    Time frame · Through study completion, every 6-12 months

  2. Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire

    Time frame · Through study completion, every 12 months

  3. Characterize maximum concentration (Cmax) of BMN 111 in plasma

    Time frame · Through study completion, every 12 months

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

    Time frame · Through study completion, every 12 months

  5. Characterize the area under the plasma concentration time-curve from time 0 to the last measurable concentration (AUC0-t)

    Time frame · Through study completion, every 12 months

  6. Characterize the elimination half-life of BMN 111 (t1⁄2)

    Time frame · Through study completion, every 12 months

  7. Characterize the apparent clearance of drug

    Time frame · Through study completion, every 12 months

  8. Characterize the apparent volume of distribution based upon the terminal phase (Vz/F)

    Time frame · Through study completion, every 12 months

  9. Characterize the amount of time BMN 111 is present at maximum concentration (Tmax)

    Time frame · Through study completion, every 12 months

  10. BMN 111 Activity Biomarkers

    Time frame · Through study completion, every 12 months

    Registry description

    BMN 111 activity will be assessed by measuring bone and collagen metabolism

  11. Evaluate change from baseline in body proportion ratios of the extremities

    Time frame · Through study completion, every 6 months

  12. Effect of BMN 111 on bone morphology and quality

    Time frame · Through study completion, every 12 months for DXA or 2 years for X-ray

    Registry description

    The effect of BMN 111 on bone morphology/quality will be assessed by measuring bone mineral density via X-Ray and Dual X-ray Absorptiometry

  13. Final Adult Height

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

    Registry description

    Height at 16 years for females and 18 years for males

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies3

Studies that cite this trial.

Studies with results

2 studies on this page

Indexing hint: Randomized controlled trial · 2025

9 results

Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study.

Savarirayan et al. 2025Med (New York, N.Y.)cited by 15

PMID 39740666DOI 10.1016/j.medj.2024.11.019PUB-39740666

Full text at the publisher9 results
Publisher pageSee results (9)

PubMed tag: none · 2024

Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model.

Qi et al. 2024Clinical pharmacokinetics

PMID 38649657PMC11106139DOI 10.1007/s40262-024-01371-6PUB-38649657

Free to read · publisher termsCC BY-NC
Read at source

Indexing hint: Randomized controlled trial · 2024

8 results

Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life.

Savarirayan et al. 2024Genetics in medicine : official journal of the American College of Medical Geneticscited by 15

PMID 39305160DOI 10.1016/j.gim.2024.101274PUB-39305160

Full text at the publisher8 results
Publisher pageSee results (8)

Eligibility

Who could join, as posted.

Sex
All
Age
from 6 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * Must have completed Study 111-301 * Female \>= 10 years old or who have begun menses must have a negative pregnancy test at the Baseline Visit and be willing to have additional pregnancy tests during the study * If sexually active, willing to use a highly effective method of contraception while participating in the study * Are willing and able to perform all study procedures * 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. Exclusion Criteria: * Permanently discontinued BMN 111 or placebo prior to completion of the 111-301 study * Have a clinically significant finding or arrhythmia on Baseline ECG that indicates abnormal cardiac function * Evidence of decreased growth velocity (\<1.5 cm/year) as assessed over a period of at least 6 months or of growth plate closure (proximal tibia, distal femur) through bilateral lower extremity X-rays. * Require any investigational agent prior to completion of study period * Current therapy with medications known to alter renal function * Pregnant or breastfeeding or plan to become pregnant during 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 or for not completing the study.

PepBank is a research reference. Talk to a doctor before you use any peptide.