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Trial · Interventional

Active, not recruitingPhase 2Registry data

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

Short name ACH

NCTNCT02724228 (opens ClinicalTrials.gov in a new tab)Other id111-205Other id2015-004004-30Other id2015

Overview

Status
Active, not recruiting
Phase
Phase 2
Enrollment
30 participants (actual)
Sponsor
BioMarin Pharmaceutical · Industry
Design
Allocation not applicable · Open label (no masking)
Countries
4 countries

United States, Australia, France, United Kingdom

Start
26 Jan 2016
Primary completion
Dec 2027 (estimated)
Completion
Feb 2028 (estimated)
First posted
31 Mar 2016 (estimated)
Last update posted
13 Mar 2026
Conditions
Achondroplasia

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20202025
  1. Ph 2

    ACH

    NCT02724228

    26 Jan 2016 to est. Feb 2028

Registry summary

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

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

  • BMN 111Drug

Arms1

  • Experimental

    BMN 111 - Subcutaneous Injection

    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.

Primary outcomes1

Outcomes the sponsor listed as primary.

  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

    Registry description

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

Secondary outcomes(4)
  1. Growth Velocity [Efficacy]

    Time frame · Until near final adult height is reached

    Registry description

    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

    Registry description

    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

    Registry description

    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

    Registry description

    Height at the age of 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

Studies2

Studies that cite this trial.

Studies with results

1 study on this page

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: Clinical trial · 2019

8 results

C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia.

Savarirayan et al. 2019The New England journal of medicinecited by 162

PMID 31269546DOI 10.1056/nejmoa1813446PUB-31269546

Full text at the publisher8 results

Eligibility

Who could join, as posted.

Sex
All
Age
from 7 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
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.

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