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Trial · Interventional

CompletedPhase 3Results postedRegistry data

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

NCTNCT03197766 (opens ClinicalTrials.gov in a new tab)Other id111-301Other id2015-003836-11Other id2015

Overview

Status
Completed
Phase
Phase 3
Enrollment
121 participants (actual)
Sponsor
BioMarin Pharmaceutical · Industry
Design
Randomized · Triple masking
Countries
7 countries

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

Start
12 Dec 2016
Primary completion
30 Oct 2019
Completion
30 Oct 2019
First posted
23 Jun 2017
Last update posted
2 Mar 2022
Conditions
Achondroplasia

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20162017201820192020
  1. Ph 3

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

    NCT03197766

    12 Dec 2016 to 30 Oct 2019

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
  • PlaceboDrug

Arms2

  • Experimental

    Active BMN 111

    Daily subcutaneous injection of 15 micrograms per kilogram BMN111

  • Placebo comparator

    Placebo

    Daily subcutaneous injection of placebo

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Change From Baseline in Annualized Growth Velocity (AGV) at Week 52

    Time frame · At Baseline and Week 52

    Registry description

    AGV at a Post-baseline Visit is defined as \[(Height at Post-baseline Visit - Height at Baseline)/(Date of Post-baseline Visit - Date of Baseline Assessment)\] x 365.25 AGV at Baseline is defined as \[(Height at Baseline - last height measurement in Study 111-901 at least 6 months prior to Baseline)/(Date of Baseline Assessment - Date of last height measurement in Study 111-901 at least 6 months prior to Baseline)\] x 365.25

Secondary outcomes(3)
  1. Change From Baseline in Height Z-score at Week 52

    Time frame · At baseline and Week 52

    Registry description

    Z-Scores were derived using age-sex specific reference data (means and SDS) for average stature children per the Centers for Disease Control and Prevention. A height Z score of 0 would indicate that the subject's height is equal to the mean height for the average stature population of the same sex and age. A positive height Z score indicates that the subjects height is above the mean height for the average stature population of the same sex and age, whilst a negative height Z score indicates that the subjects height is below the mean height for the average stature population of the same sex and age. To conclude if the height Z score increases then this means the height deficit has decreased.

  2. Change From Baseline in Upper to Lower Segment Body Ratio at Week 52

    Time frame · At baseline and Week 52

    Registry description

    Evaluate change from baseline in mean upper:lower segment body ratio in subjects treated with BMN 111 compared with control subjects in the placebo group at 52 weeks

  3. Summary of Subjects Experiencing Adverse Events (AEs) During Treatment

    Time frame · Up to Week 56

    Registry description

    AEs with onset or worsening after the initiation of study drug and up to 30 days after study drug discontinuation were included. serious adverse event (SAE)

Posted results

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

Primary outcome results

Primary outcomeLeast squares mean· At Baseline and Week 52

Change From Baseline in Annualized Growth Velocity (AGV) at Week 52

GroupValue (cm/year)
BMN 111 - 15 μg/kg1.71
Placebo0.13
Secondary and other outcome results(3)
Secondary outcomeLeast squares mean· At baseline and Week 52

Change From Baseline in Height Z-score at Week 52

GroupValue (Z-Score)
BMN 111 - 15 μg/kg0.27
Placebo-0.01
Secondary outcomeLeast squares mean· At baseline and Week 52

Change From Baseline in Upper to Lower Segment Body Ratio at Week 52

GroupValue (Ratio)
BMN 111 - 15 μg/kg-0.03
Placebo-0.02
Secondary outcomeNumber· Up to Week 56

Summary of Subjects Experiencing Adverse Events (AEs) During Treatment

GroupValue (participants)
BMN 111 - 15 μg/kg59
Placebo60

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
BMN 111 - 15 μg/kgBMN 111: Subcutaneous injection of 15 μg/kg of BMN 111 daily0 / 603 / 6059 / 60
PlaceboPlacebo: Subcutaneous injection of placebo daily0 / 614 / 6160 / 61

Each cell reads participants affected / participants at risk, as the registry posted them.

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

Studies4

Studies that cite this trial.

Studies with results

3 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

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

Indexing hint: Randomized controlled trial · 2022

16 results

Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia.

Chan et al. 2022Clinical pharmacokineticscited by 30

PMID 34431071PMC8813707DOI 10.1007/s40262-021-01059-1PUB-34431071

Free to read · publisher termsCC BY-NC16 results

Eligibility

Who could join, as posted.

Sex
All
Age
from 5 Years up to 18 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria * Parent(s) or guardian(s) consent * 5 to \< 18 years old * ACH, documented and confirmed by genetic testing * At least a 6-month period of pretreatment growth assessment in Study 111-901 before study entry * If sexually active, willing to use a highly effective method of contraception * Ambulatory and able to stand without assistance Exclusion criteria: * Hypochondroplasia or short stature condition other than ACH * Have any of the following: * Hypothyroidism or hyperthyroidism * Insulin-requiring diabetes mellitus * Autoimmune inflammatory disease * Inflammatory bowel disease * Autonomic neuropathy * History of any of the following: * Renal insufficiency defined as serum creatinine \> 2 mg/dL * Chronic anemia * Baseline systolic blood pressure (BP) \< 70 millimeters of mercury (mm Hg) or recurrent symptomatic hypotension (defined as episodes of low BP generally accompanied by symptoms ie, dizziness, fainting) or recurrent symptomatic orthostatic hypotension * Cardiac or vascular disease * Have a clinically significant finding or arrhythmia on screening electrocardiogram (ECG) that indicates abnormal cardiac function or conduction or Fridericias corrected QTc-F \> 450 msec * Have an unstable condition likely to require surgical intervention during the study (including progressive cervical medullary compression or severe untreated sleep apnea) * Decreased growth velocity (\< 1.5 cm/yr) over a period of 6 months or evidence of growth plate closure (proximal tibia, distal femur) * Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or treatment greater than 6 months at any time * Greater than 1 month treatment with oral corticosteroids (low-dose ongoing inhaled steroid for asthma, or intranasal steroids, are acceptable) in the previous 12 months * Planned or expected to have limb-lengthening surgery during the study period. Subjects with previous limb- lengthening surgery may enroll if surgery occurred at least 18 months prior to the study and healing is complete without sequelae. * Planned or expected bone-related surgery (ie. surgery involving disruption of bone cortex, excluding tooth extraction), during the study period. Subjects with previous bone-related surgery may enroll if surgery occurred at least 6 months prior to the study and healing is complete without sequelae. * Had a fracture of the long bones or spine within 6 months prior to screening * History of severe untreated sleep apnea * New initiation of sleep apnea treatment (e.g. CPAP or sleep apnea-mitigating surgery) in the previous 2 months prior to screening * History of hip surgery or hip dysplasia atypical for achondroplastic subjects * History of clinically significant hip injury in the 30 days prior to screening * History of slipped capital femoral epiphysis or avascular necrosis of the femoral head * Abnormal findings on baseline clinical hip exam or imaging assessments that are determined to be clinically significant * Concurrent disease or condition that would interfere with study participation or safety evaluations, for any reason * Condition or circumstance that places the subject at high risk for poor treatment compliance or for not completing the study

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