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Trial · Interventional

Active, not recruitingPhase 2Registry data

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

NCTNCT03989947 (opens ClinicalTrials.gov in a new tab)Other id111-208
Open at ClinicalTrials.govAsk about this trial

Overview

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

United States, Australia, Japan, United Kingdom

Start
12 Jun 2019
Primary completion
May 2038 (estimated)
Completion
May 2038 (estimated)
First posted
18 Jun 2019
Last update posted
13 Mar 2026
Conditions
Achondroplasia

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2020202520302035
  1. Ph 2

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

    NCT03989947

    12 Jun 2019 to est. May 2038

Registry summary

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

This is a Phase 2, open-label multi-center long-term extension study, with approximately 70 subjects, to evaluate the safety and efficacy of BMN111 in children with Achondroplasia until subjects reach near-adult final height. Eligible subjects will have completed 1 year of BMN111 or placebo treatment in the 111-206 study and once enrolled in the 111-208 extension study will receive a daily dose of BMN111 by subcutaneous injection according to their age as determined by 111-206.

Interventions

  • Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily.Drug

Arms1

  • Experimental

    Active BMN 111

    Once daily subcutaneous injections of recommended dose of BMN 111 based on weight-band dosing.

Primary outcomes2

Outcomes the sponsor listed as primary.

  1. Evaluate the incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

    Time frame · "Through study completion, an average of 5 years"

    Registry description

    Number of study participants with treatment-emergent adverse events or serious adverse events

  2. Evaluate change in height/length z-score in children with ACH treated with BMN 111

    Time frame · "Through study completion, an average of 5 years"

Secondary outcomes(15)

  1. Evaluate the change from baseline of mean annualized growth velocity (AGV)

    Time frame · "Through study completion, an average of 5 years"

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

    Time frame · "Through study completion, an average of 5 years"

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

    Time frame · "Through study completion, an average of 5 years"

  4. Characterize the elimination half-life of BMN 111 (t½)

    Time frame · "Through study completion, an average of 5 years"

  5. Characterize the apparent clearance of drug

    Time frame · "Through study completion, an average of 5 years"

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

    Time frame · "Through study completion, an average of 5 years"

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

    Time frame · "Through study completion, an average of 5 years"

  8. Evaluate the change from baseline on body proportion ratios of the extremities

    Time frame · "Through study completion, an average of 5 years"

  9. Effect of BMN 111 on bone morphology and quality by XRay

    Time frame · "Through study completion, an average of 5 years"

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

    Time frame · "Through study completion, an average of 5 years"

  11. Potential Changes in health-related quality of life as measured by the quality of life in Short- statured youth

    Time frame · "Through study completion, an average of 5 years"

    Registry description

    Evaluate the long-term effect of BMN 111 on health-related quality of life, developmental status and functional independence, using age-specific QoL and functional independence questionnaires (Bayley-III, WeeFIM, ITQOL, QoLISSY, PedsQL, Child Behavior Checklist 1.5-5 \[CBCL 1.5-5\], Child Behavior Checklist 6-18 \[CBCL 6-18\]).

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

    Time frame · "Through study completion, an average of 5 years"

  13. Describe the incidence of surgical and medical interventions related to achondroplasia

    Time frame · "Through study completion, an average of 5 years"

  14. Assess effect on sleep disordered breathing by polysomnography in patients up to 5 years old.

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

  15. Evaluate the effect of BMN 111 on skull and brain morphology, including foramen magnum, ventricular and brain parenchymal dimensions by MRI in patients up to 3 years old.

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

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

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 15 Months
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: 1. Must have completed Study 111-206 on investigational treatment (BMN 111 or placebo). 2. 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. 3. Are willing and able to perform all study procedures Exclusion Criteria: 1. Permanently discontinued BMN 111 or placebo prior to completion of Study 111-206 2. Have a clinically significant finding or arrhythmia on ECG that indicates abnormal cardiac function or conduction or QTc-F \> 450 msec 3. Require any investigational agent (except BMN 111) prior to completion of study period 4. Current therapy with antihypertensive medications, angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, GnRH agonists, any medication that may impair or enhance compensatory tachycardia, diuretics, or other drugs known to alter renal or tubular function 5. Pregnant or planning to become pregnant (self or partner) at any time during the study 6. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason 7. Have a condition or circumstance that, in the view of the investigator, places the subject at high risk for poor treatment compliance

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