Current partner codePEPTIDESDE
NCT03989947·Phase 2·INTERVENTIONAL

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

Status

Active, not recruiting

Phase

Phase 2

Enrollment

73

Locations

16

Results

Not posted

Publications

0

Study summary

What the protocol is testing.

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

Treatment arms and agents.

DRUG

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

Modified recombinant human C-type natriuretic peptide (subject to adjustment per protocol)

Timeline

From registration to results.

  1. First posted

    Jun 18, 2019

  2. Study start

    Jun 12, 2019

  3. Primary completion

    May 2038

  4. Study completion

    May 2038

  5. Results posted

    Not reported

  6. Registry updated

    Mar 13, 2026

Outcomes

What the study measures.

Primary outcomes

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

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

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

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

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

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

Characterize maximum concentration (Cmax) of BMN 111 in plasma

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

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"

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

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

Characterize the apparent clearance of drug

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

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

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

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

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

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

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

Effect of BMN 111 on bone morphology and quality by XRay

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

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"

Eligibility

Who can take part.

Minimum age
15 Months
Maximum age
Not reported
Sex
ALL
Healthy volunteers
No

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

Study locations

16 registered sites.

Australia · Japan · United Kingdom · United States. Showing up to 24 locations stored in the fast local snapshot.

Children's Hospital & Research Center Oakland

Oakland, California, United States

Harbor - UCLA Medical Center

Torrance, California, United States

Alfred I. duPont Hospital for Children

Wilmington, Delaware, United States

Emory University

Decatur, Georgia, United States

Ann Robert and H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Cincinnati Childrens Hospital

Cincinnati, Ohio, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Baylor College of Medicine

Houston, Texas, United States

Medical College of Wisconsin, Children's Hospital

Milwaukee, Wisconsin, United States

The Children's Hospital at Westmead

Westmead, New South Wales, Australia

Murdoch Children's Research Institute

Parkville, Victoria, Australia

Osaka University

Osaka, Japan

Saitama Children's Medical Center

Saitama, Japan

Tokushima University Hospital

Tokushima, Japan

Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital

London, United Kingdom

Sheffield Children's NHS Foundation Trust

Sheffield, United Kingdom

Publications

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

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