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NCT Number: NCT03989947

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

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.

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Key information

Conditions

Achondroplasia Bone Diseases Bone Diseases, Developmental Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dwarfism Endocrine System Diseases Genetic Diseases, Inborn Musculoskeletal Diseases Osteochondrodysplasias

Age range

15 month and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

The Children's Hospital at Westmead, Westmead, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Must have completed Study 111-206 on investigational treatment (BMN 111 or placebo).
  • 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.
  • Are willing and able to perform all study procedures

Exclusion criteria

  • Permanently discontinued BMN 111 or placebo prior to completion of Study 111-206
  • Have a clinically significant finding or arrhythmia on ECG that indicates abnormal cardiac function or conduction or QTc-F > 450 msec
  • Require any investigational agent (except BMN 111) prior to completion of study period
  • 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
  • Pregnant or planning to become pregnant (self or partner) at any time during the 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

Treatment and study plan

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

Drug

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

Other names: Vosoritide

Primary outcomes

  1. 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

  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

  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"

    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"

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study title

A Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia

Important dates

Study start
2019
Primary completion
2038
Study completion
2038
First posted
Jun 18, 2019
Registry last updated
Mar 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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