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NCT03989947 · ClinicalTrials.gov registry record · Phase 2

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

A Phase 2 study of Achondroplasia, sponsored by BioMarin Pharmaceutical.

Active
Registry status
Phase 2
Development phase
73
Enrollment target
16
Study locations

NCT03989947 is a Phase 2 study of Achondroplasia that is active but no longer recruiting, run by BioMarin Pharmaceutical. The registered enrollment target is 73 participants, below the 143-participant average among 9 other Achondroplasia trials with a reported enrollment target (49% lower). The trial reports 16 study locations across 11 states.

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The verdict

NCT03989947, a Phase 2 study of Achondroplasia, is active but no longer recruiting, sponsored by BioMarin Pharmaceutical.

ACTIVE NOT RECRUITING
Registry status
Phase 2
Development phase
73 participants
Enrollment target
16
Study locations

Study Summary

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.

Conditions Studied

Interventions

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

Study Locations (16)

Other

  • Osaka University - Osaka
  • Saitama Children's Medical Center - Saitama
  • Tokushima University Hospital - Tokushima
  • Guy's and St. Thomas NHS Foundation Trust Evelina Children's Hospital - London
  • Sheffield Children's NHS Foundation Trust - Sheffield

California

  • Children's Hospital & Research Center Oakland - Oakland
  • Harbor - UCLA Medical Center - Torrance

Delaware

  • Alfred I. duPont Hospital for Children - Wilmington

Georgia

  • Emory University - Decatur

Illinois

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

Ohio

  • Cincinnati Childrens Hospital - Cincinnati

Tennessee

  • Vanderbilt University Medical Center - Nashville

Texas

  • Baylor College of Medicine - Houston

Trial Details

FieldValue
Enrollment Target 73 participants
Start Date 2019-06-12
Est. Completion 2038-05
Phase Phase 2

Sponsor

BioMarin Pharmaceutical

80 total trials

What the Registry Record Tells You About NCT03989947

The ClinicalTrials.gov registry entry for NCT03989947 describes a study currently listed as active not recruiting, categorized as Phase 2. The registered enrollment target is 73 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 143-participant average among 9 other Achondroplasia trials with a reported enrollment target (49% lower). The listed sponsor is BioMarin Pharmaceutical, which has 80 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Achondroplasia appearing as the primary indexed condition, and to 1 intervention - of which Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. is the first listed.

NCT03989947 reports 16 study locations spanning 11 distinct geographic areas - top geographies include Other, California, Delaware.

Frequently Asked Questions

What is clinical trial NCT03989947 about?

NCT03989947 is a clinical study titled "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 treatmen...

What is the current status of trial NCT03989947?

This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 73 participants. The study started on 2019-06-12. Estimated completion is 2038-05.

What conditions does trial NCT03989947 study?

This clinical trial studies the following conditions: Achondroplasia.

What interventions are being tested in trial NCT03989947?

The interventions under investigation include: Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. (DRUG).

Who is sponsoring clinical trial NCT03989947?

This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT03989947 being conducted?

This trial has 16 study locations across California, Delaware, Georgia, Illinois, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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