A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03583697
Recruitment Status : Enrolling by invitation
First Posted : July 11, 2018
Last Update Posted : January 7, 2019
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Brief Summary:
Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia.

Condition or disease Intervention/treatment Phase
Achondroplasia Drug: BMN 111 Drug: Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia, Age 0 to < 60 Months
Actual Study Start Date : May 23, 2018
Estimated Primary Completion Date : May 2021
Estimated Study Completion Date : May 2021

Arm Intervention/treatment
Experimental: Active BMN111
Daily subcutaneous injection of 15 micrograms per kilogram BMN111
Drug: BMN 111
Subcutaneous injection of 15 μg/kg of BMN 111 daily
Other Names:
  • Vosoritide
  • Modified recombinant human C-type natriuretic peptide

Placebo Comparator: Placebo
Daily subcutaneous injection of placebo
Drug: Placebo
Subcutaneous injection of 15 μg/kg of placebo daily

Primary Outcome Measures :
  1. Evaluate the effect of BMN 111 on change from baseline in length/height Z-scores [ Time Frame: One year ]
    Evaluate change from baseline in length/height Z-score in subjects treated with BMN 111 compared with control subjects in the placebo group at 52 weeks

Secondary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: One year ]

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 59 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of ACH, confirmed by genetic testing
  • Age 0 to < 60 months at study entry (Day 1)
  • At least 6-month period of pretreatment growth assessment in Study 111-901 immediately before study entry (cohort 1 & 2) or at least 3 months of observation prior to treatment (cohort 3)

Exclusion Criteria:

  • Subject weight < 5.0 kg (cohort 1 & 2) or < 4.0 kg (cohort 3)
  • Treatment with growth hormone within 6-months prior to screening or prolonged treatment (> 3 months) at any time
  • Any history of spine or long-bone surgery or any bone-related surgery with chronic complications
  • Any history of limb-lengthening surgery or planned limb-lengthening during the study
  • Fracture of the long bones within 6 months prior to screening

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03583697

United States, California
Children's Hospital & Research Center Oakland
Oakland, California, United States, 94609
Harbor - UCLA Medical Center
Torrance, California, United States, 90509
United States, Georgia
Emory University
Decatur, Georgia, United States, 30033
United States, Illinois
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232-2578
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Australia, Victoria
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Sponsors and Collaborators
BioMarin Pharmaceutical
Study Director: Medical Director, MD BioMarin Pharmaceutical

Additional Information:
Responsible Party: BioMarin Pharmaceutical Identifier: NCT03583697     History of Changes
Other Study ID Numbers: 111-206
2016-003826-18 ( EudraCT Number )
First Posted: July 11, 2018    Key Record Dates
Last Update Posted: January 7, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by BioMarin Pharmaceutical:
Bone Diseases
Bone Diseases, Developmental
Natriuretic Peptide, C-Type
Musculoskeletal Diseases
Natriuretic Agents
Physiological Effect of Drugs
Skeletal Dysplasias
Genetic Diseases, Inborn

Additional relevant MeSH terms:
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Natriuretic Peptide, C-Type
Natriuretic Agents
Physiological Effects of Drugs