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A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) (ACH)

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.
ClinicalTrials.gov Identifier: NCT02724228
Recruitment Status : Active, not recruiting
First Posted : March 31, 2016
Last Update Posted : May 31, 2018
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Brief Summary:
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
Actual Study Start Date : January 26, 2016
Estimated Primary Completion Date : October 2022
Estimated Study Completion Date : October 2022


Arm Intervention/treatment
Experimental: BMN 111 - Subcutaneous Injection
111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study. BMN 111 will be administered in one of the following daily dosing regimens: 15 μg/kg or 30 μg/kg daily.
Drug: BMN 111
BMN 111 will be administered subcutaneously daily.
Other Names:
  • Modified recombinant human C-type natriuretic peptide
  • Vosoritide




Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: Through study completion, every 3 months ]
    • Number of study participants with treatment-emergent adverse events.
    • Number of study participants with treatment-emergent serious adverse events


Secondary Outcome Measures :
  1. Growth Velocity [Efficacy] [ Time Frame: Up to 5 years or near final adult height ]
    Annualized growth velocity (cm/yr)

  2. Growth Parameters (Efficacy) [ Time Frame: Up to 5 years or near final adult height ]
    Height standard score (Z-score)

  3. Body Proportions (Efficacy) [ Time Frame: Up to 5 years or near final adult height ]
    Upper-to-lower body segment ratio



Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Have completed 24 months of BMN 111 treatment in Study 111-202.
  • Parent(s) or guardian(s) are willing and able to provide written, signed informed consent. Subjects under the age of majority are willing and able to provide written assent (if required). Subjects who reach the age of majority in their country will be asked to provide their own written consent upon reaching the legal age of majority.
  • If sexually active, willing to use a highly effective method of contraception while participating in the study.
  • Females >= 10 years old or who have started menses must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests during the study
  • Willing and able to perform all study procedures as physically possible
  • Parents/caregivers willing to administer daily injections to the subjects and complete the required training.

Exclusion Criteria:

  • Requires any investigational agent prior to completion of study period.
  • Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study.
  • Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations for any reason.
  • Permanently discontinued BMN 111 during the 111-202 study.
  • Subject is pregnant at Baseline visit or planning to become pregnant (self or partner) at any time during the study.
  • Current chronic therapy with restricted medications.

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 ClinicalTrials.gov identifier (NCT number): NCT02724228


Locations
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, Illinois
Ann and Robert H. Lurie Childrens Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Maryland
Johns Hopkins McKusick - Institute of Genetic Medicine
Baltimore, Maryland, United States, 21287
United States, Tennessee
Vanderbilt University
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
France
Institut Necker
Paris, France, 75015
United Kingdom
Guys & St. Thomas NHS Foundation Trust Evelina Hospital
London, United Kingdom, SE1 9RT
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
Study Director: Medical Director, MD BioMarin Pharmaceutical

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT02724228     History of Changes
Other Study ID Numbers: 111-205
2015-004004-30 ( EudraCT Number )
First Posted: March 31, 2016    Key Record Dates
Last Update Posted: May 31, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by BioMarin Pharmaceutical:
Achondroplasia
Dwarfism
Bone Diseases, Developmental
Bone Diseases

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