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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 : June 30, 2020
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Tracking Information
First Submitted Date  ICMJE February 12, 2016
First Posted Date  ICMJE March 31, 2016
Last Update Posted Date June 30, 2020
Actual Study Start Date  ICMJE January 26, 2016
Estimated Primary Completion Date October 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 29, 2018)
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
Original Primary Outcome Measures  ICMJE
 (submitted: March 25, 2016)
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] [ Time Frame: 260 Weeks ]
  • Number of study participants with treatment-emergent adverse events.
  • Number of study participants with treatment-emergent serious adverse events
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 29, 2018)
  • Growth Velocity [Efficacy] [ Time Frame: Up to 5 years or near final adult height ]
    Annualized growth velocity (cm/yr)
  • Growth Parameters (Efficacy) [ Time Frame: Up to 5 years or near final adult height ]
    Height standard score (Z-score)
  • Body Proportions (Efficacy) [ Time Frame: Up to 5 years or near final adult height ]
    Upper-to-lower body segment ratio
Original Secondary Outcome Measures  ICMJE
 (submitted: March 25, 2016)
  • Growth Velocity [Efficacy] [ Time Frame: 260 Weeks ]
    Annualized growth velocity (cm/yr)
  • Growth Parameters (Efficacy) [ Time Frame: 260 weeks ]
    Height standard score (Z-score)
  • Body Proportions (Efficacy) [ Time Frame: 260 weeks ]
    Upper-to-lower body segment ratio
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Official Title  ICMJE A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With Achondroplasia
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Achondroplasia
Intervention  ICMJE Drug: BMN 111
BMN 111 will be administered subcutaneously daily.
Other Names:
  • Modified recombinant human C-type natriuretic peptide
  • Vosoritide
Study Arms  ICMJE 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.
Intervention: Drug: BMN 111
Publications * Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019 Jul 4;381(1):25-35. doi: 10.1056/NEJMoa1813446. Epub 2019 Jun 18.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: June 8, 2017)
30
Original Estimated Enrollment  ICMJE
 (submitted: March 25, 2016)
46
Estimated Study Completion Date  ICMJE October 2022
Estimated Primary Completion Date October 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 7 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   France,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02724228
Other Study ID Numbers  ICMJE 111-205
2015-004004-30 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party BioMarin Pharmaceutical
Study Sponsor  ICMJE BioMarin Pharmaceutical
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director, MD BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date June 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP