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

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ClinicalTrials.gov Identifier: NCT03424018
Recruitment Status : Enrolling by invitation
First Posted : February 6, 2018
Last Update Posted : July 30, 2018
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Brief Summary:
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia

Condition or disease Intervention/treatment Phase
Achondroplasia Drug: BMN 111 Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 110 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With Achondroplasia
Actual Study Start Date : December 12, 2017
Estimated Primary Completion Date : October 2024
Estimated Study Completion Date : December 2024


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




Primary Outcome Measures :
  1. Change from baselines in mean annualized growth velocity [ Time Frame: Through study completion, an average of 1 year ]
    Long term efficacy as measured by change in annualized growth velocity


Secondary Outcome Measures :
  1. Changes in health-related quality of life as measured by the Quality of Life in Short-Statured Youth questionnaire [ Time Frame: Through study completion, every 6 months ]
  2. Potential changes in daily activity performance as measured by Activities of Daily Living questionnaire [ Time Frame: Through study completion, every 6 months ]
  3. Characterize maximum concentration (Cmax) of BMN 111 in plasma [ Time Frame: Through study completion, every 6 months ]
  4. Characterize the area under the plasma concentration time-curve from time 0 to infinity (AUC0-∞) [ Time Frame: Through study completion, every 6 months ]
  5. Characterize the area under the plasma concentration time-curve from time 0 to the last measurable concentration (AUC0-t) [ Time Frame: Through study completion, every 6 months ]
  6. Characterize the elimination half-life of BMN 111 (t1⁄2) [ Time Frame: Through study completion, every 6 months ]
  7. Characterize the apparent clearance of drug [ Time Frame: Through study completion, every 6 months ]
  8. Characterize the apparent volume of distribution based upon the terminal phase (Vz/F) [ Time Frame: Through study completion, every 6 months ]
  9. Characterize the amount of time BMN 111 is present at maximum concentration (Tmax) [ Time Frame: Through study completion, every 6 months ]

Other Outcome Measures:
  1. BMN 111 Activity Biomarkers [ Time Frame: Through study completion, every 6 months ]
    BMN 111 activity will be assessed by measuring bone and collagen metabolism

  2. Evaluate sleep study scores by polysomnography [ Time Frame: Week 260 ]
  3. Evaluate change from baseline in body proportion ratios of the extremities [ Time Frame: Through study completion, every 6 months ]
  4. Optional exploratory genomic biomarker analysis [ Time Frame: Week 260 ]
    Exploratory genomic analysis of genes associated with CNP signaling

  5. Effect of BMN 111 on bone morphology and quality [ Time Frame: Through study completion, every 6 months ]
    The effect of BMN 111 on bone morphology will be assessed by measuring bone mineral density via Dual X-ray Absorptiometry



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Must have completed Study 111-301
  • Female >= 10 years old or who have begun menses must have a negative pregnancy test at the Baseline Visit and be willing to have additional pregnancy tests during the study
  • If sexually active, willing to use a highly effective method of contraception while participating in the study
  • Are willing and able to perform all study procedures as physically possible

Exclusion Criteria:

  • Permanently discontinued BMN 111 or placebo prior to completion of the 111-301 study
  • Have a clinically significant finding or arrhythmia on Baseline ECG that indicates abnormal cardiac function
  • Evidence of decreased growth velocity (<1.5 cm/year) or growth plate closure obtained at Baseline Visit
  • Require any investigational agent prior to completion of study period
  • Current therapy with medications known to alter renal function
  • Planned or expected bone/limb surgery during the study period
  • Fracture of the long bones or spine in the 6 months preceding the Baseline Visit
  • Pregnant or breastfeeding or plan to become pregnant during study

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): NCT03424018


Locations
Australia, Victoria
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
Sponsors and Collaborators
BioMarin Pharmaceutical

Additional Information:
Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT03424018     History of Changes
Other Study ID Numbers: 111-302
2017-002404-28 ( EudraCT Number )
First Posted: February 6, 2018    Key Record Dates
Last Update Posted: July 30, 2018
Last Verified: July 2018
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:
Achondroplasia
ACH
Bone Diseases
Bone Diseases, Developmental
Dwarfism
Genetic Diseases, Inborn
Musculoskeletal Diseases
Natriuretic Agents
Natriuretic Peptide, C-Type
Osteochondrodysplasias
Physiological Effects of Drugs
Skeletal Dysplasias

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