A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
9 other identifiers
interventional
177
17 countries
28
Brief Summary
This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome). There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) may be a potential new treatment option for MPS IVA patients. BMN 110 is administered to MPS IVA patients by IV infusion, allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes. This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate (KS) in tissue macrophages, hyaline cartilage, other connective tissues, and heart valve, and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Feb 2011
28 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 10, 2011
CompletedFirst Posted
Study publicly available on registry
January 12, 2011
CompletedStudy Start
First participant enrolled
February 1, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2012
CompletedResults Posted
Study results publicly available
April 15, 2014
CompletedJuly 7, 2014
June 1, 2014
1.5 years
January 10, 2011
March 11, 2014
June 6, 2014
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline in Endurance as Measured by the 6-minute Walk Test
Baseline to Week 24
Secondary Outcomes (2)
Change From Baseline in Endurance as Measured by the 3-minute Stair Climb Test
Baseline to Week 24
Percent Change From Baseline in Urine Keratan Sulfate Normalized for Urine Creatinine
Baseline to Week 24
Study Arms (3)
Placebo
PLACEBO COMPARATORBMN 110 Weekly
EXPERIMENTALBMN 110 Every Other Week
EXPERIMENTALInterventions
BMN 110 Weekly: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week.
BMN 110 Every Other Week: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks.
Eligibility Criteria
You may qualify if:
- At least 5 years of age.
- Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA.
- Willing and able to provide written, signed informed consent, or in the case of patients under the age of 18 (or 16 years, depending on the region), provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
- Must meet the study entrance requirements for the 6-minute walk test.
- Sexually active patients must be willing to use an acceptable method of contraception while participating in the study.
- Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study.
You may not qualify if:
- Previous hematopoietic stem cell transplant (HSCT).
- Previous treatment with BMN 110.
- Has known hypersensitivity to any of the components of BMN 110.
- Major surgery within 3 months prior to study entry or planned major surgery during the 24-week treatment period.
- Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
- Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments.
- Concurrent disease or condition, including but not limited to symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation or safety as determined by the Investigator.
- Any condition that, in the view of the Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (28)
Unknown Facility
Oakland, California, United States
Unknown Facility
Wilmington, Delaware, United States
Unknown Facility
Washington D.C., District of Columbia, United States
Unknown Facility
Chicago, Illinois, United States
Unknown Facility
New York, New York, United States
Unknown Facility
Seattle, Washington, United States
Unknown Facility
Córdoba, Argentina
Unknown Facility
Campina Grande, Brazil
Unknown Facility
Porto Alegre, Brazil
Unknown Facility
Montreal, Canada
Unknown Facility
Sherbrooke, Canada
Unknown Facility
Toronto, Canada
Unknown Facility
Bogotá, Colombia
Unknown Facility
Copenhagen, Denmark
Unknown Facility
Lyon, France
Unknown Facility
Paris, France
Unknown Facility
Mainz, Germany
Unknown Facility
Monza, Italy
Unknown Facility
Tokyo, Japan
Unknown Facility
Amsterdam, Netherlands
Unknown Facility
Coimbra, Portugal
Unknown Facility
Doha, Qatar
Unknown Facility
Riyadh, Saudi Arabia
Unknown Facility
Seoul, South Korea
Unknown Facility
Taipei, Taiwan
Unknown Facility
Birmingham, United Kingdom
Unknown Facility
London, United Kingdom
Unknown Facility
Manchester, United Kingdom
Related Publications (1)
Schweighardt B, Tompkins T, Lau K, Jesaitis L, Qi Y, Musson DG, Farmer P, Haller C, Shaywitz AJ, Yang K, O'Neill CA. Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial. Clin Ther. 2015 May 1;37(5):1012-1021.e6. doi: 10.1016/j.clinthera.2014.11.005. Epub 2014 Dec 6.
PMID: 25487082DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- BioMarin Medical Information Services
- Organization
- BioMarin Pharmaceutical Inc.
Study Officials
- STUDY DIRECTOR
Debra Lounsbury
BioMarin Pharmaceutical
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 10, 2011
First Posted
January 12, 2011
Study Start
February 1, 2011
Primary Completion
August 1, 2012
Study Completion
August 1, 2012
Last Updated
July 7, 2014
Results First Posted
April 15, 2014
Record last verified: 2014-06