A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This study is ongoing, but not recruiting participants.
Sponsor:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT01275066
First received: January 10, 2011
Last updated: August 6, 2012
Last verified: August 2012
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
MPS IV A |
Drug: BMN 110 Weekly Drug: Placebo Drug: BMN 110 Every Other Week |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | 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) |
Resource links provided by NLM:
Further study details as provided by BioMarin Pharmaceutical:
Primary Outcome Measures:
- Change from baseline in endurance as measured by the 6-minute Walk Test [ Time Frame: Screening, week 12, and week 24 ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Change from baseline in endurance as measured by the 3-minute Stair-Climb Test [ Time Frame: Screening, week 12, and week 24 ] [ Designated as safety issue: No ]
- Change from baseline in urine keratan sulfate (KS) levels (normalized to creatinine) [ Time Frame: Baseline, week 2, 4, 8, 12, 16, 20, and 24 ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 162 |
| Study Start Date: | February 2011 |
| Estimated Primary Completion Date: | August 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Placebo Comparator: Placebo |
Drug: Placebo
Patients will receive an 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.
|
| Experimental: BMN 110 Weekly |
Drug: BMN 110 Weekly
BMN 110 Weekly: Patients will receive an intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week.
Other Names:
|
| Experimental: BMN 110 Every Other Week |
Drug: BMN 110 Every Other Week
BMN 110 Every Other Week: Patients will receive an 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 will receive infusions of placebo on alternating weeks.
Other Names:
|
Eligibility| Ages Eligible for Study: | 5 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01275066
Show 28 Study Locations
Show 28 Study LocationsSponsors and Collaborators
BioMarin Pharmaceutical
Investigators
| Study Director: | Debra Lounsbury | BioMarin Pharmaceutical |
More Information
Additional Information:
No publications provided
| Responsible Party: | BioMarin Pharmaceutical |
| ClinicalTrials.gov Identifier: | NCT01275066 History of Changes |
| Other Study ID Numbers: | MOR-004, 2010-020198-18, 10/H1306/87, 18972/0213/001-0001, 2011_038#B201129, 145240, 2011-01-09, 20110012889, 0999935174 |
| Study First Received: | January 10, 2011 |
| Last Updated: | August 6, 2012 |
| Health Authority: | United States: Food and Drug Administration United States: Institutional Review Board United Kingdom: Medicines and Healthcare Products Regulatory Agency United Kingdom: Research Ethics Committee Netherlands: Medical Ethics Review Committee (METC) France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) France: Conseil National de l'Ordre des Médecins Canada: Health Canada Korea: Food and Drug Administration Taiwan : Food and Drug Administration Taiwan: Department of Health Italy: The Italian Medicines Agency Italy: Ethics Committee Argentina: Ministry of Health Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica Denmark: Ethics Committee Denmark: Danish Medicines Agency Japan: Institutional Review Board Japan: Pharmaceuticals and Medical Devices Agency Portugal: Ethics Committee for Clinical Research Brazil: Ethics Committee Brazil: National Health Surveillance Agency Brazil: National Committee of Ethics in Research Colombia: INVIMA Instituto Nacional de Vigilancia de Medicamentos y Alimentos Colombia: Institutional Review Board Germany: Ethics Commission Germany: Federal Institute for Drugs and Medical Devices Qatar: Hamad Medical Corporation Saudi Arabia: Ethics Committee Saudi Arabia: Saudi Food and Drug Authority |
Keywords provided by BioMarin Pharmaceutical:
|
Mucopolysaccharidosis IV type A MPS IV Type A Mucopolysaccharidosis IVA MPS IVA Morquio A Syndrome Lysosomal Storage Disorder LSD |
N-acetylgalactosamine-6-sulfatase N-acetylgalactosamine-6-sulfate sulfatase galactose-6-sulfatase GALNS enzyme replacement therapy ERT |
Additional relevant MeSH terms:
|
Mucopolysaccharidoses Mucopolysaccharidosis IV Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |
Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases |
ClinicalTrials.gov processed this record on October 18, 2012
