This trial has been completed.

Conditions mucopolysaccharidosis iva, morquio a syndrome, mps iva
Treatment bmn 110
Phase phase 2
Sponsor BioMarin Pharmaceutical
Start date October 2011
End date February 2016
Trial size 15 participants
Trial identifier NCT01515956, MOR-007


This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug, diagnosed with MPS IVA (Morquio A Syndrome) for up to 208 weeks.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
bmn 110 N-acetylgalactosamine-6-sulfatase
Patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/wk over a period of approximately 4 hours every week for up to 208 weeks.

Primary Outcomes

Descriptive summary of clinical safety assessments
time frame: Up to 208 weeks/ETV

Secondary Outcomes

Change in Urinary Keratan Sulfate measures over time
time frame: Baseline, and weeks: 2, 4, 8, 13, 26, 39, 52, 78, 104, 130, 156, 182, 208/ETV
Change in patient growth over time
time frame: Baseline and Weeks: 13, 26, 39, 52,78, 104, 130, 156, 182, 208/ETV

Eligibility Criteria

Male or female participants up to 5 years old.

Inclusion Criteria: - Less than 5 years of age at the time of the first study drug infusion - 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 - Written informed consent provided by parent or legally authorized representative after the nature of the study has been explained and prior to any research-related procedures. Exclusion Criteria: - Previous hematopoietic stem cell transplant (HSCT). - Previous treatment with BMN 110. - Known hypersensitivity to any of the components of BMN 110. - Major surgery within 3 months prior to stuy entry or planned major surgery during the 52-week treatment period. - 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.

Additional Information

Official title A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Trial information was received from ClinicalTrials.gov and was last updated in December 2016.
Information provided to ClinicalTrials.gov by BioMarin Pharmaceutical.