Syndrome of Morquio
Conditions
Interventions
Patients randomized to active treatment will receive intravenous (IV) infusions
of BMN 110 at a dose of 2.0 mg/kg/week or infusions of BMN 110 at a dose of 2.0
mg/kg/qow. Patients randomized to the
Sponsors
BioMarin
Eligibility
Age
2 Years to 99 Years
Inclusion 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 have an average screening 6MW test distance * 30 and * 325 meters. * 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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint of this trial is to evaluate the ability of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/qow BMN 110 compared with placebo to enhance endurance in patients with MPS IVA, as measured by an increase in the number of meters walked in the 6-minute walk (6MW) test from baseline to Week 24. | — |
Secondary
| Measure | Time frame |
|---|---|
| The secondary objectives of the study are: * To evaluate the ability of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/qow BMN 110 compared with placebo to enhance endurance in patients with MPS IVA, as measured by an increase in the number of stairs climbed per minute in the 3MSC test from baseline to Week 24. * To evaluate the ability of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/qow BMN 110 compared with placebo to reduce urine KS levels in patients with MPS IVA, as measured by a decrease in urine KS levels from baseline to Week 24. | — |
Countries
Netherlands
Outcome results
None listed