Status:Enrolling by invitation
, MPS IVa
, Mucopolysaccharidosis type IVa
There is currently no accepted, standard treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) with BMN 110, or recombinant human GALNS (rhGALNS), may be a potential new treatment option for MPS IVA patients. BMN 110 is expected to reduce the progressive, pathologic accumulation of keratan sulfate, and improve signs and symptoms of the disease.
The primary objective of the study is:
• To evaluate the long-term safety and efficacy of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA.
The exploratory objectives of the study are:
• To evaluate the long-term effect of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow on changes in biochemical markers of inflammation and bone and cartilage metabolism, in patients with MPS IVA.
• To evaluate patient perception of impairment and improvement at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA.
Principle Investigator: Dave Viskochil
Principle Department: Pediatric Genetics
Co Investigator: Nicola Longo
Co Investigator: Ashley Warnock
Co Investigator: Lorenzo Botto
Local Site Inclusion:
Must be participating at a main site.
Main Site Inclusion:
To be eligible for study participation, a patient must meet all of the following inclusion criteria:
• Must have completed MOR-004.
• Is willing and able to provide written, signed informed consent. Or, in the case of patients under the age of 18 (or other age as defined by regional law or regulation), provide written assent (if required) and have written informed consent, signed by a legally authorized representative, after the nature of the study has been explained, and prior to performance of research-related procedures.
• If sexually active, must be willing to use an acceptable method of contraception while participating in the study.
• If female, of childbearing potential, must have a negative pregnancy test at Baseline and be willing to have additional pregnancy tests done during the study.
If any of the following exclusion criteria apply, a patient will not be eligible to participate in the study:
• Is pregnant or breastfeeding, at Baseline, or planning to become pregnant (self or partner) at any time during the study.
• Has used any investigational product (other than BMN 110 in MOR-004), or investigational medical device, within 30 days prior to Baseline; or is required to use any investigational agent prior to completion of all scheduled study assessments.
• Was enrolled in a previous BMN 110 study, other than MOR-004.
• Has a 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 pose a safety risk, as determined by the Investigator.
• Has 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.