MPS IVA Syndrome of Morquio
Conditions
Interventions
During the double-blind phase of the study, patients will receive intravenous
(IV) infusions of study drug at a dose of 2.0 mg/kg/qw or 2.0 mg/kg/qow.
Patients randomized to the 2.0 mg/kg/qow arm wi
Sponsors
BioMarin
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
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.
Exclusion criteria
Exclusion criteria: - Pregnancy or breastfeeding, at Baseline, or planning to become pregnant (self or partner) at any time during the study. - Use of 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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. Efficacy Variables are assessed by means of endurance tests (6-minute walk (6MW) test and 3-minute stair climb (3MSC) test), urine KS concentration (normalized to creatinine), respiratory function tests, anthropometric measurements (standing height, length, sitting height, and weight) , skeletal radiographs of lumbar spine and lower extremity (lower extremity radiographs are done only for patients * 20 years of age) , MPS Health Assessment Questionnaire and audiometry examinations . The safety variables are assessed by collecting adverse events (AEs), performing standard clinical laboratory tests (serum chemistry, hematology, and urinalysis), assessing vital signs, echocardiograms , electrocardiograms (ECGs) , performing routine physical examinations, including standard neurologic examination , concomitant medications, immunogenicity tests and cervical spine (flexion*extension) radiographs. | — |
Secondary
| Measure | Time frame |
|---|---|
| The exploratory objective of the study is 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. This will be assessed by blood inflammatory biomarkers and blood biochemical markers of bone and cartilage metabolism. | — |
Countries
Netherlands
Outcome results
None listed