Mucopolysaccharidosis Type IVA MedDRA version: 14.1 Level: PT Classification code 10028095 Term: Mucopolysaccharidosis IV System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)
Product Code: BMN 110
Pharmaceutical Form: Solution for infusion
INN or Prop
Sponsors
BioMarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
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. Are the trial subjects under 18? yes Number of subjects for this age range: 110 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 50 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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.;Secondary Objective: 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.; Primary end point(s): Efficacy Variables: • endurance tests: - 6 minute walk (6MW) test - 3 minute stair climb (3MSC) test • urine KS concentration (normalized to creatinine) • respiratory function tests: - forced expiratory time (FET) - forced expiratory volume in 1 second (FEV1) - forced inspiratory vital capacity (FIVC) - forced vital capacity (FVC) - maximum voluntary ventilation (MVV) • anthropometric measurements (standing height, length, sitting height, and weight) • skeletal X-rays of lumbar spine and lower extremity • MPS Health Assessment Questionnaire • audiometry examinations Safety Variables: • adverse events (AEs) • standard clinical laboratory tests (serum chemistry, hematology, and urinalysis) • vital signs • echocardiograms • electrocardiograms (ECGs) • routine physical examinations (including standard neurologic examination) • concomitant medications • immunogenicity tests • cervical spine (flexion–extension) X-rays ; Timepoint(s) of evaluation of this end point: • 6MW test, 3MSC test: baseline, wk 12, every 24 wks in Part 1, every 48 wks in Part 2 • respiratory function tests: baseline, every 24 wks in Part 1, every 48 wks in Part 2 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Exploratory Variables: • blood inflammatory biomarkers • blood biochemical markers of bone and cartilage metabolism ; Timepoint(s) of evaluation of this end point: Exploratory Variables: • blood inflammatory biomarkers, and blood biochemical markers of bone and cartilage metabolism: baseline, every 24 wks in Part 1 and Part 2 •To evaluate patient perception of impairment and improvement in patients with MPS IVA. | — |
Countries
Argentina, Australia, Brazil, Canada, Colombia, Denmark, France, Germany, Italy, Japan, Korea, Republic of, Netherlands, Norway, Poland, Portugal, Qatar, Saudi Arabia, Spain, Switzerland, Taiwan, United Kingdom, United States
Contacts
Public ContactClinical Trials Information
BioMarin Pharmaceutical Inc.
Outcome results
None listed