Mucopolysaccharidosis Type IVA MedDRA version: 16.1 Level: PT Classification code 10028095 Term: Mucopolysaccharidosis IV System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: BMN 110
Product Code: N/A
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Not available
CAS Number: 9025-6
Sponsors
BioMarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • 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. • 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 Baseline and be willing to have additional pregnancy tests during the study. • Must have enrolled in a prior BioMarin sponsored clinical study of BMN 110. Are the trial subjects under 18? yes Number of subjects for this age range: 20 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Pregnant 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 a prior clinical study) or investigational medical device within 30 days prior to Baseline, 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. • Were enrolled in MOR-004 (patients from MOR-004 may be eligible to participate in a separate, appropriately designed, extension study).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the long-term safety and efficacy of weekly infusions of 2.0 mg/kg of BMN 110 administered in patients with MPS IVA.;Secondary Objective: To evaluate the long-term effect of weekly infusions of 2.0 mg/kg of BMN 110 by monitoring 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 radiographs of lumbar spine and lower extremity • MPS Health Assessment Questionnaire • EuroQoL EQ-5D-5L • PRO questionnaires Safety Variables: • adverse events (AEs) • vital signs • echocardiograms • electrocardiograms (ECGs) • cervical spine (flexion–extension) radiographs • routine physical examinations, including standard neurologic examination and evaluation of presence or absence of corneal clouding • standard clinical laboratory tests (serum chemistry, hematology, and urinalysis) • concomitant medications • immunogenicity tests ; Timepoint(s) of evaluation of this end point: • At baseline and weekly: adverse events (AEs), vital signs, concomitant medications • At | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Exploratory Variables: • blood inflammatory biomarkers • biochemical markers of bone and cartilage metabolism ; Timepoint(s) of evaluation of this end point: Exploratory Variables: • At baseline and every 24 weeks: blood inflammatory biomarkers, and biochemical markers of bone and cartilage metabolism | — |
Countries
United Kingdom
Contacts
Public ContactClinical Trials Information
BioMarin Pharmaceutical Inc.
Outcome results
None listed