Mucopolysaccharidosis Type IVA MedDRA version: 16.0 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: 9025-60-9
Current Sponsor code: rhGALNS
Other descriptive name: recomb
Sponsors
BioMarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Has 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 • Is at least 7 years of age • Is able to walk = 200 meters as assessed by the 6MWT • If sexually active, is willing to use an acceptable method of contraception while participating in the study. For purposes of this study, hormonal contraception is considered to be one of the choices of an acceptable method of contraception, but only if, in consultation with his or her physician, the patient has been using or has indicated a preference for the use of this method prior to seeking enrollment in the study. • If female of childbearing potential, must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study (Refer to the Study Protocol for a complete list of inclusion criteria) Are the trial subjects under 18? yes Number of subjects for this age range: 22 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 3 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Inability to perform an exercise test due to limited mobility • Body weight greater than 95 kg at screening • Severe, untreated sleep apnea as measured during screening with a home sleep testing device • Requirement for supplemental oxygen • Use of ventilator assistance in the 3 months prior to study entry • 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 previous hematopoietic stem cell transplant • Has received previous treatment with BMN 110 • Has a known hypersensitivity to BMN 110 or its excipients • Has had major surgery within 3 months prior to study entry or is planning to have a major surgery during the duration of the study • Is pregnant or breastfeeding during the Screening Period or planning to become pregnant (self or partner) at any time during the study. • Has clinically significant bronchial asthma. (Refer to the Study Protocol for a complete list of exclusion criteria)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is: • To evaluate the safety of 2.0 and 4.0 mg/kg/week BMN 110 administered for 27 weeks The primary objective for extending the Phase 2 study is: • To evaluate the long-term safety of 2.0 and 4.0 mg/kg/week BMN 110 in patients with MPS IVA;Secondary Objective: • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 24 weeks to enhance endurance • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 25 weeks on overall exercise capacity (VO2 max) • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 24 weeks on respiratory function tests (RFTs) • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 25 weeks on muscle strength • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 24 weeks on cardiac function • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 24 weeks on pain • To evaluate the effect of 2.0 and 4.0 mg/kg/week BMN 110 administered for 24 weeks on plasma and urinary KS levels • To determine the pharmacokinetic (PK) parameters of 2.0 mg/kg/week and 4.0 mg/kg/week BMN 110 The secondary objectives of the extension phase of the study are described on page 8 of the revised protocol.;Primary end point(s): Initial Treatment Phase: Evaluate the safety of 2.0 and 4.0 mg/kg/week BMN 110 administered for 27 weeks Extension Phase 2: To evaluate the long-term safety of 2.0 mg/kg/week BMN 110 in patients with MPS IVA;Timepoint(s) of evaluation of this end point: Assessed at Screening and each visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Evaluate the effect of 2.0 mg/kg/week BMN 110 administered for 24 weeks to enhance endurance • Evaluate the effect of 2.0 mg/kg/week BMN 110 administered for 24 weeks on respiratory function tests (RFTs) • Evaluate the effect of 2.0 mg/kg/week BMN 110 administered for 24 weeks on urinary KS levels • Determine the pharmacokinetic (PK) parameters of 2.0 mg/kg/week and 4.0 mg/kg/week BMN 110;Timepoint(s) of evaluation of this end point: • Enhance endurance: Assessed at Screening, Weeks 12, 24, 52, every 52 weeks. • Respiratory function tests (RFTs): Assessed at Screening, Weeks 12, 24, 52, every 52 weeks. • Urinary KS levels: Assessed at Screening, Weeks 1, 2, 4, 6, 12, 24, 52, every 26 weeks. • Pharmacokinetic (PK) parameters of BMN 110 Assessed at Weeks 0 and 23 | — |
Countries
Canada, Germany, United Kingdom, United States
Contacts
Public ContactClinical Trials Information
BioMarin Pharmaceutical Inc.
Outcome results
None listed