Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Condition: Mucopolysaccharidosis IV A; Morquio A Syndrome; MPS IVA
MeSH terms: Osteochondrodysplasias; Mucopolysaccharidoses; Mucopolysaccharidosis IV; Syndrome
Intervention: BMN 110 - Weekly; BMN 110 - Every Other Week
Intervention description: In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/qw administered over a period of approximately 4 hours once a week.
In Part 2, patients will continue to receive 2.0 mg/kg of BMN 110 every week, with no placebo.; In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week. Patients randomized to the 2.0 mg/kg/qow arm will receive infusions of placebo on alternating weeks, to mask active drug weeks.
In Part 2, patients will receive 2.0 mg/kg of BMN 110 every week, with no placebo.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 173.0
Sex: All
Minimum age: 5 Years
Healthy volunteers: No
Keywords: Mucopolysaccharidosis IV type A; MPS IV Type A; Mucopolysaccharidosis IVA; MPS IVA; Morquio A Syndrome; Lysosomal Storage Disorder; LSD; N-acetylgalactosamine-6-sulfatase; N-acetylgalactosamine-6-sulfate sulfatase; galactose-6-sulfatase; GALNS; enzyme replacement therapy; ERT

Summary:

      This Phase 3 extension study will evaluate the long-term efficacy and safety of BMN 110 2.0
      mg/kg/week and/or BMN 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis
      IVA (Morquio A Syndrome).
    

Eligibility 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 authorize 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:

          -  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.