Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
Condition: Mucopolysaccharidosis IVA; Morquio A Syndrome; MPS IVA
MeSH terms: Mucopolysaccharidoses; Mucopolysaccharidosis IV; Syndrome
Intervention: BMN 110
Intervention description: Drug will be delivered through a 4 hour (approximate) IV infusion at a dosage amount of 2.0 mg/kg/week for up to 144 weeks of treatment.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 16.0
Sex: All
Minimum age: 5 Years
Healthy volunteers: No
Keywords: Mucopolysaccharidosis IVA Type A; MPS IVA 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; MOR-006; CPET; Limited ambulation; Grip/ Pinch

Summary:

      The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a
      patient population that has limited ambulation, in a period of up to 144 weeks.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Is willing and able to provide written, signed informed consent (or their legally
             authorized representative) after the nature of the study has been explained and prior
             to performance of any research-related procedure. Patients who do not meet country and
             local age requirements for informed consent must be willing and able to provide
             written assent after the nature of the study has been explained and prior to
             performance of any research-related procedure.

          -  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 ≥ 5 years of age.

          -  If sexually active, is willing to use an acceptable method of contraception while
             participating in the study.

          -  Females of childbearing potential must have a negative pregnancy test at the Screening
             Visit and be willing to have additional pregnancy tests during the study.

          -  Is willing and able to perform all study procedures as physically possible.

        Exclusion Criteria:

          -  Is able to walk farther than a specified distance as assessed by the 6MWT.

          -  Has previous hematopoietic stem cell transplant (HSCT).

          -  Has received previous treatment with BMN 110.

          -  Has a known hypersensitivity to any of the components of BMN 110.

          -  Has had major surgery within 3 months prior to study entry or is planning to have a
             major surgery during the first 24 weeks of the study.

          -  Has used any other investigational product or investigational medical device within 30
             days prior to the Screening Visit or requires any investigational agent prior to
             completion of all scheduled study assessments.

          -  Is pregnant or breastfeeding at the Screening Visit or planning to become pregnant
             (self or partner) at any time during the study.

          -  Has a concurrent disease or condition, including but not limited to symptomatic
             cervical spine instability or severe cardiac disease or complete paralysis due to a
             spinal cord injury (defined as an inability to move arms and legs), that would
             interfere with study participation or safety 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.