MOR-005: BMN 110 Extension Study in Patients with Morquio A Syndrome

• Research type

  Research Study

• Full title

  A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)

• IRAS ID

  79814

• Contact name

  Christian Hendriksz

• Sponsor organisation

  BioMarin Pharmaceutical Inc.

• Eudract number

  2010-020199-45

• ISRCTN Number

  0000000000000

• Clinicaltrials.gov Identifier

  0000000000000

• Research summary

  Mucopolysaccharidosis IVA (Morquio A syndrome, MPS IVA) is an inherited autosomal recessive disorder characterized by deficient activity of N-acetylgalactosamine-6-sulfatase (GALNS), resulting in macroscopic accumulation of the glycosaminoglycan (GAG) keratan sulfate (KS) in tissue macrophages, hyaline cartilage and other connective tissues, heart valve, and cornea as well as excretion in the urine. Excessive KS accumulation manifests clinically in multiple ways, including: reduced functional capacity and physical endurance, and hence impaired quality of life.This is a multicenter, multinational, extension study to evaluate 2 dose regimens of BMN 110 treatment in patients with MPS IVA who completed study MOR-004. Initially, the study will be double blind with patients previously randomized to BMN 110 in MOR-004 remaining on their assigned BMN 110 dose regimen (every week or every other week dosing). The MOR-004 placebo patients will be re-randomized (1:1 ratio) to one of the 2 BMN 110 dose regimen groups: 2.0mg/kg/week or 2.0 mg/kg/every other weekwith alternating weeks of placebo.There will be two study parts:?½ Part 1: randomized double blind until the optimal BMN 110 dose regimen has been determined, based on the final primary efficacy analysis from MOR-004?½ Part 2: open label BMN 110 treatment with the single optimal dose regimen Long-term safety and efficacy will be assessed in this study. The study will continue for up to 240 weeks or until one of the following occurs: the patient withdraws consent and discontinues from the study, the patient is discontinued from the study at the discretion of the Investigator or BioMarin, or the study is terminated.Patients will return to the study centre each week to receive infusions of BMN 110 (or every other week in Part 2 of the study depending on the optimal dosing regimen, as determined from the MOR-004 final primary efficacy analysis).

• REC name

  Wales REC 3

• REC reference

  11/WA/0119

• Date of REC Opinion

  7 Jun 2011

• REC opinion

  Further Information Favourable Opinion