Clinical studies in MPS VII

UX003 rhGUS enzyme replacement therapy

Recombinant human β-glucuronidase (rhGUS) is an investigational enzyme replacement therapy (ERT) in studies of patients with MPS VII.

rhGUS Phase 3 study

This is a study to establish the safety and efficacy of rhGUS in patients with MPS VII.

Study details:

  • Phase 3
  • Randomized, placebo-controlled, blind-start, single-crossover design
  • Patients between 5 and 35 years of age
  • 48-week treatment period duration

For more information, visit https://clinicaltrials.gov/ct2/show/NCT02230566.

rhGUS Phase 2 <5 study

This is a study to establish the safety and efficacy of rhGUS in children with MPS VII, including infants with non-immune hydrops fetalis (NIHF).

Study details:

  • Phase 2
  • Multicenter
  • Open-label design
  • Children under 5 years of age, including infants with NIHF
  • 48-week treatment period duration

For more information, visit https://clinicaltrials.gov/ct2/show/NCT02418455.

Open-label study
ONGOING >
Open-label study in patients
>5 years of age
ENROLLING >
Open-label study
ONGOING >
Extension study
ONGOING >
A Disease Monitoring Program is planned for patients with MPS VII.
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