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A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

NCT02230566 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2020-07-30

Study results available
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Summary

The Phase 3 study will use a novel randomized, intra-subject placebo-controlled, single crossover design, referred to as Blind Start, to evaluate the safety and efficacy of UX003. The Blind Start is a novel design whereby participants will be randomized to 1 of 4 groups, each representing a different treatment sequence, and will cross over to UX003 at different pre-defined time points in a blinded manner. All groups will receive a minimum of 24 weeks treatment with 4 mg/kg UX003 every other week (QOW).

Conditions

  • MPS 7
  • Sly Syndrome
  • Mucopolysaccharidosis
  • MPS VII

Interventions

DRUG

UX003

UX003 is a sterile concentrate formulation of rhGUS for intravenous infusion

OTHER

Placebo

Placebo consisting of the UX003 formulation buffer (without rhGUS)

Sponsors & Collaborators

Principal Investigators

  • Paul Harmatz, MD · UCSF Benioff Children's Hospital Oakland

  • Raymond Wang, MD · Children's Hospital of Orange County

  • Mislen Bauer, MD · Nicklaus Children's Hospital f/k/a Miami Children's Hospital

  • Chester Whitley, MD · University of Minnesota

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
5 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-12-31
Primary Completion
2016-05-31
Completion
2016-05-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02230566 on ClinicalTrials.gov