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MPS VII clinical trials

View clinical trials related to MPS VII.

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NCT ID: NCT04532047 Recruiting - Wolman Disease Clinical Trials

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

IUERT
Start date: July 1, 2021
Phase: Phase 1
Study type: Interventional

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

NCT ID: NCT03775174 Available - Clinical trials for Mucopolysaccharidosis VII

Expanded Access to Mepsevii

Start date: n/a
Phase:
Study type: Expanded Access

Individual patient expanded access requests may be considered for patients who have no other treatment options

NCT ID: NCT03604835 Recruiting - Clinical trials for Mucopolysaccharidosis VII

Mucopolysaccharidosis VII Disease Monitoring Program

Start date: January 29, 2018
Phase:
Study type: Observational

The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.

NCT ID: NCT02432144 Completed - Clinical trials for Mucopolysaccharidosis

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

Start date: November 10, 2015
Phase: Phase 3
Study type: Interventional

The primary objective of the study is to evaluate the long-term safety of UX003 in subjects with MPS 7.

NCT ID: NCT02418455 Completed - Clinical trials for Mucopolysaccharidosis

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

Start date: July 21, 2015
Phase: Phase 2
Study type: Interventional

The primary objective was to evaluate the effect of UX003 treatment in pediatric MPS VII participants less than 5 years of age on safety, tolerability, and efficacy as determined by the reduction of urinary glycosaminoglycans (uGAG) excretion.

NCT ID: NCT02230566 Completed - Clinical trials for Mucopolysaccharidosis

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

Start date: December 2014
Phase: Phase 3
Study type: Interventional

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).