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Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

2014-08-27 03:21:18 | BioPortfolio

Summary

The purpose of this study is to compare the effect of AT1001 (migalastat hydrochloride) versus placebo on kidney GL-3.

Description

This double-blind, randomized, placebo-controlled study will be conducted in 60 patients at approximately 40 sites worldwide. The study will consist of two stages and an open-label treatment extension phase:

Stage 1 includes a screening period of up to 2 months followed by a 6-month treatment period which will involve 4 visits to the clinic. Patients will be randomized in equal proportions to receive either AT1001 or placebo.

After completing the 6-month double-blind phase, all patients will enter Stage 2 of the study and receive AT1001 in an open-label manner. Stage 2 treatment will last for 6 months and will involve 4 visits to the clinic.

Subjects who complete both Stage 1 and Stage 2 of the study as scheduled may be offered the opportunity to participate in a an open-label treatment extension phase with AT1001. The open-label treatment extension phase will last 13 months and will involve 3 visits to the clinic.

Study assessments will include clinical laboratory tests, 12-lead ECG, kidney biopsy, kidney function testing, echocardiography, and patient reported outcomes.

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Conditions

Fabry Disease

Intervention

migalastat hydrochloride, Placebo

Location

Cedars-Sinai Medical Center
Los Angeles
California
United States
90048

Status

Active, not recruiting

Source

Amicus Therapeutics

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:21:18-0400

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The purpose of this study is to determine whether AT1001 (migalastat hydrochloride) is safe and effective in female patients with Fabry disease.

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The purpose of this study is to determine whether AT1001 (migalastat hydrochloride) is safe and effective in patients with Fabry disease.

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PubMed Articles [15513 Associated PubMed Articles listed on BioPortfolio]

Migalastat: A Review in Fabry Disease.

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New insights from the application of the FAbry STabilization indEX in a large population of Fabry cases.

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Medical and Biotech [MESH] Definitions

An X-linked inherited metabolic disease caused by a deficiency of lysosomal ALPHA-GALACTOSIDASE A. It is characterized by intralysosomal accumulation of globotriaosylceramide and other GLYCOSPHINGOLIPIDS in blood vessels throughout the body leading to multi-system complications including renal, cardiac, cerebrovascular, and skin disorders.

Members of the class of neutral glycosphingolipids. They are the basic units of SPHINGOLIPIDS. They are sphingoids attached via their amino groups to a long chain fatty acyl group. They abnormally accumulate in FABRY DISEASE.

Glycosphingolipids which contain as their polar head group a trisaccharide (galactose-galactose-glucose) moiety bound in glycosidic linkage to the hydroxyl group of ceramide. Their accumulation in tissue, due to a defect in ceramide trihexosidase, is the cause of angiokeratoma corporis diffusum (FABRY DISEASE).

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