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A 24-Week Safety and Pharmacodynamic Study of AT1001 in Patients With Fabry Disease

2014-08-27 03:46:25 | BioPortfolio

Summary

The purpose of this study is to collect information on the safety of AT1001 (migalastat hydrochloride) and how AT1001 works in patients with Fabry disease.

Description

The purpose of this study is to determine the effect of AT1001 given orally to patients with Fabry disease. Patients will visit the clinic 4 weeks prior to dosing to determine their eligibility for the study, and then return for a second visit for baseline and safety assessments, which will include skin, cardiac, and renal biopsies. Patients will receive oral doses of AT1001 for 24 weeks and will visit the clinic 6 times, once every 4 weeks, for evaluation and tests. A skin biopsy will be repeated after 12 weeks, and then a final set of skin, cardiac, and renal biopsies, and functional assessments will be performed at the end of 24 weeks. Patients may be given the opportunity to enter a study extension phase for an additional 24 weeks, which will require two more clinic visits. All study participants will have a final follow up visit 2 weeks after the end of the study.

Study Design

Allocation: Non-Randomized, Control: Uncontrolled, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Fabry Disease

Intervention

AT1001 (migalastat hydrochloride)

Location

Université de Montréal, Hôpital du Sacré-Coeur de Montréal
Montréal
Quebec
Canada
H4J 1C5

Status

Completed

Source

Amicus Therapeutics

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-08-27T03:46:25-0400

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Medical and Biotech [MESH] Definitions

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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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