NCT00433147

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

A Randomized, Open-label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 18 Years – 74 Years
• Healthy volunteers: Not accepted

Summary

This study was conducted to test the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease already receiving enzyme replacement therapy.

Detailed description

This was a Phase 2, open-label study in participants with Gaucher disease, a lysosomal storage disorder. Afegostat tartrate (also known as AT2101 or isofagomine tartrate) is designed to act as a pharmacological chaperone by selectively binding to misfolded β-glucocerebrosidase (GCase) and helping it fold correctly, intended to restore GCase activity. The study consisted of a 14-day screening period, a 28-day treatment period, and a 7-day wash-out period. Participants received 1 of 4 dosing regimens for afegostat tartrate.

Conditions

• Gaucher Disease, Type 1
• Type 1 Gaucher Disease
• Gaucher Disease

Interventions

Type	Name	Description
DRUG	Afegostat tartrate

Timeline

• Start date: 2007-03-23
• Primary completion: 2008-02-19
• Completion: 2008-02-19
• First posted: 2007-02-09
• Last updated: 2018-09-25
• Results posted: 2018-08-15

Locations

10 sites across 1 country: United States