A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

• Status: Completed
• Trial end date: 2012-05-01
• Target enrollment: 0
• Participant gender: All

Summary

This study evaluated the long-term safety and efficacy of afegostat tartrate in participants with Gaucher disease who were enrolled in a previous Phase 2 study of afegostat tartrate.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amicus Therapeutics

Criteria

Inclusion Criteria:

• Male or female participants, 18 years of age or older

• Completed study GAU-CL-202 with no significant protocol violations or safety concerns

• Clinically stable

• Had not received enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) in the past 12 months and was willing not to initiate ERT or SRT during study participation

• Agreed to practice an acceptable method of contraception

• Provided written informed consent to participate in the study

Exclusion Criteria:

• During the screening period, had any clinically significant findings which would compromise the safety of the participant, or preclude the participant from completing the study as deemed by the investigator

• Had a clinically significant disease, severe complications from Gaucher disease, or serious intercurrent illness that may preclude participation in the study, in the opinion of the Investigator

• Had a history of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars (for example, miglustat)

• Had a pacemaker or other contraindication for magnetic resonance imaging scanning

• Was pregnant or breast-feeding

• Had current gastrointestinal, liver, or kidney disease, sequelae of these diseases, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs

• Participant was otherwise unsuitable for the study in the opinion of the Investigator