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

Completed · Phase 2

Conditions studied: Gaucher Disease, Type 1 Gaucher Disease, Gaucher Disease, Type 1

In brief

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.

Key facts

Study ID

NCT00813865

Run by

Amicus Therapeutics

People needed

8

Starts

2009-05-11

Expected to finish

2012-05-01

Last updated by the study team

2018-08-15

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Study site — Coral Springs, Florida, United States
• Study site — Decatur, Georgia, United States
• Study site — Boston, Massachusetts, United States
• Study site — London, United Kingdom

Full record on ClinicalTrials.gov

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