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

Sponsored by Amicus Therapeutics

About this trial

Last updated 7 years ago

Study ID

GAU-CL-202X

Status

Completed

Type

Interventional

Phase

Phase 2

Placebo

No

Accepting

18-75 Years
18+ Years
All
All

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Ended 13 years ago

What is this trial about?

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.

What are the participation requirements?

Yes

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

No

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

Locations

Location

Status