Status:
COMPLETED
A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
Lead Sponsor:
Amicus Therapeutics
Conditions:
Gaucher Disease
Type 1 Gaucher Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief 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.
Detailed Description
This was an open-label, long-term extension study of afegostat tartrate in participants with type 1 Gaucher disease who successfully completed Study GAU-CL-202 (NCT00446550). Participants could enter ...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
May 11 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2012
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT00813865
Start Date
May 11 2009
End Date
May 1 2012
Last Update
August 15 2018
Active Locations (4)
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1
Coral Springs, Florida, United States, 33065
2
Decatur, Georgia, United States, 30033
3
Boston, Massachusetts, United States, 02114
4
London, United Kingdom