Status:
COMPLETED
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
Lead Sponsor:
Amicus Therapeutics
Conditions:
Gaucher Disease, Type 1
Type 1 Gaucher Disease
Eligibility:
All Genders
18-74 years
Phase:
PHASE2
Brief 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 pharm...
Eligibility Criteria
Inclusion
- Had a confirmed diagnosis of type 1 Gaucher disease with a known documented missense gene mutation in at least 1 of the 2 gene-encoding β-glucosidase alleles
- Clinically stable
- Male or female participants, 18 to 74 years old inclusive
- All participants of childbearing potential used adequate birth control
- Provided written informed consent to participate in the study
Exclusion
- Clinically significant disease, severe complications from Gaucher disease, or serious illness that precluded participation in the study in the opinion of the Investigator that compromised the safety of the participant or precluded the participant from completing the study
- During the screening period, any clinically significant findings, as deemed by the Investigator
- Partial or total splenectomy (removal of spleen) within the 2 years prior to study entry
- History of pulmonary hypertension or Gaucher related lung disease
- History of allergy or sensitivity to the study drug or any excipients, including any prior serious adverse reaction to iminosugars (for example, N-butyldeoxynojirimycin or miglustat)
- Pregnant or breast-feeding
- Current/recent drug or alcohol abuse
- Treatment with any investigational product in the 90 days before study entry
- Treatment in the previous 90 days with any drug known to have a well-defined potential for toxicity to a major organ
- Presence or symptoms of gastrointestinal, liver or kidney disease, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs
Key Trial Info
Start Date :
March 23 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 19 2008
Estimated Enrollment :
30 Patients enrolled
Trial Details
Trial ID
NCT00433147
Start Date
March 23 2007
End Date
February 19 2008
Last Update
September 25 2018
Active Locations (10)
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1
San Francisco, California, United States, 94143
2
Coral Springs, Florida, United States, 33065
3
Decatur, Georgia, United States, 30033
4
Iowa City, Iowa, United States, 52242