A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
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ClinicalTrials.gov Identifier: NCT00813865 |
Recruitment Status :
Completed
First Posted : December 23, 2008
Results First Posted : August 15, 2018
Last Update Posted : August 15, 2018
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Condition or disease | Intervention/treatment | Phase |
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Gaucher Disease Type 1 Gaucher Disease Gaucher Disease, Type 1 | Drug: afegostat tartrate | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 8 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-Label, Multicenter, Long-Term Extension Study To Assess The Safety, Efficacy, And Pharmacodynamics Of AT2101 In Adult Patients With Type 1 Gaucher Disease |
Actual Study Start Date : | May 11, 2009 |
Actual Primary Completion Date : | May 1, 2012 |
Actual Study Completion Date : | May 1, 2012 |

Arm | Intervention/treatment |
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Experimental: Afegostat Tartrate Treatment Regimen 1
Afegostat tartrate was administered orally at a dose of 225 mg QD for 3 or 7 consecutive days followed by no study medication for 4 or 7 consecutive days (consecutive 3-days-on/4-days-off or 7-days-on/7-days-off, respectively). Amendment 2 added a MWF 3-days-on/4-days-off regimen. After Amendment 2 was implemented, all participants were assigned to one of the two 3-days-on/4-days-off regimens. Amendment 4 removed the consecutive 3-days-on/4-days-off regimen, and all participants were assigned to the MWF 3-days-on/4-days-off regimen. Participants were to receive afegostat tartrate for 30 months and be followed for 6 months after EOT.
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Drug: afegostat tartrate
Other Names:
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- Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) [ Time Frame: Day 1 (after dosing) through end of follow-up (6 months after EOT) ]A TEAE was defined as any adverse event (AE) with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe AE defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced 1 or more severe TEAEs after dosing on Day 1 through the end of follow-up is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
- Change From Baseline To EOT In Volume Of Spleen As Assessed By Magnetic Resonance Imaging (MRI) [ Time Frame: Baseline, Month 30 ]Standard MRI procedures were used to measure the volume of the spleen. The baseline value was defined as the value recorded on Days 1 to 3 of the study (certain values could have been carried over from the last assessment in the lead-in study, GAU-CL-202). A negative change from Baseline indicates that spleen volume decreased.
- Change From Baseline To EOT In Volume Of Liver As Assessed By MRI [ Time Frame: Baseline, Month 30 ]Standard MRI procedures were used to measure the volume of the liver. The baseline value was defined as the value recorded on Days 1 to 3 of the study (certain values could have been carried over from the last assessment in the lead-in study, GAU-CL-202). A negative change from Baseline indicates that liver volume decreased.

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00813865
United States, Florida | |
Coral Springs, Florida, United States, 33065 | |
United States, Georgia | |
Decatur, Georgia, United States, 30033 | |
United States, Massachusetts | |
Boston, Massachusetts, United States, 02114 | |
United Kingdom | |
London, United Kingdom |
Study Director: | Medical Director | Amicus Therapeutics, Inc. |
Responsible Party: | Amicus Therapeutics |
ClinicalTrials.gov Identifier: | NCT00813865 |
Other Study ID Numbers: |
GAU-CL-202X |
First Posted: | December 23, 2008 Key Record Dates |
Results First Posted: | August 15, 2018 |
Last Update Posted: | August 15, 2018 |
Last Verified: | July 2018 |
Amicus Therapeutics AT2101 afegostat tartrate isofagomine tartrate |
Gaucher Disease Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Metabolism, Inborn Errors Genetic Diseases, Inborn Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |