- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00813865
A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
July 23, 2018 updated by: Amicus Therapeutics
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
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.
Study Overview
Status
Completed
Intervention / Treatment
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 the study immediately upon completion of participation in the lead-in study GAU-CL-202, or at any later time point.
Participants received 225 milligram (mg) afegostat tartrate, administered orally for 30 months, and remained in 1 of the 2 randomized treatment regimens of Study GAU-CL-202: afegostat tartrate once daily (QD) for 3 consecutive days, then no afegostat tartrate for 4 consecutive days (consecutive 3-days-on/4-days-off) or QD for 7 consecutive days, then no afegostat tartrate for 7 days (consecutive 7-days-on/7-days-off).
Amendment 2 removed the 7-days-on/7-days-off regimen and added an alternative 3-days-on/4-days-off regimen: afegostat tartrate QD on Monday, Wednesday, and Friday, then no afegostat tartrate on Tuesday, Thursday, Saturday, and Sunday (MWF 3-days-on/4-days-off).
Once Amendment 2 was implemented at a site, participants assigned to the 7-days-on/7-days-off regimen switched to a 3-days-on/4-days-off regimen; those on the original 3-days-on/4-days-off regimen could switch to the MWF 3-days-on/4-days-off regimen.
Amendment 4 removed the original 3-days-on/4-days-off regimen and any participants still on that regimen switched to the MWF 3-days-on/4-days-off regimen.
Study visits occurred every 3 months for 30 months.
Participants were contacted approximately 1, 3, and 6 months after the end of treatment (EOT) or early termination for follow-up assessments.
Study Type
Interventional
Enrollment (Actual)
8
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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London, United Kingdom
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Florida
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Coral Springs, Florida, United States, 33065
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Georgia
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Decatur, Georgia, United States, 30033
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Massachusetts
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Boston, Massachusetts, United States, 02114
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / 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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Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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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)
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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.
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Day 1 (after dosing) through end of follow-up (6 months after EOT)
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Change From Baseline To EOT In Volume Of Spleen As Assessed By Magnetic Resonance Imaging (MRI)
Time Frame: Baseline, Month 30
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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.
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Baseline, Month 30
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Change From Baseline To EOT In Volume Of Liver As Assessed By MRI
Time Frame: Baseline, Month 30
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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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Baseline, Month 30
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Medical Director, Amicus Therapeutics, Inc.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
May 11, 2009
Primary Completion (Actual)
May 1, 2012
Study Completion (Actual)
May 1, 2012
Study Registration Dates
First Submitted
December 22, 2008
First Submitted That Met QC Criteria
December 22, 2008
First Posted (Estimate)
December 23, 2008
Study Record Updates
Last Update Posted (Actual)
August 15, 2018
Last Update Submitted That Met QC Criteria
July 23, 2018
Last Verified
July 1, 2018
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Gaucher Disease
Other Study ID Numbers
- GAU-CL-202X
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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