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

NCT00813865 | Completed Phase 2 Results

• 1 other identifier: GAU-CL-202X
• Study Type: interventional
• Target: 8
• Locations: 2 countries
• Sites: 4

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.

Conditions

Gaucher Disease; Type 1 Gaucher Disease; Gaucher Disease, Type 1

Keywords

Amicus Therapeutics; AT2101; afegostat tartrate; isofagomine tartrate

Outcome Measures

Primary Outcomes (1)

• Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)

  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.

  Day 1 (after dosing) through end of follow-up (6 months after EOT)

Secondary Outcomes (2)

• Change From Baseline To EOT In Volume Of Spleen As Assessed By Magnetic Resonance Imaging (MRI)

  Baseline, Month 30

• Change From Baseline To EOT In Volume Of Liver As Assessed By MRI

  Baseline, Month 30

Study Arms (1)

Afegostat Tartrate Treatment Regimen 1

EXPERIMENTAL

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.

Drug: afegostat tartrate

Interventions

afegostat tartrate DRUG

Also known as: AT2101, isofagomine tartrate

Afegostat Tartrate Treatment Regimen 1

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Amicus Therapeutics: lead

Study Sites (4)

Unknown Facility

Coral Springs, Florida, 33065, United States

Location

Unknown Facility

Decatur, Georgia, 30033, United States

Location

Unknown Facility

Boston, Massachusetts, 02114, United States

Location

Unknown Facility

London, United Kingdom

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

AT2101

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Limitations and Caveats

Due to the early withdrawal of 7 of the 8 study participants, there were no comparative data. Accordingly, no definitive conclusions regarding afegostat tartrate safety, efficacy, or pharmacodynamic effects can be made based on this information.

Results Point of Contact

• Title: Amicus Therapeutics
• Organization: Patient Advocacy

Clinicaltrials@amicusrx.com

+1-609-662-2000

Study Officials

• Medical Director

  Amicus Therapeutics, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 22, 2008
• First Posted: December 23, 2008
• Study Start: May 11, 2009
• Primary Completion: May 1, 2012
• Study Completion: May 1, 2012
• Last Updated: August 15, 2018
• Results First Posted: August 15, 2018

Record last verified: 2018-07

Locations

GB (1); US (3)