A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

August 28, 2018 updated by: Amicus Therapeutics

A Randomized, Open-label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 pharmacological chaperone by selectively binding to misfolded β-glucocerebrosidase (GCase) and helping it fold correctly, intended to restore GCase activity. The study consisted of a 14-day screening period, a 28-day treatment period, and a 7-day wash-out period. Participants received 1 of 4 dosing regimens for afegostat tartrate.

Study Type

Interventional

Enrollment (Actual)

30

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

  • United States
    • California
      • San Francisco, California, United States, 94143
    • Florida
      • Coral Springs, Florida, United States, 33065
    • Georgia
      • Decatur, Georgia, United States, 30033
    • Iowa
      • Iowa City, Iowa, United States, 52242
    • New York
      • New York, New York, United States, 10016
    • Ohio
      • Cincinnati, Ohio, United States, 45229
    • Oregon
      • Portland, Oregon, United States, 97239
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
      • Pittsburgh, Pennsylvania, United States, 15213
    • Texas
      • Houston, Texas, United States, 77030

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 to 74 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 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 Criteria:

  • 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

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: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Afegostat tartrate 25 milligrams (mg) once per day
Afegostat tartrate was administered orally during the 4-week treatment period.
Drug: Afegostat tartrate
Other Names:
  • isofagomine tartrate
  • AT2101
EXPERIMENTAL: Afegostat tartrate 150 mg once per day
Afegostat tartrate was administered orally once per day during the 4-week treatment period.
Drug: Afegostat tartrate
Other Names:
  • isofagomine tartrate
  • AT2101
EXPERIMENTAL: Afegostat tartrate 150 mg once every four days
Afegostat tartrate was administered orally once every 4 days during the 4-week treatment period.
Drug: Afegostat tartrate
Other Names:
  • isofagomine tartrate
  • AT2101
EXPERIMENTAL: Afegostat tartrate 150 mg once every seven days
Afegostat tartrate was administered orally once every 7 days during the 4-week treatment period.
Drug: Afegostat tartrate
Other Names:
  • isofagomine tartrate
  • AT2101

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
Time Frame: Day 1 (after dosing) through Day 35
TEAEs were defined as any adverse event (AE) with a start date on or after administration of the study drug (on Day 1). A severe AE was 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 7 days after the last dose of study drug (Day 35) 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 Day 35

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline To End Of Treatment In Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)
Time Frame: Baseline, Day 28
GCase is a biomarker used to assess the PD effects of afegostat tartrate. Blood samples were collected to assess GCase levels in WBC. The baseline value was defined as the last non-missing value before the start of study drug.
Baseline, Day 28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Amicus Therapeutics

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)

March 23, 2007

Primary Completion (ACTUAL)

February 19, 2008

Study Completion (ACTUAL)

February 19, 2008

Study Registration Dates

First Submitted

February 7, 2007

First Submitted That Met QC Criteria

February 7, 2007

First Posted (ESTIMATE)

February 9, 2007

Study Record Updates

Last Update Posted (ACTUAL)

September 25, 2018

Last Update Submitted That Met QC Criteria

August 28, 2018

Last Verified

August 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GAU-CL-201

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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