A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease

• Status: Completed
• Trial end date: 2009-08-20
• Target enrollment: 0
• Participant gender: All

Summary

This study evaluated the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease who were not receiving enzyme replacement therapy (ERT) or substrate reduction therapy (SRT).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amicus Therapeutics

Criteria

Inclusion Criteria:

• Confirmed diagnosis of type 1 Gaucher disease with a known genotype and a documented missense gene mutation in at least 1 of the 2 gene-encoding β-glucosidase (GBA) alleles

• Clinically stable

• Treatment naïve to ERT and SRT or had not received ERT or SRT in the 12 months before screening

• Willing to not initiate ERT or SRT treatment during study participation

• Male or female participants, 18 to 74 years old, inclusive

• At the screening period (Day -21 to Day -1), participants must have met at least 2 of the following criteria: platelet count of ≤150,000 per microliter, hemoglobin ≤12 grams/deciliter (g/dL) for females and ≤13 g/dL for males, liver volume ≥1.25 multiples of normal (MN), and spleen volume ≥2 MN

• All participants of reproductive potential were required to practice an acceptable method of contraception

• Provided written informed consent to participate in the study

Exclusion Criteria:

• A clinically significant disease other than Gaucher disease, severe complications from Gaucher disease, or serious intercurrent illness that precluded participation in the study in the opinion of the investigator

• During the screening period, had any clinically significant findings as deemed by the investigator

• Partial or total splenectomy

• Documentation of moderate or severe pulmonary hypertension, defined as pulmonary arterial pressure >35 millimeters of mercury (mmHg) or significant Gaucher-related lung disease

• History of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars

• Pacemaker or other contraindication for magnetic resonance imaging (MRI) scanning

• Pregnant or breast-feeding

• Current/recent drug or alcohol abuse

• Treatment with any investigational product in the last 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 of symptoms of gastrointestinal, liver or kidney disease, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs