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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00446550
• Date of registration: 09/03/2007
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• Public title: A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
• Scientific title: A Randomized, Open-label Study To Assess the Safety and Tolerability of AT2101 in Treatment-naive Adult Patients With Type 1 Gaucher Disease
• Date of first enrolment: June 11, 2008
• Target sample size: 19
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00446550
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Germany, Israel, South Africa, United Kingdom, United States

Contacts

• Name: Medical Monitor, Clinical Research
• Affiliation: Amicus Therapeutics

Key inclusion & exclusion 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 ß-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



Age minimum: 18 Years
Age maximum: 74 Years
Gender: All

Health Condition(s) or Problem(s) studied

Type 1 Gaucher Disease

Gaucher Disease, Type 1

Gaucher Disease

Intervention(s)

Drug: afegostat tartrate

Primary Outcome(s)

Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) [Time Frame: Day 1 (after dosing) through Day 183]

Secondary Outcome(s)

Change From Baseline To End Of Treatment In ß-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC) [Time Frame: Baseline, Day 169]

Secondary ID(s)

GAU-CL-202

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 15/08/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00446550