Completed
A Randomized, Open-label Study To Assess the Safety and Tolerability of AT2101 in Treatment-naive Adult Patients With Type 1 Gaucher Disease
Led by Amicus Therapeutics · Updated on 2018-08-15
19
Participants Needed
11
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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
You will not qualify if you...
- 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
Trial Site Locations
Total: 11 locations
1
Beverly Hills, California, United States, 90211
Status Unknown
2
Coral Springs, Florida, United States, 33065
Status Unknown
3
Decatur, Georgia, United States, 30033
Status Unknown
4
Iowa City, Iowa, United States, 52242
Status Unknown
5
Kansas City, Kansas, United States, 66160
Status Unknown
6
Boston, Massachusetts, United States, 02114
Status Unknown
7
Cincinnati, Ohio, United States, 45229
Status Unknown
8
Haifa, Israel
Status Unknown
9
Tel Aviv, Israel
Status Unknown
10
Johannesburg, South Africa
Status Unknown
11
London, United Kingdom
Status Unknown
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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