Phase 2
N=19
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
Gaucher Disease · Type 1 Gaucher Disease · Gaucher Disease, Type 1
Bottom Line
View on ClinicalTrials.gov: NCT00446550 ↗Enrolled (actual)
19
Serious AEs
0.0%
Results posted
Aug 2018
Primary outcome: Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 2; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- afegostat tartrate (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Amicus Therapeutics
- Primary completion
- Aug 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) |
2; 0 | — |
| SECONDARY Change From Baseline To End Of Treatment In β-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC) |
10.2; 3.9 | — |
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).
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT00446550). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.