Phase 2
N=8
A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
Gaucher Disease · Type 1 Gaucher Disease · Gaucher Disease, Type 1
Bottom Line
View on ClinicalTrials.gov: NCT00813865 ↗Enrolled (actual)
8
Serious AEs
0.0%
Results posted
Aug 2018
Primary outcome: Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 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
- May 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) |
— | — |
| SECONDARY Change From Baseline To EOT In Volume Of Spleen As Assessed By Magnetic Resonance Imaging (MRI) |
-138 | — |
| SECONDARY Change From Baseline To EOT In Volume Of Liver As Assessed By MRI |
57 | — |
Summary
This study evaluated the long-term safety and efficacy of afegostat tartrate in participants with Gaucher disease who were enrolled in a previous Phase 2 study of afegostat tartrate.
Eligibility Criteria
Inclusion Criteria
- Male or female participants, 18 years of age or older
- Completed study GAU-CL-202 with no significant protocol violations or safety concerns
- Clinically stable
- Had not received enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) in the past 12 months and was willing not to initiate ERT or SRT during study participation
- Agreed to practice an acceptable method of contraception
- Provided written informed consent to participate in the study
Exclusion Criteria
- During the screening period, had any clinically significant findings which would compromise the safety of the participant, or preclude the participant from completing the study as deemed by the investigator
- Had a clinically significant disease, severe complications from Gaucher disease, or serious intercurrent illness that may preclude participation in the study, in the opinion of the Investigator
- Had a history of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars (for example, miglustat)
- Had a pacemaker or other contraindication for magnetic resonance imaging scanning
- Was pregnant or breast-feeding
- Had current gastrointestinal, liver, or kidney disease, sequelae of these diseases, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs
- Participant was otherwise unsuitable for the study in the opinion of the Investigator
Data sourced from ClinicalTrials.gov (NCT00813865). 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.