Adults 18 to 74, any sex, with Gaucher Disease or Type 1 Gaucher Disease. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)Primary· Day 1 (after dosing) through Day 183
A TEAE was defined as any adverse event (AE) with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe AE defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced 1 or more severe TEAEs after dosing on Day 1 through the end of follow-up (Day 183) is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Group
Value
95% CI
Afegostat Tartrate Treatment Regimen 1
2
Afegostat Tartrate Treatment Regimen 2
0
Change From Baseline To End Of Treatment In β-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)Secondary· Baseline, Day 169
GCase is a biomarker used to assess the PD effects of afegostat tartrate. Blood samples were collected to assess GCase levels in WBC. The baseline value was defined as the last non-missing value before the start of study drug.
Group
Value
95% CI
Afegostat Tartrate Treatment Regimen 1
10.2
± 15.87
Afegostat Tartrate Treatment Regimen 2
3.9
± 4.12
Adverse events — posted to ClinicalTrials.gov
Time frame: Day 1 (after dosing) to Day 183.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
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).
Publications & conference data
6 peer-reviewed publications reference this trial (live from Europe PMC):
NCT00813865 — A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
· Phase 2
· completed
Other recruiting trials for Gaucher Disease
Currently open trials in the same condition.
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· EARLY_PHASE1
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NCT06539169 — FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
· recruiting
NCT05843552 — Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
· recruiting
NCT05487599 — A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
· Phase 1, PHASE2
· recruiting
Other Amicus Therapeutics trials
Trials by the same sponsor.
NCT06121011 — A Global Prospective Observational Registry of Patients With Pompe Disease
· recruiting
NCT04804566 — Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients
· completed
NCT04020055 — A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
· Phase 3
· active not recruiting
NCT04281537 — A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme R
· completed
NCT04138277 — A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LO
· Phase 3
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Amicus Therapeutics
Last refreshed: 15 August 2018
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00446550.