Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00813865 | A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients | PHASE2 | COMPLETED | 8 | — | — | May 11, 2009 | May 1, 2012 | Aug 15, 2018 | 4 | United States, United Kingdom |
| NCT00446550 | A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease | PHASE2 | COMPLETED | 19 | — | — | Jun 11, 2008 | Aug 20, 2009 | Aug 15, 2018 | 11 | United States, Israel +2 |
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 is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
| Arm | Type | Description |
|---|---|---|
| Afegostat Tartrate Treatment Regimen 1 | EXPERIMENTAL | Afegostat tartrate was administered orally at a dose of 225 mg QD for 3 or 7 consecutive days followed by no study medication for 4 or 7 consecutive days (consecutive 3-days-on/4-days-off or 7-days-on/7-days-off, respectively). Amendment 2 added a MWF 3-days-on/4-days-off regimen. After Amendment 2 was implemented, all participants were assigned to one of the two 3-days-on/4-days-off regimens. Amendment 4 removed the consecutive 3-days-on/4-days-off regimen, and all participants were assigned to the MWF 3-days-on/4-days-off regimen. Participants were to receive afegostat tartrate for 30 months and be followed for 6 months after EOT. |
| Afegostat Tartrate Treatment Regimen 2 | EXPERIMENTAL | Afegostat tartrate was administered orally at a dose of 225 mg QD for 7 consecutive days, followed by no study medication for 7 consecutive days. This 7-days-on/7-days-off treatment regimen was followed for 24 weeks. |
| Name | Type | Description |
|---|---|---|
| afegostat tartrate | DRUG | - |
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 ...