Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
afegostat · 3 trials · 3 indications
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. |
| Afegostat tartrate 25 milligrams (mg) once per day | EXPERIMENTAL | Afegostat tartrate was administered orally during the 4-week treatment period. |
| Afegostat tartrate 150 mg once per day | EXPERIMENTAL | Afegostat tartrate was administered orally once per day during the 4-week treatment period. |
| Afegostat tartrate 150 mg once every four days | EXPERIMENTAL | Afegostat tartrate was administered orally once every 4 days during the 4-week treatment period. |
| Afegostat tartrate 150 mg once every seven days | EXPERIMENTAL | Afegostat tartrate was administered orally once every 7 days during the 4-week treatment period. |
| 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 ...