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 ...
Afegostat is an investigational small molecule being studied for the treatment of Gaucher disease, including Type 1 Gaucher disease. It is being developed by Amicus Therapeutics, Inc. (NASDAQ: FOLD) and is currently in Phase 2 clinical development.
Afegostat is a small molecule being studied as a potential treatment for Gaucher disease. It is being evaluated in patients with Type 1 Gaucher disease, including those currently receiving enzyme replacement therapy and treatment-naive patients.
Afegostat is being developed by Amicus Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol FOLD. The drug is currently in Phase 2 clinical trials for the treatment of Gaucher disease.
Afegostat is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Clinical trials have been completed in patients with Type 1 Gaucher disease.
Afegostat has been studied in three completed Phase 2 clinical trials. These include NCT00433147 in adult patients with Type 1 Gaucher disease receiving enzyme replacement therapy, NCT00446550 in treatment-naive patients, and NCT00813865, a long-term extension study.
Yes, Afegostat is also known as AT2101, and in clinical trials it is referred to as afegostat tartrate. The drug has been studied under these names in Phase 2 trials for Gaucher disease.