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Also known as afegostat tartrate, AT2101, isofagomine tartrate
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 Gaucher disease, specifically Type 1 Gaucher disease, a rare inherited lysosomal storage disorder. It has been evaluated in Phase 2 clinical trials, including in treatment-naive patients and in patients already receiving enzyme replacement therapy.
Afegostat targets GBA1, the gene encoding the enzyme glucocerebrosidase. It is classified as a stabiliser, meaning it is designed to bind and stabilise the misfolded enzyme so it can function properly. This mechanism is distinct from enzyme replacement therapy, which supplies the enzyme externally.
Afegostat is being developed by Amicus Therapeutics, Inc., which trades on the Nasdaq under the ticker FOLD. The company is focused on rare diseases, and afegostat is its small molecule candidate for Type 1 Gaucher disease.
Afegostat is in Phase 2 clinical development. It is investigational and has not been approved by the FDA. The Phase 2 program includes completed trials in Type 1 Gaucher disease, and no active trials are currently listed.
Afegostat has been studied in three completed Phase 2 trials: NCT00813865, a long-term extension study; NCT00446550, in treatment-naive patients; and NCT00433147, in adults receiving enzyme replacement therapy. These trials enrolled patients in the United States, United Kingdom, Israel, and South Africa.
Yes, afegostat tartrate is the salt form of afegostat and is the name used in the clinical trial titles, such as AT2101 (afegostat tartrate). The two names refer to the same investigational drug candidate developed by Amicus Therapeutics for Type 1 Gaucher disease.