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SGT-501 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Cohort 1 (≥ 18 years of age) | EXPERIMENTAL | Participants will receive a single intravenous (IV) infusion (dose 1) of SGT-501. |
| Cohort 2 (≥ 18 years of age) | EXPERIMENTAL | Participants will receive a single IV infusion (dose 2) of SGT-501. |
| Cohort 3 (≥ 7 to < 18 years) | EXPERIMENTAL | Participants will receive a single IV infusion (level at or below dose(s) assessed in adults) of SGT-501. |
| Name | Type | Description |
|---|---|---|
| SGT-501 | DRUG | IV for infusion |
Inclusion Criteria: Type of Participant and Disease Characteristics: * Clinical diagnosis of CPVT, based on documented history of polymorphic or bidirectional non-sustained ventricular tachycardia with exercise or ventricular ectopy in a pattern consistent with CPVT on EST. * Central Screening lab...
SGT-501 is an investigational small molecule being developed for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), a rare inherited heart rhythm disorder. It is designed to address the underlying genetic cause of the condition. The drug is currently in Phase 1 clinical development and has not yet been approved by the FDA.
SGT-501 targets the CASQ2 protein, which is involved in calcium handling within heart muscle cells. Mutations in the CASQ2 gene can lead to CPVT, a condition characterized by abnormal heart rhythms. By targeting this protein, SGT-501 aims to correct the dysfunction that contributes to the disease.
SGT-501 is being developed by Solid Biosciences Inc. (NASDAQ: SLDB), a biopharmaceutical company focused on genetic medicines. The company is conducting clinical trials to evaluate the safety and efficacy of SGT-501 in patients with Catecholaminergic Polymorphic Ventricular Tachycardia.
SGT-501 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not yet received FDA approval. The drug has been granted Fast Track designation by the FDA, which is intended to expedite the development and review of therapies for serious conditions with unmet medical needs.
SGT-501 is being studied in a Phase 1 clinical trial with the identifier NCT07148089. This trial is recruiting participants with Catecholaminergic Polymorphic Ventricular Tachycardia and is being conducted in the United States and Canada. The study is designed to enroll 18 patients aged 7 years and older.
Although SGT-501 is described as a small molecule, the ongoing clinical trial for SGT-501 is titled 'A Study of SGT-501 Gene Therapy in Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)'. The trial is actively recruiting and is designed to evaluate the drug's effects in patients with this condition.