Table 1.
Summary of ongoing and recently completed clinical trials for AS. This table provides an overview of interventional studies currently evaluating or recently completing evaluation of investigational therapies in AS, including mechanism of action, target population, and study status
| Study | Drug | Mechanism | Population | Status | Sponsor |
|---|---|---|---|---|---|
| EPPIK | Sparsentan | Dual ETA/AT1 receptor blocker |
Pediatric (1–18yrs) |
Completed (Phase 2) |
Travere Therapeutics |
| ALPESTRIA-1 | Vonafexor | FXR agonist |
Adolescents and adults |
Completed (Phase 2) |
Genfit |
| ELX-02 | ELX-02 | Read-through compound (COL4 mutations) |
Pediatric (6–30 yrs) |
Ongoing (Phase 2) |
Eloxx Pharmaceuticals |
| FIONA | Finerenone | Non-steroidal MRA |
Pediatric (2–18 yrs) |
Recruiting (Phase 3) |
Bayer |
| R3R01-201 | R3R01 | Mediates cholesterol efflux by ABCA1 induction | Adults |
Ongoing (Phase 2) |
Chinook Therapeutics |
| VAR200-0301 | 2HPβCD | Mediates cholesterol efflux | Adults |
Ongoing (Phase 2) |
ZyVersa |
| MSC Trial | hUC-MSC | Stem cell therapy (anti-inflammation) | Not specified |
Planned (Phase 2) |
Guangzhou Women& Children’s |
| Setanaxib Study | Setanaxib | NOX1/4 inhibitor (anti-fibrotic) | Adults |
Completed (Phase 2) |
Calliditas Therapeutics |
| AFFINITY Study | Atrasentan | Selective ETA receptor antagonist | Adults |
Exploratory (Phase 2) |
Chinook Therapeutics |
| CARDINAL | Bardoxolone methyl | Nrf2 activator | Adolescents and adults |
Completed, negative (Phase 3) |
Reata Pharmaceuticals |
| EAGLE | Bardoxolone methyl | Nrf2 activator (long-term safety) | Adults |
Terminated (Phase 3) |
Reata Pharmaceuticals |
| HERA | Lademirsen (anti-miR-21) | microRNA inhibitor | Adolescents and adults | Terminated for futility | Sanofi |
| DOUBLE PRO-TECT Alport | Dapagliflozin | SGLT2 inhibitor | Adolescents and young adults | Recruiting | AstraZeneca |