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VX-407 · 5 trials · 1 indication
| Arm | Type | Description |
|---|---|---|
| VX-407 | EXPERIMENTAL | Participants will receive VX-407 for up to 52 weeks. |
| Part A: VX-407 With Levonorgestrel/Ethinyl Estradiol (LNG/EE) | EXPERIMENTAL | Participants will receive a single dose of LNG/EE on Days 1 and 21 in fasted state. Participants will also receive VX-407 every 12 hours (q12h) from Days 8 through 26 in fasted state. |
| Part B (Optional): VX-407 With Norgestimate/Ethinyl Estradiol (NGM/EE) | EXPERIMENTAL | Participants will receive a single dose of NGM/EE on Days 1 and 23 in fasted state. Participants will also receive VX-407 q12h from Days 10 through 30 in fasted state. |
| Part C (Optional): VX-407 With Norethindrone/Ethinyl Estradiol (NET/EE) | EXPERIMENTAL | Participants will receive a single dose of NET/EE on Days 1 and 19 in fasted state. Participants will also receive VX-407 q12h from Days 6 through 22 in fasted state. |
| Part D (Optional): VX-407 With Drospirenone/Ethinyl Estradiol (DRSP/EE) | EXPERIMENTAL | Participants will receive a single dose of DRSP/EE on Days 1 and 21 in fasted state. Participants will also receive VX-407 q12h from Days 8 through 26 in fasted state. |
| Part A: Single Ascending Dose | EXPERIMENTAL | Participants will be randomized to receive a single dose of VX-407. |
| Placebo Part A | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-407. |
| Part B: Multiple Ascending Dose | EXPERIMENTAL | Participants will be randomized to receive multiple doses of VX-407. |
| Placebo Part B | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-407. |
| Part A: Single Ascending Dose (SAD) | EXPERIMENTAL | Participants will be randomized to receive a single dose of different dose levels of VX-407. |
| Part A: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-407. |
| Part B: Multiple Ascending Dose (MAD) | EXPERIMENTAL | Participants will be randomized to receive multiple doses of different dose levels of VX-407. The dose levels will be determined based on the data from Part A. |
| Part B: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive multiple doses of placebo matched to VX-407. |
| Part C: Drug-Drug Interaction | EXPERIMENTAL | Participants will be administered Midazolam (MDZ) in the presence or absence of VX-407. The dose levels will be determined based on the data from Part B. |
| Part D | EXPERIMENTAL | Participants will be randomized to receive VX-407 in 1 of 3 treatment sequences with 3 dosing periods to assess the relative bioavailability of VX-407 formulations and the effect of food on the pharmacokinetics of VX-407. |
| Name | Type | Description |
|---|---|---|
| VX-407 | DRUG | Tablets for oral administration. |
| LNG/EE | DRUG | Combination Tablets for Oral Administration. |
| NGM/EE | DRUG | Combination Tablets for Oral Administration. |
| NET/EE | DRUG | Combination Tablets for Oral Administration. |
| DRSP/EE | DRUG | Combination Tablets for Oral Administration. |
| Placebo | DRUG | Suspension for oral administration. |
| Midazolam | DRUG | Syrup for oral administration. |
Key Inclusion Criteria: * A pre-existing diagnosis of ADPKD as defined in the protocol * Willing and able to comply with scheduled visits and other study procedures * Participants with ADPKD with Mayo imaging classification (MIC) status of 1B (with htTKV ≥250 mL/m), 1C, 1D, or 1E confirmed by abdom...
VX-407 is an investigational small molecule being developed for autosomal dominant polycystic kidney disease, or ADPKD. It is in Phase 2 clinical development and has not been approved for any indication. ADPKD is a genetic kidney disease characterized by the growth of fluid-filled cysts in the kidneys.
VX-407 targets PC1, a protein encoded by the PKD1 gene. PC1 is a large transmembrane protein, and the Phase 2a AGLOW study specifically enrolls participants with ADPKD who have a subset of PKD1 gene variants. Targeting PC1 links the drug's mechanism directly to the genetic cause of disease in this population.
VX-407 is being developed by Vertex Pharmaceuticals Incorporated, which trades on the Nasdaq under the ticker VRTX. Vertex is the sole sponsor listed for the drug's clinical program in autosomal dominant polycystic kidney disease.
VX-407 is in Phase 2 development. The Phase 2a AGLOW study, NCT07161037, is active but no longer recruiting participants. VX-407 is investigational and has not been approved by the FDA or any other regulatory agency for autosomal dominant polycystic kidney disease.
VX-407 has been studied across five clinical trials. The Phase 2a AGLOW study, NCT07161037, enrolled 26 participants with ADPKD across the United States and seven European countries plus Canada. Phase 1 trials include NCT07022119, NCT07074327, and the recruiting NCT07792408, which evaluates effects on combined oral contraceptives.
Across the five reported VX-407 trials, total enrollment is 439 participants. The largest is the recruiting Phase 1 contraceptive pharmacokinetic study NCT07792408 with 148 female healthy volunteers in Canada. The Phase 2a AGLOW study, NCT07161037, enrolled 26 participants with ADPKD.