Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as VX-121 (Suspension), VX-121
Alyftrek · 12 trials · 1 indication
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Arm | Type | Description |
|---|---|---|
| VNZ/TEZ/D-IVA | EXPERIMENTAL | Part A: Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (\<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study. Part B: Participants in Cohort 1 who meet the eligibility criteria will receive VNZ/TEZ/D-IVA for an additional 96 weeks. |
| VX-121/TEZ/D-IVA | EXPERIMENTAL | Part A: Participants will receive VX-121/TEZ/D-IVA once daily for 96 weeks. Part B: Participants will receive VX-121/TEZ/D-IVA once daily for an additional 48 weeks. |
| Part A: VX-121/TEZ/D-IVA | EXPERIMENTAL | Participants will receive VX-121/TEZ/D-IVA in the morning. |
| Part B: VX-121/TEZ/D-IVA | EXPERIMENTAL | Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A. |
| ELX/TEZ/IVA | ACTIVE_COMPARATOR | Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks. |
| Part 1: Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to VX-121/TEZ/VX-561 triple combination (TC) for 4 weeks in the treatment period and placebo matched to TEZ/VX-561 for 18 days in the washout period. |
| Part 1: VX-121/TEZ/VX-561 TC - Low Dose | EXPERIMENTAL | Participants received VX-121 5 milligram (mg) once daily (qd)/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period |
| Part 1: VX-121/TEZ/VX-561 TC - Medium Dose | EXPERIMENTAL | Participants received VX-121 10 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. |
| Part 1: VX-121/TEZ/VX-561 TC - High Dose | EXPERIMENTAL | Participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. |
| Part 2: TEZ/IVA | ACTIVE_COMPARATOR | Following run-in period with TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the treatment period and TEZ 100 mg/IVA 150 mg q12h for 4 weeks in the washout period. |
| Part 2: VX-121/TEZ/VX-561 TC - High Dose | EXPERIMENTAL | Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period. |
| VNZ/TEZ/D-IVA and Rosuvastatin | EXPERIMENTAL | Rosuvastatin will be administered to participants as a single oral dose on Day 1 and then co-administered with VNZ/TEZ/D-IVA on Day 18.VNZ/TEZ/D-IVA dose will be administered once daily (qd) from Day 5 to Day 22. |
| Part A: Sequence 1 | EXPERIMENTAL | Participants will receive VNZ/TEZ/D-IVA reference fixed dose combination (FDC) tablet in dosing period 1, then VNZ/TEZ/D-IVA test FDC granules dose level 1 in dosing period 2, and finally VNZ/TEZ/D-IVA test FDC granules dose level 2 in dosing period 3. A washout period of 14 days will be maintained between 3 dosing periods. |
| Part A: Sequence 2 | EXPERIMENTAL | Participants will receive VNZ/TEZ/D-IVA test FDC granules dose level 1 in dosing period 1, then VNZ/TEZ/D-IVA test FDC granules dose level 2 in dosing period 2, and finally VNZ/TEZ/D-IVA reference FDC tablet in dosing period 3. A washout period of 14 days will be maintained between 3 dosing periods. |
| Part A: Sequence 3 | EXPERIMENTAL | Participants will receive VNZ/TEZ/D-IVA test FDC granules dose level 2 in dosing period 1, then VNZ/TEZ/D-IVA reference FDC tablet in dosing period 2, and finally VNZ/TEZ/D-IVA test FDC granules dose level 1 in dosing period 3. A washout period of 14 days will be maintained between 3 dosing periods. |
| Part B: Sequence 1 | EXPERIMENTAL | Participants will receive VNZ/TEZ/D-IVA test FDC granules under fasted condition in dosing period 1, then VNZ/TEZ/D-IVA test FDC granules under fed state in dosing period 2. A washout period of 18 days will be maintained between 2 dosing periods. |
| Part B: Sequence 2 | EXPERIMENTAL | Participants will receive VNZ/TEZ/D-IVA test FDC granules under fed state in dosing period 1, then VNZ/TEZ/D-IVA test FDC granules under fasted condition in dosing period 2. A washout period of 18 days will be maintained between 2 dosing periods. |
| Sequence 1 | EXPERIMENTAL | Participants will receive a single dose of reference FDC tablet of VX-121/TEZ/D-IVA in dosing period 1, followed by a single dose of test FDC tablet of VX-121/TEZ/D-IVA in dosing period 2. A washout period of 14 days will be maintained between the 2 dosing periods. |
| Sequence 2 | EXPERIMENTAL | Participants will receive a single dose of test FDC tablet of VX-121/TEZ/D-IVA in dosing period 1, followed by a single dose of reference FDC tablet of VX-121/TEZ/D-IVA in dosing period 2. A washout period of 14 days will be maintained between the 2 dosing periods. |
| Cohort 1: Moderate Hepatic Impairment | EXPERIMENTAL | Participants with moderate hepatic impairment will receive single dose of VX-121/TEZ/D-IVA . |
| Cohort 2: Matched Healthy Participants | EXPERIMENTAL | Healthy participants will receive single dose of VX-121/TEZ/D-IVA. |
| Part A: Pooled Placebo (Cohorts A1-5; Except A3) | PLACEBO_COMPARATOR | Participants received single dose of placebo matched to VX-121. |
| Part A: VX-121 (Cohort A1) | EXPERIMENTAL | Participants received single dose of VX-121 10 milligrams (mg). |
| Part A: VX-121 (Cohort A2) | EXPERIMENTAL | Participants received single dose of VX-121 20 mg. |
| Part A: VX-121 (Cohort A3) | EXPERIMENTAL | Participants received single dose of VX-121 5 mg or matched placebo without milk, followed by open label VX-121 5 mg with milk. |
| Part A: VX-121 (Cohort A4) | EXPERIMENTAL | Participants received single dose of VX-121 40 mg. |
| Part A: VX-121 (Cohort A5) | EXPERIMENTAL | Participants received single dose of VX-121 60 mg. |
| Part A: VX-121 (Cohort A9) | EXPERIMENTAL | Participants received single dose of VX-121 10 mg suspension on Day 1, VX-121 10 mg tablet on Day 9, followed by VX-121 10 mg tablet with milk on Day 17. |
| Part B: Pooled Placebo (Cohorts B1-4) | PLACEBO_COMPARATOR | Participants received placebo matched to VX-121 for 10 days. |
| Part B: VX-121 (Cohort B1) | EXPERIMENTAL | Participants received VX-121 10 mg once daily (qd) for 10 days. |
| Part B: VX-121 (Cohort B2) | EXPERIMENTAL | Participants received VX-121 20 mg qd for 10 days. |
| Part B: VX-121 (Cohort B3) | EXPERIMENTAL | Participants received VX-121 40 mg qd for 10 days. |
| Part B: VX-121 (Cohort B4) | EXPERIMENTAL | Participants received VX-121 60 mg qd for 10 days. |
| Part C: Pooled Placebo (Cohorts C1-3) | PLACEBO_COMPARATOR | Participants received placebo matched to VX-121/TEZ/IVA for 14 days. |
| Part C: VX-121 (Cohort C1) | EXPERIMENTAL | Participants received VX-121 10 mg qd/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) for 14 days. |
| Part C: VX-121 (Cohort C2) | EXPERIMENTAL | Participants received VX-121 20 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
| Part C: VX-121 (Cohort C3) | EXPERIMENTAL | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 14 days. |
| Part D: Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to VX-121/TEZ/IVA for 4 weeks. |
| Part D: VX-121/TEZ/IVA | EXPERIMENTAL | Participants received VX-121 5 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks. |
| Name | Type | Description |
|---|---|---|
| VNZ/TEZ/D-IVA | DRUG | Fixed-dose combination tablets or granules for oral administration. |
| VX-121/TEZ/D-IVA | DRUG | Fixed-dose combination tablets for oral administration. |
| ELX/TEZ/IVA | DRUG | Fixed-dose combination tablets for oral administration. |
| IVA | DRUG | Tablet for oral administration. |
| Placebo (matched to VX-121/TEZ/D-IVA) | DRUG | Placebo matched to VX-121/TEZ/D-IVA for oral administration. |
| Placebo (matched to ELX/TEZ/IVA) | DRUG | Placebo matched to ELX/TEZ/IVA for oral administration. |
| Placebo (matched to IVA) | DRUG | Placebo matched to IVA for oral administration. |
| VX-121 | DRUG | Tablets for oral administration. |
| TEZ | DRUG | TEZ tablet for oral administration. |
| VX-561 | DRUG | Tablets for oral administration. |
| TEZ/IVA | DRUG | Fixed-dose combination tablets for oral administration. |
| Placebo | DRUG | Placebos matched to VX-121, TEZ, and VX-561 for oral administration. |
| Rosuvastatin | DRUG | Tablet for oral administration. |
| Placebo (matched to VX-121 suspension) | DRUG | Placebo matched to VX-121 suspension for oral administration. |
| VX-121 (Suspension) | DRUG | Suspension for oral administration. |
| Placebo (matched to TEZ/IVA) | DRUG | Placebo matched to TEZ/IVA for oral administration. |
| VX-121 (Tablet) | DRUG | Tablet for oral administration. |
| Placebo (matched to VX-121 tablet) | DRUG | Placebo matched to VX-121 tablet for oral administration. |
Key Inclusion Criteria: Parts A and B: • Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222) Part B: -Meets at least 1 of the following criteria: * Completed study drug treatment in Part A * Had study drug interruption(s) in Part A, b...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |