Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05444257 | A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy | PHASE3 | ACTIVE NOT_RECRUITING | 822 | — | — | Nov 8, 2022 | Oct 30, 2026 | May 5, 2026 | 195 | United States, Australia +21 |
| NCT05422222 | Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age | PHASE3 | ACTIVE NOT_RECRUITING | 210 | — | — | Jun 21, 2022 | Jun 30, 2030 | May 28, 2026 | 38 | United States, Australia +8 |
| NCT05076149 | A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation | PHASE3 | COMPLETED | 597 | — | — | Oct 27, 2021 | Nov 30, 2023 | Jun 13, 2024 | 170 | United States, Australia +18 |
| NCT05033080 | A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF) | PHASE3 | COMPLETED | 435 | — | — | Sep 14, 2021 | Nov 21, 2023 | Oct 3, 2024 | 139 | United States, Australia +10 |
| NCT03912233 | A Study to Evaluate the Safety and Efficacy of VX-121 Combination Therapy in Subjects With Cystic Fibrosis | PHASE2 | COMPLETED | 87 | — | — | Apr 30, 2019 | Dec 10, 2019 | Apr 20, 2023 | 26 | United States, Germany +3 |
| NCT05535959 | A Study to Evaluate the Relative Bioavailability of a Fixed-dose Combination Tablet of VX-121/Tezacaftor/Deutivacaftor | PHASE1 | COMPLETED | 16 | — | — | Sep 15, 2022 | Nov 9, 2022 | Dec 12, 2022 | 1 | United States |
| NCT05437120 | Pharmacokinetics and Safety Assessment of VX-121/Tezacaftor/Deutivacaftor in Participants With Moderate Hepatic Impairment | PHASE1 | COMPLETED | 16 | — | — | Jul 22, 2022 | Mar 16, 2023 | Mar 30, 2023 | 2 | United States |
| NCT03768089 | Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis | PHASE1 | COMPLETED | 115 | — | — | Mar 20, 2018 | May 3, 2019 | Jul 14, 2022 | 7 | Netherlands, United Kingdom |
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 |
|---|---|---|
| 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. |
| 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 |
|---|---|---|
| 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. |
| 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: * Part A: Completed study drug treatment in a parent study VX20-121-102 (NCT05033080) and VX20-121-103 (NCT05076149); or had study drug interruption(s) in a parent study but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 9 | PHASE3 | VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719, SION-451, SION-2222, SION-109 |
| BiomX Ltd | 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 |