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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06861413 | A Phase 1 Study to Evaluate the Relative Bioavailability of a VX-828 Tablet Formulation | PHASE1 | COMPLETED | 68 | — | — | Apr 1, 2025 | May 5, 2025 | Jun 4, 2025 | 1 | United States |
| NCT06154447 | Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis | PHASE1 | ACTIVE NOT_RECRUITING | 165 | — | — | Dec 12, 2023 | Jul 16, 2026 | May 26, 2026 | 12 | United States |
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | ACTIVE_COMPARATOR | Participants will be randomized to receive a single dose of VX-828 suspension. |
| Cohort 2 | EXPERIMENTAL | Participants will be randomized to receive a single dose of VX-828 tablets. |
| Part A: Single Ascending Dose (SAD) | EXPERIMENTAL | Participants will be randomized to receive a single dose of different dose levels of VX-828. |
| Part A: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-828. |
| Part B: Multiple Ascending Dose (MAD) | EXPERIMENTAL | Participants will be randomized to receive multiple doses of different dose levels of VX-828. The dose levels will be determined based on the data from Part A. |
| Part B: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-828. |
| Part C: Drug Drug Interaction | EXPERIMENTAL | Participants will receive a single dose of VX-828, followed by a washout period, Itraconazole administration, and concomitant administration of itraconazole and VX-828; or participants will receive Midazolam followed by VX-828/TEZ/D-IVA administration, and concomitant administration of VX-828/TEZ/D-IVA and Midazolam. Part C will be an open-label optional cohort. |
| Part D: VX-828 in combination with TEZ/VX-118 ,TEZ/D-IVA or D-IVA | EXPERIMENTAL | Participants will be randomized to receive VX-828 in combination with TEZ/VX-118, TEZ/D-IVA, or D-IVA. |
| Part D: Placebo | PLACEBO_COMPARATOR | Participants will be randomized to receive placebo matched to VX-828/TEZ/VX-118 or placebo matched to VX-828 in combination with TEZ/D-IVA or D-IVA |
| Part E: VX-828 in Combination with D-IVA with or without TEZ in CF | EXPERIMENTAL | Participants with cystic fibrosis will receive VX-828 in combination with D-IVA with or without TEZ. |
| Name | Type | Description |
|---|---|---|
| VX-828 | DRUG | Tablets for Oral Administration. |
| Placebo | DRUG | Suspension for Oral Administration |
| Itraconazole | DRUG | Solution for Oral Administration |
| Midazolam | DRUG | Syrup for Oral Administration |
| Tezacaftor | DRUG | Tablets for Oral Administration |
| VX-118 | DRUG | Tablets for Oral Administration |
| Deutivacaftor | DRUG | Tablets for Oral Administration |
Key Inclusion Criteria: * Body mass index (BMI) of 18.0 to 32.0 kilogram per square meter (kg/m\^2) * A total body weight of more than (\>) 50 Kg * Nonsmoker or ex-smoker for at least 3 months before screening * Participants of non-childbearing potential Key Exclusion Criteria: * History of febri...
| 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 |