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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03911713 | A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis | PHASE2 | COMPLETED | 77 | — | — | Apr 17, 2019 | Aug 20, 2020 | Jan 25, 2022 | 48 | United States, Australia +5 |
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Arm | Type | Description |
|---|---|---|
| Ivacaftor | ACTIVE_COMPARATOR | Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks. |
| VX-561: 25 mg | EXPERIMENTAL | Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks. |
| VX-561: 50 mg | EXPERIMENTAL | Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks. |
| VX-561: 150 mg | EXPERIMENTAL | Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks. |
| VX-561: 250 mg | EXPERIMENTAL | Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks. |
| Name | Type | Description |
|---|---|---|
| VX-561 | DRUG | VX-561 tablets for oral administration. |
| IVA | DRUG | 150-mg film-coated tablet for oral administration. |
| Placebo | DRUG | Placebo matched to IVA. |
Key Inclusion Criteria: * Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D * On ivacaftor therapy * FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height Key Exclusion Criteria: * History of cli...
| 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 |