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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01161537 | Study of the Effect of VX-770 on Hyperpolarized Helium-3 Magnetic Resonance Imaging in Subjects With Cystic Fibrosis and the G551D Mutation | PHASE2 | COMPLETED | 13 | — | — | Oct 1, 2010 | Feb 1, 2013 | Jul 31, 2014 | 1 | United States |
| NCT01060566 | Study of VX-770 on Midazolam and Rosiglitazone and the Effect of Fluconazole on VX-770 | PHASE1 | COMPLETED | 24 | — | — | Feb 1, 2010 | Apr 1, 2010 | Apr 16, 2010 | 1 | United States |
| NCT01018368 | Study of VX-770 and Rifampin in Healthy Male Subjects | PHASE1 | COMPLETED | 24 | — | — | Nov 1, 2009 | Mar 1, 2010 | May 10, 2010 | 1 | United States |
Subjects inhaled hyperpolarized helium-3 (3He) gas mixed with nitrogen to make a total volume of approximately one-third forced vital capacity (FVC) to a maximum of 1 liter and hold their breath for 20 seconds or less. Rapid magnetic resonance imaging (MRI) was performed during inhalation/exhalation and/or breath-hold. Areas of decreased ventilation were observed as ventilation defects that are visualized as decreased (and/or absent) 3He intensity in 3He-MRI. The total ventilation defect was defined as the ratio of total ventilation defect volume (L) to total lung volume (L), expressed as a percentage. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in VX-770 treatment phase (Day 15 to 42).
Subjects were asked to inhale hyperpolarized 3 He gas mixed with nitrogen to make a total volume of approximately one-third forced vital capacity (FVC) to a maximum of 1 liter and hold their breath for 20 seconds or less. Rapid MRI was performed during inhalation/exhalation and/or breath-hold. Areas of decreased ventilation were observed as ventilation defects that are visualized as decreased (and/or absent) 3He intensity in 3He MRI. The total ventilation defect was defined as the ratio of total ventilation defect volume (L) to total lung volume (L), expressed as a percentage. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in Part B (48 weeks).
| Arm | Type | Description |
|---|---|---|
| VX-770 | EXPERIMENTAL | Part A: Subjects received placebo tablets matched to VX-770 150 milligram (mg) orally twice daily from Day 1 to 14 (Placebo run-in period), followed by VX-770 150 mg tablets orally twice daily from Day 15 to 42 (VX-770 treatment period), and then placebo tablets matched to VX-770 150 mg orally twice daily from Day 43 to 57 (Placebo washout period) during Part A of the study. Part B: Subjects received VX-770 150 mg tablets orally twice daily for 48 weeks during Part B of the study. Part B included subjects from Part A and newly enrolled subjects. |
| Midazolam | EXPERIMENTAL | - |
| Rosiglitazone | EXPERIMENTAL | - |
| Fluconazole | EXPERIMENTAL | - |
| Rifampin | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| VX-770 | DRUG | Tablet. |
| Placebo | DRUG | Tablet. |
| Rifampin | DRUG | In Period 2, subjects will receive a daily oral dose of rifampin 600 mg on Days 1 through 10 |
Inclusion Criteria: * Male or female with Cystic Fibrosis * Must have the G551D-CFTR mutation on at least 1 allele * FEV1 ≥40% of predicted normal for age, gender, and height at Screening * 12 years of age or older * Must be able to swallow tablets Exclusion Criteria: * History of solid organ or ...
| 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 |