| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02514473 | A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation | PHASE3 | COMPLETED | 206 | — | — | Jul 1, 2015 | Sep 1, 2016 | Oct 23, 2017 | 53 | United States, Australia +7 |
| NCT00865904 | Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation | PHASE2 | COMPLETED | 93 | — | — | Mar 1, 2009 | Dec 1, 2009 | Aug 28, 2015 | 25 | United States, Belgium +3 |
| NCT01216046 | Drug-Drug Interaction Study of VX-770 and VX-809 in Healthy Subjects | PHASE1 | COMPLETED | 48 | — | — | Oct 1, 2010 | May 1, 2011 | Jan 18, 2012 | 1 | United States |
| NCT00966602 | Drug-Drug Interaction Study of VX-809 and VX-770 in Healthy Subjects | PHASE1 | COMPLETED | 24 | — | — | Sep 1, 2009 | Dec 1, 2009 | Jan 5, 2010 | 1 | Netherlands |
Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple breath washout test using Nitrogen (N2). LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. Serious adverse event (SAE) (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. An AE that started at or after initial dosing of study drug, or increased in severity after initial dosing of study drug visit is considered treatment-emergent.
Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-809 and its metabolite.
Blood samples drawn during the study will be analyzed to measure the PK parameters, such as concentration, exposure and half-life of VX-770 and its metabolites.
| Arm | Type | Description |
|---|---|---|
| LUM/IVA | EXPERIMENTAL | Fixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h |
| Placebo | PLACEBO_COMPARATOR | Matching placebo q12h |
| VX-809, 25 mg | EXPERIMENTAL | VX-809, 25 milligram (mg) capsule orally once daily for 28 days. |
| VX-809, 50 mg | EXPERIMENTAL | VX-809, 50 mg capsule orally once daily for 28 days. |
| VX-809, 100 mg | EXPERIMENTAL | VX-809, 100 mg capsule orally once daily for 28 days. |
| VX-809, 200 mg | EXPERIMENTAL | VX-809, 200 mg capsule orally once daily for 28 days. |
| Treatment Arm | EXPERIMENTAL | Subjects randomized to study drug will take VX-809 for 14 days followed by a 14 day washout. Next subjects will take VX-770 for 14 days followed by a 14 day washout. Lastly, subjects will take both VX-809 and VX-770 for 14 days. |
| Placebo Arm | PLACEBO_COMPARATOR | Subjects randomized to placebo will take VX-809 placebo for 14 days followed by a 14 day washout. Next subjects will take VX-770 placebo for 14 days followed by a 14 day washout. Lastly, subjects will take both VX-809 and VX-770 placebo for 14 days. |
| Treatment Period 1 | EXPERIMENTAL | - |
| Treatment Period 2 | EXPERIMENTAL | - |
| Treatment Period 3 | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| VX-809 | DRUG | - |
| Placebo | DRUG | - |
| VX-770 | DRUG | - |
| VX-809 placebo | DRUG | capsule, taken once daily |
| VX-770 placebo | DRUG | tablet, taken once every 12 hours |
| VX-809 & VX-770 | DRUG | VX-809 capsule, once daily, and VX-770 tablets, once every 12 hours, for 14 days |
Inclusion Criteria: * Subjects who weigh ≥15 kg without shoes a the Screening Visit * Subjects with confirmed diagnosis of CF at the Screening Visit. * Subjects who are homozygous for the F508del CFTR mutation * Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, and height * Subje...
| 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 |