On CFF website. Study not enrolling yet, no participating sites listed yet. See below for list of mutations that are eligible.
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| Study Name: | Vertex 661 and ivacaftor (Vx-661-107) in people with cystic fibrosis ages 12 and older with one copy of the F508del-CFTR mutation | |
| Study Type: | Interventional | |
| Intervention Category: | CFTR Modulation | |
| Study Sponsor: | Vertex | |
| Study Phase: | 3 | |
| Recruitment Status: | Not Yet Recruiting | |
| Study Drug(s): | VX-661/VX-770 | |
| Number of Participants Being Recruited: | 278 | |
| Single / Multi-Center: | Multi-Center |
| STUDY BACKGROUND INFORMATION: |
| This is a Phase 3, multi-center trial that will look at the safety and effectiveness of the drug VX-661 in combination with ivacaftor. Researchers will test the drug's effectiveness by measuring participant's lung function in addition to other outcome measures. This trial is for people ages 12 years and older with one copy of the F508del-CFTR mutation and a second CFTR Mutation that is not likely to respond to therapy. The trial will last approximately 6 months. It will require blood draws, sweat tests and lung function tests. Mutations eligible for this study include: Nonsense mutations: Q39X, W57X, E60X, R75X, E92X, Q98X, Y122X, L218X, Q220X, C276X, Q290X, G330X, W401X, Q414X, S434X, S466X, S489X, Q493X, W496X, Q525X, G542X, Q552X, R553X, E585X, G673X, R709X, K710X, L732X, R764X, R785X, R792X, E822X, W846X, R851X, Q890X, S912X, W1089X, Y1092X, E1104X, R1158X, R1162X, S1196X, W1204X, S1255X, W1282X, Q1313X Canonical splice mutations: 621+1G?T, 711+1G?T, 711+5G?A, 712-1G?T, 405+1G?A, 405+3A?C, 406-1G?A, 621+1G?T, 1248+1G?A, 1341+1G?A, 1717-1G?A, 1811+1.6kbA?G, 1811+1G?C, 1812-1G?A, 1898+1G?A, 2622+1G?A, 3120+1G?A, 3120G?A, 3850-3T?G, 3850-1G?A, 4005+1G?A, 4374+1G?T Frameshift mutations: 663delT, 2183AA?G, CFTRdel2,3, 3659delC, 394delTT, 2184insA, 3905insT, 2184delA, 1078delT, 1154insTC, 2183delAA?G, 2143delT, 1677delTA, 3876delA, 2307insA, 4382delA7, 4016insT, 2347delG, 3007delG, 574delA, 2711delT, 3791delC, CFTRdele22-23, 457TAT?G, 2043delG, 2869insG, 3600+2insT, 3737delA, 4040delA, 541delC Missense mutations: A46D6, T338I8, R347P, L927P, G85E, S341P6, L467P6, I507del, V520F, A559T6, R560T, R560S, A561E, Y569D6, L1065P, R1066C, R1066M, L1077P6, H1085R6, M1101K, N1303K |
| ELIGIBILITY |
| Age: | >= 12 Years |
| FEV1: | 40 - 90 Percent Predicted |
| P. aeruginosa status: | Not applicable |
| B. cepacia status: | Not applicable |
| Other Primary Eligibility Requirements: | |
| Note: Detailed eligibility criteria information may be available on clinicaltrials.gov. If a specific trial listing for this trial is available, a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below. |