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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03150719 | A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF) | PHASE3 | COMPLETED | 98 | — | — | May 24, 2017 | Aug 9, 2018 | Sep 12, 2019 | 53 | United States, France +1 |
| NCT04006873 | Gut Imaging for Function & Transit in Cystic Fibrosis Study 2 | PHASE2 | COMPLETED | 12 | — | — | Sep 3, 2019 | Oct 29, 2020 | May 24, 2023 | 1 | United Kingdom |
| NCT02730208 | A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation | PHASE2 | COMPLETED | 41 | — | — | Sep 1, 2016 | Jul 1, 2018 | Oct 23, 2019 | 9 | Australia |
| NCT02508207 | A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation | PHASE2 | COMPLETED | 34 | — | — | Feb 1, 2016 | Jun 1, 2017 | Jul 19, 2021 | 7 | United States |
RAESIs included chest discomfort, dyspnea (shortness of breath), respiration abnormal (chest tightness), asthma, bronchial hyperreactivity, bronchospasm, and wheezing.
Time taken after eating for ingested food to be identifiable at the caecum on MRI
The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease.
MCC was assessed using an imaging technique that enables the tracking of mucus within the airways. MCC was expressed as the percentage of whole-lung clearance through 60 minutes at Baseline and Day 28.
| Arm | Type | Description |
|---|---|---|
| Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to TEZ/IVA fixed-dose combination tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 56 days. |
| TEZ/IVA | EXPERIMENTAL | Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed-dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 56 days. |
| Tezacaftor/Ivacaftor in combination with Ivacaftor | ACTIVE_COMPARATOR | A film-coated tablet containing 100mg tezacaftor and 150mg ivacaftor will be taken in the morning. A film-coated tablet containing 150mg ivacaftor will be taken in the evening. Participants will take these tablets for 28 days. All tablets are licensed for use in the EU. |
| Name | Type | Description |
|---|---|---|
| Tezacaftor/Ivacaftor | DRUG | TEZ 100 mg/IVA 150 mg fixed-dose combination tablet. |
| Ivacaftor | DRUG | IVA 150 mg tablet. |
| Placebo | DRUG | Placebo matched to TEZ/IVA fixed-dose combination tablet. |
| Tezacaftor/Ivacaftor + Ivacaftor | DRUG | As per description in arms. |
| Placebo oral tablet | DRUG | As per description in arms |
| Tezacaftor/Ivacaftor matching placebo | DRUG | - |
| Ivacaftor matching placebo | DRUG | - |
Inclusion Criteria: * Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures. * Prior discontinuation of Orkambi, with at least 1 respiratory sign or symptom considered related to therapy. * Resolu...
| 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 |