Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Trikafta · 19 trials · 1 indication
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Percent reduction in cough frequency was analyzed with a mixed effects model for repeated measures (MMRM), with change from baseline at each post-baseline visit on the natural log scale as the dependent variable. The percent reduction was estimated as 100% × (1-exponential form of LS mean change estimate from the MMRM).
Baseline 2-hour post-OGTT blood glucose level was defined as the average of valid pre-dose measurements at screening and Day 1. OGTT results were considered valid only when the participant was fasting for at least 8 hours.
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Arm | Type | Description |
|---|---|---|
| ELX/TEZ/IVA | EXPERIMENTAL | Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening. |
| Part A: ELX/TEZ/IVA | EXPERIMENTAL | Participants received ELX 40 milligram (mg)/ TEZ 20 mg/IVA 30 mg fixed-dose combination (FDC) granules once every morning (qAM) and 25 mg IVA once every evening (qPM) for 15 Days. |
| Part B: ELX/TEZ/IVA | EXPERIMENTAL | Participants received one of three dosing regimens based on their body weight at Day 1 . Dosage 1: ELX 80 mg qAM/TEZ 40 mg qAM /IVA 60 mg qAM FDC granules and 59.5 IVA mg qPM; Dosage 2: ELX 60 mg qAM /TEZ 30 mg qAM /IVA 45 mg qAM FDC granules and 50 mg IVA qPM; Dosage 3: ELX 40 mg qAM / TEZ 20 mg qAM /IVA 30 mg qAM FDC granules and 25mg IVA qPM. Part B included some participants from Part A and newly enrolled participants. |
| Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks. |
| TEZ/IVA | ACTIVE_COMPARATOR | Following TEZ/IVA run-in period of 4 weeks, participants received TEZ 100 milligrams (mg) once daily (qd)/IVA 150 mg every 12 hours (q12h) in the treatment period for 24 weeks. |
| Control: IVA or TEZ/IVA | ACTIVE_COMPARATOR | Following an IVA or TEZ/IVA run-in period of 4 weeks, participants either received IVA 150 milligrams (mg) every 12 hours (q12h) or TEZ 100 mg once daily (qd)/IVA 150 mg q12h in the treatment period for 8 weeks. |
| TC: ELX/TEZ/IVA | EXPERIMENTAL | Following an IVA or TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 8 weeks. |
| Name | Type | Description |
|---|---|---|
| ELX/TEZ/IVA | DRUG | Fixed-dose combination granules for oral administration. |
| IVA | DRUG | Granules for oral administration |
| Placebo (matched to ELX/TEZ/IVA) | OTHER | Placebo matched to ELX/TEZ/IVA for oral administration. |
| Placebo (matched to IVA) | OTHER | Placebo matched to IVA for oral administration. |
| TEZ/IVA | DRUG | Fixed-dose combination (FDC) tablet for oral administration. |
Key Inclusion Criteria: * Completed study drug treatment in the parent study VX22-445-122 Part B (NCT05882357) OR had study drug interruption(s) in the parent study but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the Treatment Period of th...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-272, Tezacaftor |
| BiomX Inc. | 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 |
| Roivant Sciences Ltd. | ROIV | 1 | PHASE1 | SP-101 |
Trikafta is used to treat cystic fibrosis, a genetic disease affecting the lungs and other organs. It is a small molecule therapy developed by Vertex Pharmaceuticals. Trikafta has been studied in Phase 3 clinical trials in people with cystic fibrosis, including participants with and without the F508del mutation.
Trikafta is made by Vertex Pharmaceuticals Incorporated, which trades on Nasdaq under the ticker VRTX. Vertex is the developer of the elexacaftor, tezacaftor, and ivacaftor combination and has run the Phase 3 cystic fibrosis program for the therapy across multiple countries.
Trikafta is in Phase 3 development for cystic fibrosis. Its clinical program includes 19 trials in total, of which 17 are completed and 2 are active but not recruiting. Across these studies, total enrollment is 3,323 participants, and the trials are randomized, double-blind, and active-controlled.
Trikafta trials include NCT06460506, a Phase 3 long-term safety and efficacy study in cystic fibrosis participants 12 months and older that is active and not recruiting, and NCT05882357, a completed Phase 3 study in participants 12 to less than 24 months of age. Other Phase 3 studies include NCT05331183 and NCT05274269 in participants without F508del.
Trikafta has been studied in 19 clinical trials, with 17 completed and 2 active but not recruiting. Total enrollment across these trials is 3,323 participants. The studies are randomized, double-blind, and active-controlled, and include biomarker-selected cystic fibrosis populations, with Phase 3 trials conducted across Europe, Canada, and Australia.