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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03277196 | A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation | PHASE3 | COMPLETED | 86 | — | — | Aug 16, 2017 | Oct 2, 2023 | Oct 23, 2024 | 29 | United States, Australia +4 |
| NCT02725567 | A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation | PHASE3 | COMPLETED | 57 | — | — | Jun 2, 2016 | Jun 28, 2022 | Sep 7, 2023 | 23 | United States, Australia +3 |
| NCT03227471 | A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis | PHASE1 | COMPLETED | 225 | — | — | Jan 23, 2017 | Mar 27, 2018 | Jan 18, 2022 | 38 | United States, Australia +2 |
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Arm | Type | Description |
|---|---|---|
| Ivacaftor Arm | EXPERIMENTAL | Participants less than (\<) 24 months of age and weighing 5 to less than (\<) 7 kilogram (kg) received 25 mg IVA every 12 hours (q12h), 7 to \<14 kg received 50 mg IVA q12h, and those weighing 14 to \<25 kg received 75 mg IVA q12h. Participants more than or equal (\>=) 24 months of age and weighing \<14 kg received 50 mg IVA q12h, and those weighing more than or equal to (\>=)14 kg received 75 mg IVA q12h in the treatment period for up to 96 weeks. Doses were determined based on safety and pharmacokinetic (PK) data from parent study, age and weight. |
| Observational Arm | NO_INTERVENTION | - |
| Part A: 3 to <24 months | EXPERIMENTAL | Participants weighing 5 to less than (\<) 7 kilogram (kg) received 25 milligram (mg) IVA, 7 to \<14 kg received 50 mg IVA, and those weighing 14 to \<25 kg received 75 mg IVA administered every 12 hours (q12h) on Days 1 through 3 and 1 morning dose on Day 4. |
| Part B + A/B:1 to < 24 months | EXPERIMENTAL | Participants 4 to \<6 months of age and weighing greater than or equal to (≥) 5 kg received 25 mg IVA q12h. At 6 months of age and older, participants weighing 5 to \<7 kg received 25 mg IVA, 7 to \<14 kg received 50 mg IVA, and those weighing 14 to \<25 kg received 75 mg IVA q12h for 24 weeks on Part B. For Part A/B, participants 1 to \<4 months weighing 3 kg to \<5 kg received an initial low dose of 5.7 mg q12h IVA and those weighing ≥5 kg received 11.4 mg q12h IVA for the first 15 days of IVA treatment. Doses were maintained or adjusted upward at Day 15 and based on weight and/or age once they reached 4 months of age. |
| Part A: Pooled Placebo (Except Cohort A7) | PLACEBO_COMPARATOR | Participants without CF who received single dose of placebo matched to VX-445 in Cohort A1 to A5. |
| Part A: VX-445 (Except Cohort A7) | EXPERIMENTAL | Participants without CF who received single ascending dose of VX-445 tablet starting from 20 milligrams (mg) to 360 mg in Cohort A1 to A5. |
| Part A: VX-445 (Cohort A7) | EXPERIMENTAL | Participants without CF who received single dose of VX-445 100 mg tablet on Day 1 in fasted state and on Day 7 in fed state, followed by VX-445 20 mg intravenous (IV) injection on Day 13 in fed state in Cohort A7. |
| Part B: Pooled Placebo (Cohort B1 to B4) | PLACEBO_COMPARATOR | Participants without CF who received multiple doses of placebo matched to VX-445 once daily (qd) for 10 days in Cohort B1 to B4. |
| Part B: VX-445 (Cohort B1 to B4) | EXPERIMENTAL | Participants without CF who received VX-445 tablet qd for 10 days in Cohort B1 (60 mg), B2 (120 mg), B3 (240 mg) and B4 (340 mg). |
| Part C: Pooled Placebo (Cohort C1 to C3) | PLACEBO_COMPARATOR | Participants without CF who received placebo matched to VX-445/TEZ/IVA triple combination (TC) qd in the morning and placebo matched to IVA in the evening for 14 days. |
| Part C: VX-445/TEZ/IVA TC (Cohort C1 to C3) | EXPERIMENTAL | Participants without CF who received VX-445 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in Cohort C1; VX-445 280 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in Cohort C2 and VX-445 100 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in Cohort C3 for 14 days. |
| Part D: Placebo | PLACEBO_COMPARATOR | Participants with CF, F/MF genotype who received placebo matched to VX-445/TEZ/IVA TC qd in the morning and placebo matched to IVA qd in the evening for 4 weeks in the TC treatment period. |
| Part D: VX-445/TEZ/IVA TC - Low Dose | EXPERIMENTAL | Participants with CF, F/MF genotype who received VX-445 50 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period. |
| Part D: VX-445/TEZ/IVA TC - Medium Dose | EXPERIMENTAL | Participants with CF, F/MF genotype who received VX-445 100 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period. |
| Part D: VX-445/TEZ/IVA TC - High Dose | EXPERIMENTAL | Participants with CF, F/MF genotype who received VX-445 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the TC treatment period. |
| Part E: TEZ/IVA | ACTIVE_COMPARATOR | Following run-in period of 4 weeks with TEZ/IVA, participants with CF, F/F genotype who received TEZ 100 mg qd/IVA 150 mg q12h and placebo matched to VX-445 for 4 weeks in the TC treatment period. |
| Part E: VX-445/TEZ/IVA TC | EXPERIMENTAL | Following run-in period of 4 weeks with TEZ/IVA, participants with CF, F/F genotype who received VX-445 200 mg qd/TEZ 100 mg qd /IVA 150 mg q12h for 4 weeks in the TC treatment period. |
| Part F: Placebo | PLACEBO_COMPARATOR | Participants with CF, F/MF genotype who received placebo matched to VX-445/TEZ/VX-561 for 4 weeks in the TC treatment period. |
| Part F: VX-445/TEZ/VX-561 TC | EXPERIMENTAL | Participants with CF, F/MF genotype who received VX-445 200 mg qd/TEZ 100 mg qd/VX-561 150 mg qd for 4 weeks in the TC treatment period. |
| Name | Type | Description |
|---|---|---|
| IVA | DRUG | Granules for oral administration. |
| TEZ/IVA | DRUG | TEZ/IVA fixed-dose combination for oral administration. |
| VX-445 | DRUG | VX-445 tablet for oral administration. |
| Matched Placebo | DRUG | Matched placebo. |
| TEZ | DRUG | Tablet for oral administration. |
| VX-561 | DRUG | Tablet for oral administration. |
Inclusion Criteria: Ivacaftor Arm: Participants From Study 124 (NCT02725567 ) Part B: * Participants transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B. * As judged by the investigator, parent or legal guardian must be able to understand protocol requi...
| 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 |