Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
LUM/IVA · 7 trials · 1 indication
MRI scans assessed semi-quantitatively via a standardized chest MRI scoring system. Each participant had 6 lobes scored using 7 scoring parameters:1) Bronchiectasis/wall thickening 2) Mucus plugging 3) Abscesses/sacculations 4) Consolidations 5) Special findings 6)Mosaic pattern 7) Perfusion abnormalities. For each of 7 parameter, there were scores of 6 lobes (score of each lobe : 0= normal value, 1 = \<50% of lobe involved and 2 = \>=50% of lobe involved). MRI global score was calculated as sum of parameters 1 to 7. MRI total score is ranged from 0-84. Higher score indicate more lobe involvement.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Arm | Type | Description |
|---|---|---|
| LUM/IVA | EXPERIMENTAL | Participants weighing 7 to less than (\<) 9 kilograms (kg) received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants weighing greater than or equal to (\>=)14 kg received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 weeks. |
| Part A: LUM/IVA | EXPERIMENTAL | Participants weighing 7 to less than (\<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to \<14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days. |
| Part B: LUM/IVA | EXPERIMENTAL | Participants weighing 7 to \<9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight. |
| Lumacaftor/Ivacaftor (LUM/IVA) | EXPERIMENTAL | Part A (\<14 kg): Participants weighing less than (\<) 14 kilograms (kg) at screening received LUM 100 milligram (mg)/IVA 125 mg fixed-dose combination every 12 hours for 15 days in Part A. Part A (\>=14 kg): Participants weighing greater than or equal to (\>=) 14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 15 days in Part A. Part B (\<14 kg): Participants weighing \<14 kg at screening received LUM 100 mg/IVA 125 mg fixed-dose combination every 12 hours for 24 weeks in Part B. Part B (\>=14 kg): Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg fixed-dose combination every 12 hours for 24 weeks in Part B. |
| Treatment Period 1: LUM/IVA to LUM/IVA | EXPERIMENTAL | - |
| Treatment Period 1: Placebo (PBO) to LUM/IVA | EXPERIMENTAL | - |
| Treatment Period 1: Observational Cohort | NO_INTERVENTION | - |
| Treatment Period 2: LUM/IVA | EXPERIMENTAL | - |
| Part 1: Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks. |
| Part 1: LUM/IVA | EXPERIMENTAL | Participants weighing less than (\<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Participants weighing greater than or equals to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks. |
| Part 2: Overall LUM/IVA | EXPERIMENTAL | Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \>=6 years of age at week 48) in open-label period for 48 weeks. |
| Treatment Sequence 1 | EXPERIMENTAL | LUM/IVA in Treatment Period 1; washout; placebo in Treatment Period 2 |
| Treatment Sequence 2 | EXPERIMENTAL | Placebo in Treatment Period 1; washout; LUM/IVA in Treatment Period 2 |
| Name | Type | Description |
|---|---|---|
| LUM/IVA | DRUG | LUM/IVA granules for oral administration |
| LUM | DRUG | Fixed Dose Combination (FDC) granules (LUM/IVA). |
| IVA | DRUG | FDC granules (LUM/IVA). |
| Placebo | DRUG | Placebo matched to LUM/IVA for oral administration. |
Key Inclusion Criteria: * Participants From Study VX16-809-122 Part B (Study 122) * Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B * Participants Not From Study 122 * Subjects will be 1 to less than 2 years of age * Homozygous for the F508del mutation (F...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| 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 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
LUM/IVA is used for cystic fibrosis, a genetic respiratory condition. It is a combination of lumacaftor and ivacaftor, two small molecules, and is being developed by Vertex Pharmaceuticals. The drug has been studied in patients with specific CFTR mutations, including those homozygous for F508del and those with the A455E mutation.
LUM/IVA targets the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Lumacaftor is a corrector that helps the defective protein fold properly, while ivacaftor is a potentiator that enhances its function. This combination aims to improve chloride transport in patients with cystic fibrosis.
LUM/IVA is developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker VRTX. The company has conducted multiple clinical trials of this combination therapy for cystic fibrosis.
LUM/IVA has completed Phase 3 clinical trials for cystic fibrosis. It is an investigational drug that has been studied in both Phase 2 and Phase 3 trials, with all seven trials completed. It is not yet approved, as it remains in clinical development.
LUM/IVA has completed seven clinical trials, including NCT02797132, a Phase 3 study in children aged 2-5 with cystic fibrosis homozygous for F508del, and NCT03061331, a Phase 2 study in patients with the A455E mutation. Other trials include NCT03625466 and NCT04235140, both completed.
Yes, LUM/IVA is the same as lumacaftor/ivacaftor. It is a combination of two small molecules, lumacaftor and ivacaftor, used for cystic fibrosis. The drug has been studied in patients with specific CFTR mutations, including F508del and A455E.