Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Lumacaftor Plus Ivacaftor Combination · 3 trials · 2 indications
AE: as any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Any AE that increased in severity or newly developed at or after initial dosing of study drug was considered treatment-emergent.
AE: as any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, In-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Any AE that increased in severity or newly developed at or after initial dosing of study drug was considered treatment-emergent.
Absolute change from baseline at Week 24 was assessed as the average treatment effect at Week 16 and at Week 24. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years.
| Arm | Type | Description |
|---|---|---|
| Arm 1 Part A: LUM 600 mg qd/ IVA 250 mg q12h | EXPERIMENTAL | Participants who received lumacaftor (LUM, VX-809) 600 milligram (mg) plus ivacaftor (IVA, VX-770) 250 mg fixed-dose combination (FDC) tablet orally in the morning and IVA 250 mg film-coated tablet orally in the evening, in the previous study VX12-809-103 or VX12-809-104, and will receive the same treatment in this study VX12-809-105 up to Week 96. |
| Arm 2 Part A: Placebo - LUM 600 mg qd/ IVA 250 mg q12h | EXPERIMENTAL | Participants who received placebo matched to LUM and IVA tablet in the previous study VX12-809-103 or VX12-809-104, and will receive LUM 600 mg plus IVA 250 mg FDC tablet orally in the morning and IVA 250 mg film-coated tablet orally in the evening in this study VX12-809-105 up to Week 96. |
| Arm 3 Part A: LUM 400 mg q12h/ IVA 250 mg q12h | EXPERIMENTAL | Participants who received LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in the previous study VX12-809-103 or VX12-809-104, and will receive the same treatment in this study VX12-809-105 up to Week 96. |
| Arm 4 Part A: Placebo - LUM 400 mg q12h/ IVA 250 mg q12h | EXPERIMENTAL | Participants who received placebo matched to LUM and IVA tablet in the previous study VX12-809-103 or VX12-809-104, and will receive LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in this study VX12-809-105 up to Week 96. |
| Arm 5 Part A: Observational Cohort | NO_INTERVENTION | Participants who received either LUM 600 mg plus IVA 250 mg FDC tablet orally in the morning and IVA 250 mg film-coated tablet orally in the evening OR LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening OR placebo matched to LUM and IVA in the morning and evening, in the previous study VX12-809-103 or VX12-809-104, and will be observed (will not receive study drug) in this study VX12-809-105 for up to 2 years. |
| Arm 6 Part B: LUM 400 mg q12h/ IVA 250 mg q12h | EXPERIMENTAL | Participants who received LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in Cohort 4 of the previous study VX09-809-102, and will receive the same treatment in this study VX12-809-105 up to Week 96. |
| Arm 7 Part B: Placebo - LUM 400 mg q12h/ IVA 250 mg q12h | EXPERIMENTAL | Participants who received placebo matched to LUM and IVA tablet in Cohort 4 of the previous study VX09-809-102, and will receive LUM 400 mg plus IVA 250 mg FDC tablet orally in the morning and evening in this study VX12-809-105 up to Week 96. |
| Placebo | PLACEBO_COMPARATOR | Placebo matched to lumacaftor (LUM, VX-809) and ivacaftor (IVA, VX-770) tablet every 12 hours (q12h), up to Week 24. |
| LUM 600 mg qd/IVA 250 mg q12h | EXPERIMENTAL | LUM 600 milligram (mg) plus IVA 250 mg fixed-dose combination (FDC) tablet in the morning and IVA 250 mg film-coated tablet in the evening, up to Week 24. |
| LUM 400 mg q12h/ IVA 250 mg q12h | EXPERIMENTAL | LUM 400 mg plus IVA 250 mg FDC tablet in the morning and in the evening, up to Week 24. |
| Name | Type | Description |
|---|---|---|
| Lumacaftor Plus Ivacaftor Combination | DRUG | Fixed dose combination tablet, oral use |
| Ivacaftor | DRUG | Film-coated tablet, oral use |
| Placebo | DRUG | Matching placebo tablet |
Inclusion Criteria: * Signed informed consent form (ICF), and where appropriate, signed assent form. * Participants entering the Part A Treatment Cohort: Completed 24 weeks of study drug treatment in Study 103 or Study 104 and elect to enroll in Part A treatment cohort. * Participants entering the ...
Lumacaftor is used for cystic fibrosis, specifically in patients who are homozygous for the F508del CFTR mutation and in those who are homozygous or heterozygous for the F508del-CFTR mutation. It is a small molecule developed by Vertex Pharmaceuticals Incorporated for the respiratory therapeutic area.
Lumacaftor targets the cystic fibrosis transmembrane conductance regulator (CFTR) protein, specifically the F508del mutation. It is designed to correct the folding and trafficking of the defective CFTR protein, thereby improving its function at the cell surface.
Lumacaftor is developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker VRTX. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with cystic fibrosis.
Lumacaftor is in Phase 3 clinical development for cystic fibrosis. It is an investigational drug and has not yet been approved by regulatory authorities. The drug is being studied in patients who are homozygous or heterozygous for the F508del-CFTR mutation.
Lumacaftor has been studied in several clinical trials, including NCT01225211, a Phase 2 study in cystic fibrosis patients homozygous or heterozygous for the F508del-CFTR mutation, and NCT01768663, a Phase 1 drug-drug interaction study. All six trials are completed, with no active trials ongoing.
Lumacaftor is also known as VX-809. In clinical trials, it is often studied in combination with ivacaftor (VX-770). The drug is being developed by Vertex Pharmaceuticals for the treatment of cystic fibrosis caused by the F508del-CFTR mutation.