Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Lumacaftor · 6 trials · 2 indications
AE: any untoward medical occurrence in a participant during the study; event does not necessarily have a causal relationship with treatment. This includes any newly occurring event/previous condition that has increased in severity/frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event, which falls into any of the following categories, regardless of its relationship to 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. TEAEs: AEs that started/ worsened on/after the start of study drug through the Safety Follow up Visit (4 weeks after the last dose of study drug). Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification.
The AUCtau is the area under the concentration versus time curve from time 0 to time tau, where tau is the time at the end of dosing interval.
AE: 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 the 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, inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first study drug dose to Week 26 were considered treatment-emergent.
AE: any untoward medical occurrence in a participant during study; irrespective of relationship with treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after informed consent. AE includes serious AEs (SAEs) as well as Non-SAEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to 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. Number of participants with AEs and SAEs are reported. AE that started at/after initial dosing of study drug, or increased in severity after initial dosing of study drug is considered treatment-emergent. Results are reported separately for monotherapy period (Period 1: Day 1 to Day 14) and combination therapy period (Period 2: Day 15 to Day 21).
Detailed description is provided in Outcome Measure 1. Results are reported separately for monotherapy period (Period 1: Day 1 to Day 28) and combination therapy period (Period 2: Day 29 to Day 56).
AEs and SAEs are defined in Outcome Measure 1.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. ppFEV1 (predicted for age, gender, and height) was calculated using the Hankinson method.
| Arm | Type | Description |
|---|---|---|
| Lumacaftor/Ivacaftor combination | EXPERIMENTAL | Lumacaftor 400 milligram (mg) and ivacaftor 250 mg combination tablet orally twice daily for 24 weeks. |
| Lumacaftor/Ivacaftor (LUM/IVA) | EXPERIMENTAL | Part A Cohort 1: Participants aged 6 through 8 years will receive LUM 200 milligram (mg) in fixed-dose combination with IVA 250 mg orally every 12 hours (q12h) for 14 days. Part A Cohort 2: Participants aged 9 through 11 years will receive LUM 200 mg in fixed-dose combination with IVA 250 mg orally q12h for 14 days. Part B: Participants aged 6 through 11 years will receive LUM 200 mg in fixed-dose combination with IVA 250 mg orally q12h for 24 weeks. |
| Cohort 1: Placebo | PLACEBO_COMPARATOR | Participants homozygous (HO) for the F508del-CF transmembrane conductance regulator gene (CFTR) mutation received lumacaftor matched placebo once daily (qd) (Day 1 through Day 14), followed by lumacaftor matched placebo qd in combination with ivacaftor matched placebo every 12 hours (q12h) (Day 15 through Day 21). |
| Cohort 1: LUM 200 mg qd/LUM 200 mg qd+IVA 150 mg q12h | EXPERIMENTAL | Participants homozygous for the F508del-CFTR mutation received 200 milligram (mg) of lumacaftor (LUM) qd (Day 1 through Day 14), followed by 200 mg of lumacaftor qd in combination with 150 mg of ivacaftor (IVA) q12h (Day 15 through Day 21). |
| Cohort 1: LUM 200 mg qd/LUM 200 mg qd+IVA 250 mg q12h | EXPERIMENTAL | Participants homozygous for the F508del-CFTR mutation received 200 mg of lumacaftor alone qd (Day 1 through Day 14), followed by 200 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 15 through Day 21). |
| Cohort 2 and 3: Placebo (HO and HE) | PLACEBO_COMPARATOR | Participants homozygous or heterozygous for the F508del-CFTR mutation received lumacaftor matched placebo qd (Day 1 through Day 28), followed by lumacaftor matched placebo in combination with ivacaftor matched placebo q12h (Day 29 through Day 56). |
| Cohort 2: LUM 200 mg qd/LUM 200 mg qd+IVA 250 mg q12h (HO) | EXPERIMENTAL | Participants homozygous for the F508del-CFTR mutation received 200 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 200 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56). |
| Cohort 2: LUM 400 mg qd/LUM 400 mg qd+IVA 250 mg q12h (HO) | EXPERIMENTAL | Participants homozygous for the F508del-CFTR mutation received 400 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 400 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56). |
| Cohort 2: LUM 600 mg qd/LUM 600 mg qd+IVA 250 mg q12h (HO&HE) | EXPERIMENTAL | Participants homozygous or heterozygous for the F508del-CFTR mutation received 600 mg of lumacaftor alone qd (Day 1 through Day 28), followed by 600 mg of lumacaftor qd in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56). |
| Cohort 3: LUM 400 mg q12h/LUM 400 mg q12h+IVA 250 mg q12h (HO) | EXPERIMENTAL | Participants homozygous for the F508del-CFTR mutation received 400 mg of lumacaftor alone q12h (Day 1 through Day 28), followed by 400 mg of lumacaftor q12h in combination with 250 mg of ivacaftor q12h (Day 29 through Day 56). |
| Cohort 4: Placebo | PLACEBO_COMPARATOR | Participants heterozygous for the F508del-CFTR mutation received lumacaftor matched placebo in combination with ivacaftor matched placebo q12h (Day 1 through Day 56). |
| Cohort 4: LUM 400 mg q12h+IVA 250 mg q12h | EXPERIMENTAL | Participants heterozygous for the F508del-CFTR mutation received 400 mg of lumacaftor q12h in combination with 250 mg of ivacaftor q12h (Day 1 through Day 56). |
| Part A | EXPERIMENTAL | A single dose of 2 fixed-dose combination tablets of 200mg lumacaftor/125mg ivacaftor (total of 400mg lumacaftor/250mg ivacaftor) in the fed state and fasted state with a 14 day washout period in between each dosing occasion |
| Part B | EXPERIMENTAL | A single dose of 3 fixed-dose combination tablets of 200mg lumacaftor/83mg ivacaftor (total of 600mg lumacaftor and 250mg ivacaftor) in the fed with a 14 day washout period in between dosing occasions |
| Treatment Group (Cohort 1) | EXPERIMENTAL | Subjects will take lumacaftor in combination with ivacaftor for 14 days. Beginning on Day 15, subjects will take lumacaftor in combination with ivacaftor and ciprofloxacin through Day 21. |
| Treatment Group (Cohort 2) | EXPERIMENTAL | Subjects will take lumacaftor in combination with ivacaftor for 14 days. Beginning on Day 15, subjects will take lumacaftor in combination with ivacaftor and itraconazole through Day 21. |
| Treatment Group (Cohort 3) | EXPERIMENTAL | Subjects will take lumacaftor in combination with ivacaftor for 14 days. Beginning on Day 15, subjects will take lumacaftor in combination with ivacaftor and rifampin through Day 24. |
| Treatment Group (Cohort 4) | EXPERIMENTAL | Subjects will take a single dose of lumacaftor in combination with ivacaftor on 3 occasions separated by 7 days. |
| Name | Type | Description |
|---|---|---|
| Lumacaftor | DRUG | - |
| Ivacaftor | DRUG | - |
| Lumacaftor Placebo | DRUG | Matching placebo tablet. |
| Ivacaftor Placebo | DRUG | Matching placebo tablet. |
| moxifloxacin hydrochloride | DRUG | - |
| Ciprofloxacin | DRUG | 750 mg taken every 12 hours |
| Itraconazole | DRUG | 200mg taken once daily |
| Rifampin | DRUG | 600mg taken once daily |
Inclusion Criteria: * Homozygous for the F508del-CFTR mutation; historical genotype must be documented in the participant's source documents. * Percent predicted FEV1 \<40 of adjusted for age, sex, and height at Screening Exclusion Criteria: * Participant currently receiving invasive mechanical v...
| 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 |
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.