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LUM/IVA

Phase 3

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Sep 19, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials7
Total Enrollment549
FDA Designations
No designations recorded
Clinical Trials (7)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04235140Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment InitiationPHASE3 COMPLETED 52 — — Feb 24, 2020Aug 22, 2023Sep 19, 202423 United States, Canada
NCT03601637Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508delPHASE3 COMPLETED 61 — — Sep 7, 2018Oct 29, 2021Jan 6, 202327 United States, Canada
NCT03125395A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR MutationPHASE3 COMPLETED 57 — — May 12, 2017Jul 17, 2019Aug 7, 202020 United States, Canada
NCT02797132Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508delPHASE3 COMPLETED 62 — — May 1, 2016Sep 1, 2017Oct 30, 201820 United States, Canada
NCT02544451Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With IvacaftorPHASE3 COMPLETED 246 — — Aug 1, 2015Apr 1, 2020May 24, 202160 United States, Australia +7
NCT03625466A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508delPHASE2 COMPLETED 51 — — Aug 10, 2018Oct 7, 2021Nov 2, 20225 Germany
NCT03061331Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR MutationPHASE2 COMPLETED 20 — — Jan 31, 2017Oct 4, 2017Oct 2, 20182 Netherlands
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Study Endpoints
Primary Endpoints
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 120
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
Day 1 and Day 15
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
Pre-dose at Day 8 and Day 15
Part B : Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Day 1 up to Week 26
Safety as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 98
Part A: Pre-dose Concentration (Ctrough) of LUM and IVA
Day 15
Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 26
Treatment Period 1 (Treatment Cohorts): Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 100
Part 1: Absolute Change From Baseline in MRI Global Chest Score at Week 48
From Baseline at Week 48

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.

Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 8
Study Baseline, Through Week 8

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Secondary Endpoints
Absolute Change in Sweat Chloride (SwCl)
From Baseline at Week 96
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Day 1 up to Day 25
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
Pre-dose at Day 8 and Day 15
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LUM/IVAEXPERIMENTALParticipants 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/IVAEXPERIMENTALParticipants 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/IVAEXPERIMENTALParticipants 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)EXPERIMENTALPart 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/IVAEXPERIMENTAL -
Treatment Period 1: Placebo (PBO) to LUM/IVAEXPERIMENTAL -
Treatment Period 1: Observational CohortNO_INTERVENTION -
Treatment Period 2: LUM/IVAEXPERIMENTAL -
Part 1: PlaceboPLACEBO_COMPARATORParticipants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.
Part 1: LUM/IVAEXPERIMENTALParticipants 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/IVAEXPERIMENTALParticipants 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 1EXPERIMENTALLUM/IVA in Treatment Period 1; washout; placebo in Treatment Period 2
Treatment Sequence 2EXPERIMENTALPlacebo in Treatment Period 1; washout; LUM/IVA in Treatment Period 2
Interventions
NameTypeDescription
LUM/IVADRUGLUM/IVA granules for oral administration
LUMDRUGFixed Dose Combination (FDC) granules (LUM/IVA).
IVADRUGFDC granules (LUM/IVA).
PlaceboDRUGPlacebo matched to LUM/IVA for oral administration.
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Eligibility Criteria
Age Range12 Months — N/A
SexALL
Healthy VolunteersNo
Study Sites23

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...

Countries:United StatesCanadaAustraliaBelgiumDenmarkFranceGermanySwedenUnited KingdomNetherlands
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Competitive Landscape -Cystic Fibrosis 18 trials
CompanyTickerTrialsLead PhaseDrugs
Vertex Pharmaceuticals IncorporatedVRTX9PHASE3VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy
Sionna Therapeutics, Inc.SION2PHASE2SION-719, SION-451, SION-2222, SION-109
BiomX LtdPHGE1PHASE2BX004
4D Molecular Therapeutics, Inc.FDMT1PHASE24D-710
Arcturus Therapeutics Holdings, Inc.ARCT1PHASE2ARCT-032
Krystal Biotech, Inc.KRYS1PHASE1KB407
Illumina, Inc.ILMN1 — Undisclosed
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