Recent Updates
Recently added Catalysts

TEZ

Phase 3

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

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials5
Total Enrollment1,485

FDA Designations

No designations recorded

Clinical trial landscape

TEZ · 5 trials · 1 indication

Phase 3 4Phase 2 1
NCT03559062A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic FibrosisCystic Fibrosis
COMPLETED67 Analytics
NCT03537651A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR MutationCystic Fibrosis
COMPLETED130 Analytics
NCT02953314A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF)Cystic Fibrosis
COMPLETED83 Analytics
NCT02565914A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR MutationCystic Fibrosis
COMPLETED1,131 Analytics
PHASE3COMPLETED
A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
Cystic FibrosisUnlock trial analytics
PHASE3COMPLETED
A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation
Cystic FibrosisUnlock trial analytics
PHASE3COMPLETED
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of VX-661/Ivacaftor in Pediatric Subjects With Cystic Fibrosis (CF)
Cystic FibrosisUnlock trial analytics
PHASE3COMPLETED
A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation
Cystic FibrosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8
From baseline through Week 8

LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

Part A: 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 100
Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA
Day 1 and Day 14
Part A: Area Under the Concentration Versus Time Curve During Dosing Interval (AUCtau) of TEZ and IVA
Day 1 and Day 14
Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 28
Part A: 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 B: 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 C: 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 196
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
From first dose of Study Drug in the Treatment Period through Safety Follow-up Visit (Up to Day 57 for Part 1 and Day 85 for Part 2)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
From Baseline through Day 29

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 At Week 8
From baseline at Week 8
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8
From baseline through Week 8
Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit
From first dose of study drug up to safety follow-up visit (up to Week 12)
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
PlaceboOTHERParticipants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks.
TEZ/IVAEXPERIMENTALParticipants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
IvacaftorEXPERIMENTALParticipants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.
Part AEXPERIMENTALParticipants weighing \<25 kg received TEZ 50 mg once daily/IVA 75 mg q12h orally for 14 days. Participants weighing ≥25 kg received TEZ 50 mg once daily/IVA 150 mg q12h orally for 14 days.
Part BEXPERIMENTALParticipants weighing \<40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination orally once daily in the morning and IVA 75 mg orally once daily in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination orally once daily in the morning and IVA 150 mg orally once daily in the evening for 24 weeks.
Part 1: Placebo - Cohort 1A and 1B CombinedPLACEBO_COMPARATORParticipants received placebo matched to VX-440/TEZ/IVA as triple combination for 4 weeks.
Part 1 Cohort 1A: Triple Combination (TC)EXPERIMENTALParticipants received VX-440 200 milligram (mg) every 12 hours (q12h)/TEZ 100 mg once daily (qd)/IVA 150 mg q12h as triple combination for 4 weeks.
Part 1 Cohort 1B: TC Low DoseEXPERIMENTALParticipants received VX-440 200 mg q12h/TEZ 50 mg q12h/IVA 150 mg q12h as triple combination for 4 weeks.
Part 1 Cohort 1B: TC High DoseEXPERIMENTALParticipants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks.
Part 2: TEZ/IVAACTIVE_COMPARATORFollowing a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Part 2: TC-2EXPERIMENTALFollowing a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.

Interventions

NameTypeDescription
TEZ/IVADRUGParticipants weighing \<40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing ≥40 kg received TEZ 100 mg/IVA 150 mg FDC tablet.
IVADRUGParticipants weighing \<40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet.
PlaceboDRUGPlacebo matched to TEZ/IVA FDC
TEZDRUG -
VX-440DRUG -
Matched PlaceboDRUG -
Unlock Study Design Details

Eligibility Criteria

Age Range6 Years to 11 Years
SexALL
Healthy VolunteersNo
Study Sites27

Key Inclusion Criteria: * Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol). * Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height. * Participants with a screening LCI2.5 result ≥7.5. * Participants who are able to swallow...

Countries:AustraliaBelgiumDenmarkFranceGermanyIrelandPolandSwitzerlandUnited KingdomUnited StatesCanadaAustriaIsraelItalyNetherlandsSpainSweden
Unlock Eligibility Criteria

Frequently asked questions about TEZ

What is TEZ used for in Cystic Fibrosis?

TEZ is an investigational small molecule being developed for the treatment of Cystic Fibrosis. It is studied in combination with other therapies, such as ivacaftor, in patients with specific CFTR mutations, including the F508del-CFTR mutation. The drug is currently in Phase 3 clinical development.

Who makes TEZ?

TEZ is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker VRTX. Vertex is conducting clinical trials to evaluate the safety and efficacy of TEZ in combination with other cystic fibrosis treatments.

What phase is TEZ in?

TEZ is in Phase 3 clinical development for Cystic Fibrosis. It is an investigational drug and has not been approved by regulatory authorities. Vertex has completed multiple Phase 3 trials evaluating TEZ in combination with ivacaftor in patients with CFTR mutations.

What clinical trials is TEZ in?

TEZ has been studied in several completed clinical trials, including NCT02565914, a Phase 3 study of VX-661 (tezacaftor) with ivacaftor in patients with F508del-CFTR mutation, and NCT03537651, a Phase 3 long-term safety study of TEZ/IVA. Other trials include NCT02951182 and NCT02953314.

Is TEZ the same as VX-661?

Yes, TEZ is also known as VX-661. Clinical trials such as NCT02565914 and NCT02953314 refer to the drug as VX-661, while later studies like NCT03537651 use the abbreviation TEZ. Both names refer to the same investigational compound developed by Vertex Pharmaceuticals.

How does TEZ work?

TEZ is a small molecule that targets the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It is designed to correct the defective CFTR protein caused by mutations like F508del, improving its function and helping to restore chloride transport in patients with Cystic Fibrosis.