VX-121 Combination Therapy for Cystic Fibrosis

Recruiting at 138 trial locations

Overseen By Medical Information

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Vertex Pharmaceuticals Incorporated

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

435 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial tests a combination of three drugs to help cystic fibrosis patients with a specific genetic mutation. The drugs aim to fix a faulty protein in their lungs, improving their ability to breathe. Trikafta is a combination of three drugs that target the F508del mutation in the CFTR gene.

Eligibility Criteria

This trial is for people with cystic fibrosis who have one F508del mutation and another mutation that minimally functions. They should be able to breathe out at least 40% of the air in their lungs in one second, but not more than 80-90%, depending on current treatments. People with severe liver problems, certain lung infections, or those pregnant or breastfeeding cannot join.

Inclusion Criteria

Your lung function, measured by FEV1, should be between 40% and 80% if you are not currently taking ELX/TEZ/IVA.

Your lung function, measured by FEV1, should be between 40% and 90% of the expected value based on your age, sex, and height if you are taking ELX/TEZ/IVA therapy.

You have a specific combination of genetic mutations called F508del and minimal function mutation.

Exclusion Criteria

You have a lung infection caused by certain germs that can make your breathing get worse quickly.

Pregnant or breast-feeding females

You have severe liver problems or high blood pressure in the liver.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Run-in

Participants undergo a run-in period with ELX/TEZ/IVA for 4 weeks

4 weeks

Treatment

Participants receive VX-121/TEZ/D-IVA or ELX/TEZ/IVA for 52 weeks

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• ELX/TEZ/IVA
• IVA
• Placebo (matched to ELX/TEZ/IVA)
• Placebo (matched to IVA)
• Placebo (matched to VX-121/TEZ/D-IVA)
• VX-121/TEZ/D-IVA

Trial Overview The study tests VX-121/TEZ/D-IVA's effectiveness and safety against cystic fibrosis compared to a placebo and other therapies like ELX/TEZ/IVA. Participants will randomly receive either the test drug combination or a matching placebo.

Participant Groups

Group I: VX-121/TEZ/D-IVA Experimental Treatment 3 Interventions

Following ELX/TEZ/IVA run-in period of 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.

Group II: ELX/TEZ/IVA Active Control 3 Interventions

Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, participants received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks.

ELX/TEZ/IVA is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Trikafta for:

• Cystic fibrosis in patients aged 2 years and older with at least one copy of the F508del mutation in the CFTR gene or another mutation responsive to treatment

Approved in European Union as Kaftrio for:

• Cystic fibrosis in patients aged 2 years and older with at least one copy of the F508del mutation in the CFTR gene or another mutation responsive to treatment

Approved in Canada as Trikafta for:

• Cystic fibrosis in patients aged 2 years and older with at least one copy of the F508del mutation in the CFTR gene or another mutation responsive to treatment

Who Is Running the Clinical Trial?

Vertex Pharmaceuticals Incorporated

Lead Sponsor

Trials

267

Recruited

36,100+

Dr. David Altshuler

Vertex Pharmaceuticals Incorporated

Chief Medical Officer since 2020

MD, PhD

Dr. Reshma Kewalramani

Vertex Pharmaceuticals Incorporated

Chief Executive Officer since 2020

MD, trained in internal medicine and nephrology