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CFTR Modulator

VX-121 Combination Therapy for Cystic Fibrosis

Phase 3

Waitlist Available

Research Sponsored by Vertex Pharmaceuticals Incorporated

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Completed study drug treatment in a parent study VX20-121-102 (NCT05033080) and VX20-121-103 (NCT05076149); or had study drug interruption(s) in a parent study but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period in the parent study

Must not have

Pregnant or breast-feeding females

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trialstudies the safety and effectiveness of a drug for people with cystic fibrosis over the long-term.

Who is the study for?

This trial is for individuals with cystic fibrosis who previously participated in certain VX-121 studies and completed the treatment or visits, even if they had interruptions. It's not open to pregnant or breastfeeding women or those who couldn't tolerate the drug before.

What is being tested?

The study is testing the long-term safety and effectiveness of a combination therapy called VX-121/TEZ/D-IVA designed for people with cystic fibrosis to see how well it works over an extended period.

What are the potential side effects?

While specific side effects are not listed here, participants will be monitored for any adverse reactions to ensure the long-term safety and tolerability of the VX-121 combination therapy.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I completed or was part of the VX20-121-102 or VX20-121-103 study without permanently stopping the study drug.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2023 Phase 3 trial • 435 Patients • NCT05033080

32%

Infective pulmonary exacerbation of cystic fibrosis

26%

COVID-19

20%

Cough

17%

Nasopharyngitis

13%

Upper respiratory tract infection

12%

Nasal congestion

11%

Oropharyngeal pain

11%

Blood creatine phosphokinase increased

11%

Headache

10%

Pyrexia

10%

Sputum increased

Viral upper respiratory tract infection

Back pain

Nausea

Fatigue

Rhinorrhoea

Diarrhoea

Abdominal pain

Respiratory tract infection

Arthralgia

Abdominal distension

Haemoptysis

Influenza

Alanine aminotransferase increased

Sinusitis

Aspartate aminotransferase increased

Rash

Abdominal pain upper

Sinus congestion

Productive cough

Constipation

100%

80%

60%

40%

20%

Study treatment Arm

ELX/TEZ/IVA

VNZ/TEZ/D-IVA

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: VX-121/TEZ/D-IVAExperimental Treatment1 Intervention

Part A: Participants will receive VX-121/TEZ/D-IVA once daily for 96 weeks. Part B: Participants will receive VX-121/TEZ/D-IVA once daily for an additional 48 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Deutivacaftor

Not yet FDA approved

0 / 2Eligibility criteria met