What side effects are associated with this type of intervention?

CFTR modulators like Elexacaftor/Tezacaftor/Ivacaftor are generally well-tolerated but can cause side effects such as liver enzyme elevations, rash, and gastrointestinal disturbances including nausea and diarrhea. Other common treatments for CF, such as inhaled antibiotics (e.g., tobramycin, aztreonam) and mucolytics (e.g., hypertonic saline, DNase), can cause bronchospasm, throat irritation, and nephrotoxicity. It is important for patients to discuss these potential side effects with their healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

The FDA has approved several CFTR modulators for the treatment of Cystic Fibrosis, which target the underlying genetic defect in CF. Ivacaftor (Kalydeco) was the first CFTR modulator approved in 2012, specifically for patients with certain gating mutations. Lumacaftor/Ivacaftor (Orkambi) was approved in 2015 for patients with two copies of the F508del mutation. Tezacaftor/Ivacaftor (Symdeko) followed in 2018, offering an alternative for patients with the F508del mutation or other specific mutations. These treatments have significantly improved the management of CF by enhancing the function of the defective CFTR protein, leading to better respiratory and overall health outcomes for patients.

What other types of research exist?

Promising novel alternatives to Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis patients include next-generation CFTR modulators currently in clinical trials, such as VX-121/Tezacaftor/VX-561 and ABBV-3067/ABBV-2222. These combinations aim to enhance CFTR function further and may offer improved efficacy and safety profiles. Additionally, gene therapy approaches and mRNA-based treatments are being explored as potential future therapies for Cystic Fibrosis.

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CFTR Modulator

ELX/TEZ/IVA 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

Must not have

History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

No Placebo-Only Group

Summary

This trial is testing a combination of three medications to help people with cystic fibrosis. The medications work together to fix a broken protein in the body, which can improve lung function and overall health.

Who is the study for?

This trial is for people aged 2 and older with cystic fibrosis who have previously participated in a related study (VX20-445-111 Part B) without permanently stopping the study drug. Those with a history of not following treatment or procedures well, or intolerance to the study drug in the previous research, cannot join.

What is being tested?

The trial is looking at long-term safety and effectiveness of a combination medication (elexacaftor/tezacaftor/ivacaftor) for cystic fibrosis. It will assess how well patients tolerate this treatment over time and its impact on their condition.

What are the potential side effects?

While specific side effects are not listed here, participants may experience issues related to long-term use of elexacaftor/tezacaftor/ivacaftor. These could include digestive problems, respiratory symptoms, or other reactions based on individual tolerance.

Eligibility Criteria

Exclusion Criteria

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

Select...

I have had issues following treatment or study rules in the past.

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%

Blood creatine phosphokinase increased

11%

Oropharyngeal pain

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

Pivotal Trial

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

No Placebo-Only Group

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

Trial Design

1Treatment groups

Experimental Treatment

Group I: ELX/TEZ/IVAExperimental Treatment2 Interventions

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

IVA

2018

Completed Phase 3

~5260

ELX/TEZ/IVA

2019

Completed Phase 3

~3870

0 / 1Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Cystic Fibrosis treatments primarily aim to manage symptoms and improve quality of life. CFTR modulators, such as Elexacaftor/Tezacaftor/Ivacaftor, work by targeting the defective CFTR protein caused by genetic mutations in CF patients. These drugs help to enhance the function of the CFTR protein, improving chloride ion transport across cell membranes, which helps to hydrate and clear mucus from the airways. This is crucial for CF patients as it reduces the frequency of lung infections and improves lung function, thereby enhancing overall respiratory health and quality of life.