What side effects are associated with this type of intervention?

Common treatments for cystic fibrosis include CFTR modulators like ivacaftor, lumacaftor, and tezacaftor, as well as antibiotics such as tobramycin and aztreonam. CFTR modulators can cause side effects like liver enzyme elevations, cataracts, and respiratory symptoms. Inhaled antibiotics, used to manage chronic Pseudomonas aeruginosa infections, may lead to bronchospasm, cough, and throat irritation. Long-term use of these antibiotics can also result in ototoxicity and nephrotoxicity.

What prior FDA approvals exist?

The FDA has approved several CFTR modulators for the treatment of cystic fibrosis, targeting the underlying genetic defect in the CFTR protein. Key drugs include Ivacaftor (Kalydeco), approved in 2012 for patients with specific CFTR mutations, and Lumacaftor/Ivacaftor (Orkambi), approved in 2015 for patients with two copies of the F508del mutation. Additionally, Tezacaftor/Ivacaftor (Symdeko) was approved in 2018, and Elexacaftor/Tezacaftor/Ivacaftor (Trikafta) received approval in 2019 for patients with at least one F508del mutation. These treatments have significantly improved outcomes for many cystic fibrosis patients by enhancing the function of the defective CFTR protein.

What other types of research exist?

Promising alternatives to VX-522 for cystic fibrosis treatment include VX-770 (Ivacaftor) and VX-809 (Lumacaftor). These modulators have shown efficacy in improving CFTR function and lung function in clinical trials. Combination therapies, such as VX-770 with VX-809, have also demonstrated synergistic effects in restoring CFTR function and could be considered for treatment in 2024.

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mRNA therapy

VX-522 mRNA Therapy for Cystic Fibrosis

Q: What is the mechanism of action of this treatment?

CFTR modulators, such as those being studied in the VX-522 trial, target the defective CFTR protein responsible for regulating chloride and sodium ion flow across cell membranes. By improving CFTR function, these treatments help hydrate and clear mucus from the airways, reduce inflammation, and enhance lung function. This is crucial for CF patients as it addresses the root cause of the disease, leading to improved respiratory health and quality of life.

Phase 1 & 2

Recruiting

Research Sponsored by Vertex Pharmaceuticals Incorporated

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing VX-522, a new treatment for adults with cystic fibrosis who don't respond to current treatments. The treatment aims to fix or improve the faulty gene causing the disease, potentially reducing symptoms and improving health.

Who is the study for?

This trial is for adults with cystic fibrosis who have specific CFTR gene mutations not treatable with current modulator therapies. Participants must have stable disease, a minimum lung function (FEV1 ≥40%), weigh over 50 kg, and have a BMI under 30. Excluded are those with low oxygen saturation (<94% on room air), uncontrolled asthma in the past year, any organ transplants, or moderate to severe liver issues.

What is being tested?

The study tests VX-522 mRNA therapy's safety and how well participants tolerate it. It targets individuals aged 18+ whose cystic fibrosis results from certain CFTR gene mutations that don't respond to existing treatments.

What are the potential side effects?

While the side effects of VX-522 are being studied in this trial and aren't fully known yet, common risks may include immune reactions, injection site discomfort, fatigue, fever-like symptoms or potential respiratory complications.

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

Awards & Highlights

No Placebo-Only Group

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

Trial Design

3Treatment groups

Experimental Treatment

Group I: Single Ascending Dose (SAD)Experimental Treatment1 Intervention

Participants grouped into different cohorts will receive a single ascending dose of VX-522.

Group II: Multiple Ascending Dose (MAD) Arm 1: VX-522Experimental Treatment1 Intervention

Participants grouped into different cohorts will receive multiple ascending doses of VX-522 in treatment arm 1 (T1).

Group III: MAD Arm 2: VX522+ IVAExperimental Treatment2 Interventions

Following run-in period with ivacaftor (IVA), participants grouped into different cohorts will receive multiple ascending doses of VX-522 with IVA in treatment arm (T2).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

IVA

2018

Completed Phase 3

~5260

Do I qualify?Apply below to find out

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

CFTR modulators, such as those being studied in the VX-522 trial, target the defective CFTR protein responsible for regulating chloride and sodium ion flow across cell membranes. By improving CFTR function, these treatments help hydrate and clear mucus from the airways, reduce inflammation, and enhance lung function. This is crucial for CF patients as it addresses the root cause of the disease, leading to improved respiratory health and quality of life.

Cellular heterogeneity of CFTR expression and function in the lung: implications for gene therapy of cystic fibrosis.