ELX/TEZ/IVA for Cystic Fibrosis
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a combination treatment called ELX/TEZ/IVA (also known as Trikafta or Kaftrio) for children with cystic fibrosis (CF), a condition affecting the lungs and digestive system. The goal is to understand how the body processes these drugs, assess their safety, and evaluate their effectiveness. The trial consists of two parts: participants receive the treatment in the morning and an additional drug in the evening, with adjustments based on early results. It suits children with a specific genetic mutation linked to CF. As a Phase 3 trial, this treatment is in the final step before FDA approval, offering a promising opportunity for children to access potentially life-changing therapy.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. Please consult with the trial coordinator or your doctor for guidance.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research shows that ELX/TEZ/IVA is generally easy for people to tolerate. Studies have found that those taking this treatment experience fewer lung flare-ups, improved lung function, and fewer hospital visits. Long-term research confirms that ELX/TEZ/IVA is safe and provides lasting benefits for people with cystic fibrosis (CF).
Research indicates that IVA, or ivacaftor, helps improve lung function and weight in people with CF. Ivacaftor is approved for use in babies as young as one month who have specific CF gene mutations, suggesting it is safe for young children. Studies in older patients also support its safety and effectiveness.
In summary, both treatments have strong safety records. They are well-researched and considered safe for people with CF, including young children.12345Why are researchers excited about this trial's treatments?
Researchers are excited about ELX/TEZ/IVA for cystic fibrosis because it targets the underlying cause of the condition rather than just managing symptoms. Unlike standard treatments that primarily address the symptoms or improve lung function, ELX/TEZ/IVA works by correcting the defective CFTR protein, which is the root cause of cystic fibrosis. This approach has the potential to significantly improve lung function and overall health, offering a more comprehensive solution for patients. Additionally, the combination of three active ingredients in ELX/TEZ/IVA provides a more robust treatment, potentially benefiting a broader range of cystic fibrosis patients compared to existing therapies.
What evidence suggests that this trial's treatments could be effective for cystic fibrosis?
Research has shown that Trikafta (ELX/TEZ/IVA) effectively treats cystic fibrosis. Studies found it improved lung function and lowered sweat chloride levels, both key disease markers. Patients experienced 63% fewer lung flare-ups and required fewer hospital visits. In this trial, participants will take ELX/TEZ/IVA in the morning and Ivacaftor (IVA) in the evening. Previous studies demonstrated that Ivacaftor reduced lung flare-ups by about 42% and hospital stays by 30%. Both treatments have provided lasting improvements in lung health and overall well-being for people with cystic fibrosis.23678
Are You a Good Fit for This Trial?
This trial is for young children aged 12 to less than 24 months with Cystic Fibrosis who have at least one F508del mutation or another mutation responsive to the study drugs. Children should not have other illnesses that could confuse the results or increase risks from the study drugs.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part A
Participants receive ELX/TEZ/IVA in the morning and IVA in the evening
Treatment Part B
Participants receive ELX/TEZ/IVA in the morning and IVA in the evening with doses based on Part A outcomes
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- ELX/TEZ/IVA
- IVA
ELX/TEZ/IVA is already approved in United States, European Union, Canada for the following indications:
- 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
- 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
- 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
Find a Clinic Near You
Who Is Running the Clinical Trial?
Vertex Pharmaceuticals Incorporated
Lead Sponsor
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