Not recruitingRare disease

Study on Long-term Safety and Efficacy of Elexacaftor, Tezacaftor, and Ivacaftor for Cystic Fibrosis in Patients Aged 12 Months and Older

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the long-term safety and effectiveness of a treatment for Cystic Fibrosis, a genetic disorder that affects the lungs and digestive system. The treatment being tested includes a combination of three medications: elexacaftor, tezacaftor, and ivacaftor. These medications are designed to help improve the function of a protein that is defective in people with Cystic Fibrosis.

The purpose of the study is to evaluate how well this combination of medications works over a long period and to monitor any potential side effects. Participants in the study will take the medications in the form of granules, which are taken orally. The study will last for up to 96 weeks, during which participants will regularly visit the clinic for check-ups and assessments. These assessments will include monitoring vital signs, conducting laboratory tests, and checking heart function using an ECG, which is a test that records the electrical activity of the heart.

Throughout the study, participants will be closely observed to ensure their safety and to gather information on how the treatment affects their health. The study will also measure changes in sweat chloride levels, which is an indicator of how well the treatment is working. This trial is open-label, meaning that both the participants and the researchers know which treatment is being administered. The study is intended for individuals aged 12 months and older who have Cystic Fibrosis and have previously participated in a related study.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the legally appointed representative will sign and date the informed consent form. This ensures understanding of the study requirements and confirms willingness to participate.
Step 2
Initial assessment
An initial assessment will be conducted to confirm eligibility. This includes reviewing previous study participation and ensuring a stable cystic fibrosis treatment regimen is in place.
Step 3
Medication administration
Participants will receive elexacaftor, tezacaftor, and ivacaftor in the form of granules or oral solution. The specific product names include Kaftrio and Kalydeco. The dosage and frequency will be determined by the study protocol and communicated by the study team.
Step 4
Ongoing monitoring
Throughout the study, regular monitoring will occur to assess safety and tolerability. This includes checking for any adverse events, conducting laboratory tests, and measuring vital signs.
Step 5
Sweat chloride test
A sweat chloride test will be performed to measure changes in chloride levels. This test helps evaluate the effectiveness of the treatment.
Step 6
Completion of study
The study is expected to continue until August 2027. Upon completion, a final assessment will be conducted to evaluate the long-term safety and efficacy of the treatment.

Who can join the trial?

6 criteria

The patient's legally appointed and authorized representative must sign and date the informed consent form. This means someone who is legally allowed to make decisions for the patient agrees to the study.
The legal representative or guardian must understand the study requirements and be able to ensure the patient follows the study plan.
The patient must not have withdrawn consent from the previous related study.
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The patient must meet at least one of the following conditions:
- Completed the study drug treatment in the previous study.
- Had temporary breaks in the study drug treatment in the previous study but did not stop permanently and completed all required visits up to the last scheduled visit of the treatment period.
- The patient must be willing to continue with a stable treatment plan for Cystic Fibrosis throughout the study.

Who cannot join the trial?

8 criteria

Patients who do not have Cystic Fibrosis cannot participate.
Patients who are not within the specified age range cannot participate.
Patients who are not willing to follow the study procedures cannot participate.
Patients who have other medical conditions that might interfere with the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who are taking medications that might interfere with the study cannot participate.
Patients who have participated in another clinical trial recently cannot participate.
Patients who have a history of non-compliance with medical treatments cannot participate.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Charite Universitaetsmedizin Berlin KöR
Berlin, Germany
Responsive
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Rotterdam, The Netherlands
Verified
Medizinische Hochschule Hannover
Hanover, Germany

Trial locations

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Investigated drugs

Elexacaftor
is a medication used to help people with cystic fibrosis, a genetic condition that affects the lungs and digestive system. It works by improving the function of a protein that is faulty in people with this condition, helping to reduce symptoms and improve breathing.
Tezacaftor
is another medication used in the treatment of cystic fibrosis. It helps the faulty protein in the body work better, which can lead to improved lung function and overall health in people with this condition.
Ivacaftor
is a medication that helps improve the function of the protein affected by cystic fibrosis. It works by helping the protein stay open longer, which can improve lung function and reduce symptoms in people with cystic fibrosis.

What is already known about the treatment

Elexacaftor/Tezacaftor/Ivacaftor – This medication is taken orally and is used to treat cystic fibrosis in patients aged 12 months and older. It is currently being studied in a Phase 3 clinical trial to assess its long-term safety and effectiveness. The medication works by targeting specific proteins in the body to help improve lung function and reduce symptoms of cystic fibrosis. It belongs to a class of drugs known as CFTR modulators, which help correct the function of the defective protein in cystic fibrosis patients.

Investigated diseases

Cystic Fibrosis – Cystic Fibrosis is a genetic disorder that affects the respiratory, digestive, and reproductive systems. It is characterized by the production of thick and sticky mucus that can clog the airways and lead to respiratory issues. Over time, this mucus buildup can cause persistent lung infections and damage to the lungs. In the digestive system, the thick mucus can block the ducts in the pancreas, preventing digestive enzymes from reaching the intestines and leading to malnutrition. The disease progresses with increasing respiratory difficulties and digestive problems. It is a lifelong condition that requires ongoing management to address the symptoms and complications.