Study on Long-term Safety and Efficacy of Elexacaftor, Tezacaftor, and Ivacaftor in Children and Adults with Cystic Fibrosis Aged 2 Years and Older

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What is this study about?

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 is a combination of three medications: Elexacaftor, Tezacaftor, and Ivacaftor. These medications are taken together in a fixed-dose combination, which means they are combined into a single form for easier administration.

The purpose of the study is to evaluate how safe and tolerable this combination therapy is for individuals with Cystic Fibrosis who are 2 years of age and older. Participants in the study will receive the treatment over a period of time, and their health will be monitored to observe any side effects or changes in their condition. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment.

Throughout the study, participants will undergo regular health checks, including assessments of their lung function and other health indicators. The study aims to provide valuable information on the long-term use of this combination therapy, helping to improve treatment options for those living with Cystic Fibrosis.

1 joining the study

Upon joining the study, the legal representative or guardian must sign an informed consent form. This step ensures understanding of the study’s requirements and confirms the participant’s willingness to comply with the study protocol.

2 medication administration

Participants will receive a combination of medications: elexacaftor, tezacaftor, and ivacaftor. These medications are administered orally.

The specific products used include VX-445/VX-661/VX-770 fixed-dose combination granules and Kaftrio film-coated tablets. The dosage and form depend on the participant’s age and specific needs.

The medication regimen is designed to be stable throughout the study duration.

3 monitoring and assessments

Participants will undergo regular monitoring to evaluate the safety and tolerability of the medications. This includes checking for any adverse events, conducting laboratory tests, and performing standard 12-lead ECGs, vital signs, and pulse oximetry.

Secondary assessments include measuring changes in sweat chloride levels and lung function.

4 completion of study

The study is expected to conclude by June 20, 2025. Participants are required to remain on a stable cystic fibrosis treatment regimen until the study’s completion.

Who Can Join the Study?

The patient must have Cystic Fibrosis, a genetic condition that affects the lungs and digestive system.
The patient must be 2 years of age or older.
The patient’s legal representative or guardian must sign and date an informed consent form (ICF), which is a document that explains the study and confirms their agreement to participate.
The patient must be willing to stay on a stable Cystic Fibrosis treatment plan throughout the study.
The legal representative or guardian must understand the study’s requirements and ensure the patient follows them.
The patient must not have withdrawn consent from a previous related study.
The patient must meet at least one of the following conditions:
- Completed the study drug treatment in a previous related study.
- Had interruptions in the study drug treatment but did not stop it permanently and completed all required study visits.

Who Cannot Join the Study?

Patients who do not have cystic fibrosis cannot participate. Cystic fibrosis is a genetic condition that affects the lungs and digestive system.
Patients younger than 2 years old cannot participate.
Patients who are unable to take the study medication as directed cannot participate.
Patients with certain other medical conditions that might interfere with the study cannot participate. These conditions will be assessed by the study doctor.
Patients who are currently participating in another clinical trial cannot participate.
Patients who have had a recent lung transplant cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have a history of drug or alcohol abuse that might interfere with the study cannot participate.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Unxdesmvtbzuwkawhadka Etoqb Aer	Essen	Germany

Trial status

Country	Status	Recruitment Start
Germany	Not recruiting	14.04.2022

Trial locations

Investigated drugs:

Elexacaftor is a medication used in combination with other drugs to help improve the function of a protein that is defective in people with cystic fibrosis. It works by helping the protein move to the correct place in the cell, which can improve lung function and reduce symptoms of the disease.

Tezacaftor is another medication used alongside elexacaftor and ivacaftor. It helps the defective protein in cystic fibrosis patients to fold correctly, which can enhance its function and help alleviate the symptoms associated with the disease.

Ivacaftor is a medication that helps increase the activity of the protein affected by cystic fibrosis. It works by opening the protein channels, allowing for better movement of salts and water in and out of the cells, which can improve lung function and overall health in patients with cystic fibrosis.

Investigated diseases:

Cystic fibrosis

Cystic Fibrosis – Cystic fibrosis is a genetic disorder that affects the respiratory and digestive systems. It is caused by mutations in the CFTR gene, leading to the production of thick and sticky mucus. This mucus can clog the airways, causing breathing difficulties and frequent lung infections. Over time, the buildup of mucus can also affect the pancreas, hindering the digestion and absorption of nutrients. The disease is progressive, meaning symptoms typically worsen over time. It is most commonly diagnosed in childhood, but symptoms can vary widely among individuals.

Trial ID:

2024-515606-90-00

Protocol code:

VX20-445-112

NCT ID:

NCT05153317

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Long-term Safety and Efficacy of Elexacaftor, Tezacaftor, and Ivacaftor in Children and Adults with Cystic Fibrosis Aged 2 Years and Older

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?