Not recruitingRare disease

Study on the Safety and Tolerability of Elexacaftor, Tezacaftor, and Ivacaftor in Children with Cystic Fibrosis Aged 12 to Less Than 24 Months

Verified siteRegistered drugNo placebo

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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 Cystic Fibrosis, a genetic condition that affects the lungs and digestive system. The study will evaluate the safety and tolerability of a treatment combination known as ELX/TEZ/IVA, which includes the active substances Elexacaftor, Tezacaftor, and Ivacaftor. These substances are designed to help improve the function of a protein that is defective in people with Cystic Fibrosis. The trial will also involve the use of VX-770 granules and Kalydeco granules, which contain Ivacaftor, as well as a placebo.

The purpose of the study is to assess how well these treatments are tolerated by young children with Cystic Fibrosis, specifically those aged 12 to less than 24 months. Participants will receive the treatment orally in the form of granules. The study will last for a period of 24 weeks, during which the safety of the treatment will be closely monitored. This includes checking for any side effects, changes in vital signs, and other health indicators.

Throughout the study, researchers will also measure the levels of the treatment in the body and observe any changes in sweat chloride levels, which is a marker used to assess the effectiveness of the treatment in Cystic Fibrosis. The trial aims to provide valuable information on the safety and potential benefits of these treatments for young children with this condition.

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 subject's legally appointed representative, such as a parent or legal guardian, must provide consent by signing an informed consent form.
The subject must be between 12 to less than 24 months of age and have a confirmed diagnosis of cystic fibrosis.
Step 2
Genotype confirmation
The subject must have at least one F508del mutation in the CFTR gene or another mutation responsive to the medications being studied.
A central laboratory will confirm the subject's genotype on Day 1, although a historical genotype report may allow for earlier dosing.
Step 3
Treatment period
The subject will receive oral granules containing elexacaftor, tezacaftor, and ivacaftor as part of the treatment.
The treatment aims to evaluate the safety and tolerability of these medications in young subjects with cystic fibrosis.
Step 4
Medication regimen
The subject's parent or legal guardian must ensure that the subject remains on a stable cystic fibrosis medication regimen, excluding CFTR modulators, throughout the study period, which lasts up to 24 weeks.
Step 5
Safety and tolerability assessments
The study will include regular assessments of safety and tolerability through monitoring of adverse events, laboratory values, ECGs, vital signs, and pulse oximetry.
Step 6
Secondary assessments
Secondary assessments will include measuring the pharmacokinetic parameters of the medications and changes in sweat chloride levels from baseline through Week 24.

Who can join the trial?

7 criteria

The child's parent or legal guardian must sign a form agreeing to participate in the study.
The child must be between 12 months and less than 24 months old on the first day of the study.
The child must have a confirmed diagnosis of Cystic Fibrosis (CF), which is a genetic condition affecting the lungs and digestive system.
The child must have at least one specific genetic mutation called F508del in the CFTR gene, or another mutation that responds to the study medication. This will be confirmed by a laboratory test.
The child must have stable CF health at the start of the treatment, as determined by the study doctor.
The child's parent or legal guardian must agree to keep the child on a consistent CF medication plan, except for certain CF medications, until the end of the study.
The child's parent or legal guardian must understand the study's requirements and be able to ensure the child follows the study plan.

Who cannot join the trial?

6 criteria

Patients who have a history of severe allergic reactions to any of the study medications.
Patients who are currently participating in another clinical trial.
Patients who have a serious medical condition that might interfere with the study. A serious medical condition is a health problem that could make it unsafe for the patient to participate.
Patients who are pregnant or breastfeeding. This is because the study medications might affect the baby.
Patients who have had a recent organ transplant. An organ transplant is when a person receives a new organ, like a kidney or liver, from another person.
Patients who are unable to follow the study procedures. This means they might have difficulty understanding or doing what is required in the study.

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Verified sites

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

Deutsches Herzzentrum Berlin
Verified
Berlin, Germany
Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Responsive
Rotterdam, The Netherlands
Medizinische Hochschule Hannover
Verified
Hanover, Germany

Trial locations

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Investigated drugs

Elexacaftor, Tezacaftor, and Ivacaftor are medications used together to help treat cystic fibrosis. They work by improving the function of a protein that is defective in people with this condition. This combination helps to improve breathing and reduce the risk of lung infections. The study aims to understand how safe and tolerable these medications are for young children with cystic fibrosis.

What is already known about the treatment

Elexacaftor/Tezacaftor/Ivacaftor – This medication is administered orally and is used to treat cystic fibrosis. It is currently being studied in clinical trials to assess its safety and tolerability in young children. The main therapeutic indication is for cystic fibrosis, a genetic disorder affecting the lungs and digestive system. At the molecular level, this combination works by improving the function of the CFTR protein, which is defective in cystic fibrosis patients. It belongs to the pharmacological class of CFTR modulators, which help enhance the protein's activity to alleviate symptoms of the disease.

Investigated diseases

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, leading to 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.