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

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What is this study about?

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.

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.

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.

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.

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.

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.

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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Medizinische Hochschule Hannover Hanover Germany

Other Sites

Site Name City Country Status
Rigshospitalet Copenhagen Denmark
Deutsches Herzzentrum Berlin Berlin Germany
Eufibeq Uycqfrsbuqwp Mrcxohy Cawltpr Ryjvuoqat (zkczuln Mqu Rotterdam The Netherlands
Uterktzbgldiubudzgzrz Ejqmc Aok Essen Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Denmark Denmark
Not recruiting
14.05.2024
Germany Germany
Not recruiting
14.05.2024
The Netherlands The Netherlands
Not recruiting
14.05.2024

Trial locations

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.

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.

Trial ID:
2023-503230-49-00
Protocol code:
VX22-445-122
Trial Phase:
Therapeutic confirmatory (Phase III)

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