Study on the Effects of Dirocaftor, Posenacaftor, and Nesolicaftor for Adults with Cystic Fibrosis

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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 effectiveness and safety of three investigational medications: Dirocaftor, Posenacaftor, and Nesolicaftor. These medications are taken as hard capsules and are designed to help improve lung function in people with cystic fibrosis. The trial will also include a placebo, which is a substance with no active medication, to compare the effects of the investigational drugs.

The purpose of the study is to assess how well these medications work over an 8-week period in adults aged 18 and older who have cystic fibrosis with specific rare genetic mutations. Participants will be randomly assigned to receive either the investigational medications or a placebo. The study is designed as a crossover trial, meaning that participants will receive both the investigational medications and the placebo at different times during the study. This helps researchers understand the effects of the medications more clearly.

Throughout the study, participants will have regular check-ups to monitor their health and any changes in their condition. These check-ups will include tests to measure lung function, sweat chloride levels, and body weight, as well as assessments of overall health and any side effects. The study aims to provide valuable information on the potential benefits and safety of Dirocaftor, Posenacaftor, and Nesolicaftor for people living with cystic fibrosis.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria, including age, diagnosis of cystic fibrosis, and health stability.

Participants must have completed a prior study and meet health and lifestyle requirements, such as being a non-smoker.

2 treatment phase

The treatment phase involves taking medications in the form of hard capsules. These include posenacaftor, dirocaftor, and nesolicaftor, all administered orally.

The treatment lasts for 8 weeks, with regular monitoring of health indicators such as lung function, sweat chloride levels, and body weight.

3 monitoring and assessments

Throughout the study, assessments are conducted to monitor the effectiveness and safety of the treatment.

These assessments include measuring lung function, sweat chloride levels, body weight, and completing a questionnaire about respiratory health.

4 safety evaluations

Safety evaluations are performed to identify any adverse effects from the treatment.

These evaluations include laboratory tests, physical examinations, and monitoring of vital signs and heart activity.

5 completion of the study

At the end of the 8-week treatment period, final assessments are conducted to evaluate the overall impact of the treatment.

Participants’ data is analyzed to determine the effectiveness and safety of the medications.

Who Can Join the Study?

  • Must be a male or female who has completed the HIT-CF Organoid Study and is at least 18 years old on the date of giving consent to participate.
  • Must have a confirmed diagnosis of Cystic Fibrosis (CF). This can be shown by either a sweat chloride value of 60 mmol/L or higher, which is a test that measures the amount of salt in sweat, or by having two CF-causing genetic mutations along with chronic lung or digestive problems.
  • Must have a stable CF condition, meaning no major changes in health, as judged by the study doctor, for at least 28 days before starting the study.
  • Must have a lung function test result, called FEV1, between 40% and 90% of what is expected for a healthy person. FEV1 stands for Forced Expiratory Volume in one second, which measures how much air you can forcefully exhale in one second.
  • Must have a Body Mass Index (BMI) between 16 and 30. BMI is a number calculated from a person’s weight and height to assess if they are underweight, normal weight, overweight, or obese.
  • Must not smoke or use tobacco for at least 30 days before the screening and must agree not to smoke or use tobacco during the study.
  • Must be chosen by a team that knows the study details, either based on how their cells respond to the study treatment or by random selection.

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 who are not within the specified age range cannot participate. The study is for children aged 3 to 4 years old.
  • Patients who do not belong to the specified clinical trial group cannot participate. This group is determined by specific medical criteria.
  • Both male and female patients are eligible, so gender is not an exclusion factor.
  • Patients who are not considered part of a vulnerable population cannot participate. A vulnerable population includes individuals who may have additional health or social challenges.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Oncopole Claudius Regaud Toulouse France
Katholieke Universiteit te Leuven Leuven Belgium
Medizinische Hochschule Hannover Hanover Germany

Other Sites

Site Name City Country Status
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Universita’ Degli Studi Di Verona Verona Italy
Centre Hospitalier Universitaire De Nice Nice France
IRCCS Istituto Giannina Gaslini Genoa Italy
Deutsches Herzzentrum Berlin Berlin Germany
Hospital De Santa Maria E.P.E. Lisbon Portugal
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Fmbtsvrq necmfretn Mgmtz a Hyljsby Prague Czechia
Svkhdzineax Uxqhrdzunt Hugwzggibbuywcp Gyuzlcmywiltbvnbq Gothenburg Sweden
Hnpsjfwq Vfrg dksvcemx Barcelona Spain

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

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
01.05.2024
Czechia Czechia
Not recruiting
01.05.2024
France France
Not recruiting
01.05.2024
Germany Germany
Not recruiting
01.05.2024
Italy Italy
Not recruiting
01.05.2024
Portugal Portugal
Not recruiting
01.05.2024
Spain Spain
Not recruiting
01.05.2024
Sweden Sweden
Not recruiting
01.05.2024
The Netherlands The Netherlands
Not recruiting
01.05.2024

Trial locations

Dirocaftor is a medication being studied for its potential to help people with cystic fibrosis. It works by targeting specific proteins in the body that are affected by the disease, aiming to improve their function and help alleviate symptoms.

Posenacaftor is another medication included in the study for cystic fibrosis. It is designed to work alongside other treatments to enhance their effectiveness, focusing on improving the health of the lungs and other affected organs.

Nesolicaftor is part of the combination therapy being tested in the trial. It aims to support the other medications by further improving the function of proteins that are not working properly in people with cystic fibrosis, potentially leading to better overall health outcomes.

Investigated diseases:

Cystic Fibrosis – Cystic Fibrosis is a genetic disorder that primarily affects the lungs and digestive system. It is caused by mutations in the CFTR gene, leading to the production of thick and sticky mucus that can clog airways and trap bacteria, resulting in frequent lung infections. Over time, this can cause damage to the lungs and reduce lung function. The disease also affects the pancreas, leading to difficulties in digesting food and absorbing nutrients. Symptoms often include persistent coughing, frequent lung infections, and poor growth or weight gain despite a good appetite. The progression of the disease varies among individuals, with some experiencing more severe symptoms than others.

Trial ID:
2022-500410-26-01
Protocol code:
HIT-CF-001
Trial Phase:
Therapeutic exploratory (Phase II)

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