Study of anakinra safety and effectiveness given by injection under the skin in patients with cystic fibrosis

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What is this study about?

This study focuses on patients with Cystic Fibrosis, a genetic condition that affects the lungs and other organs. The research evaluates a medication called anakinra, which is given as an injection under the skin. The study compares anakinra (sold under the brand name Kineret) with a placebo to determine if it can help improve lung function in people with Cystic Fibrosis.

The study uses a crossover design, which means participants receive both anakinra and placebo at different times during the study. The treatment involves daily injections of 100 mg anakinra or placebo for 28 days. The main purpose is to assess whether anakinra can improve breathing function in adults with Cystic Fibrosis, with the possibility of later including adolescents if the treatment proves effective in adults.

During the study, researchers will monitor lung function through various breathing tests. They will also check how well participants tolerate the medication by performing physical examinations and laboratory tests. Additional assessments include examining changes in lung structure using MRI scans, testing samples of mucus from the lungs, and evaluating quality of life through questionnaires.

1 Initial treatment period start

You will begin a 28-day treatment period receiving either anakinra or placebo through subcutaneous injection (injection under the skin).

The medication used will be either Kineret 100 mg/0.67 ml solution for injection in pre-filled syringe or a matching placebo.

2 Medical assessments during first period

Your lung function will be measured using the lung clearance index (LCI) test.

Regular physical examinations will be conducted.

Blood tests will be performed to monitor safety parameters.

You will complete a quality of life questionnaire called CFQ-R.

Sputum samples will be collected to analyze immune cells and inflammation markers.

3 Washout period

After the first 28-day treatment period, there will be a break period.

This period may last up to 12 weeks if any changes in your regular cystic fibrosis medication are needed.

4 Second treatment period

You will begin another 28-day treatment period.

If you received anakinra in the first period, you will now receive placebo, or vice versa.

The same medical assessments from the first period will be repeated.

5 Optional procedures

You may participate in an optional chest MRI study to examine lung structure and perfusion.

Additional sputum tests may be conducted to assess bronchial infection status and sputum characteristics.

Who Can Join the Study?

You must be at least 18 years old for the first group of participants. A second group aged 12-17 years may be included later if initial results are positive
You must have adequate bone marrow function showing:
– Normal white blood cell count (neutrophils >1.5 x 10^9/L)
– Normal platelet count (>100 x 10^9/L)
– Adequate hemoglobin levels (>9.0 g/dL)
Your liver function tests must be less than 3 times the normal upper limit
Your blood clotting tests must be within normal ranges
You must test negative for HIV, Hepatitis B, and Hepatitis C infections
If you are a woman who can become pregnant, you must have a negative pregnancy test
If you are sexually active and female, you must use effective birth control (abstinence, hormonal contraceptives, or IUD)
You must provide informed consent to participate
You must be fluent enough in German to complete study questionnaires
You must have a confirmed diagnosis of cystic fibrosis through one of these:
– High sweat chloride test (≥60 mEq/L)
– Two CF-causing genetic mutations
– Specific changes in nasal or rectal tissue electrical measurements
Your lung function test (FEV1) must be at least 50% of normal
Your lung clearance index must be 7.05 or higher
You must be able to perform breathing tests correctly
Your blood oxygen level must be 90% or higher when breathing room air
Your CF medications must have been stable for at least 4 weeks before starting the study

Who Cannot Join the Study?

Active lung infection or significant worsening of respiratory symptoms in the past 4 weeks
Known allergies or hypersensitivity to anakinra or its components
Pregnant women or those planning pregnancy during the study period
Breastfeeding mothers
Severe liver problems (as shown by blood tests)
Severe kidney problems (as shown by blood tests)
Active cancer or history of cancer in the past 5 years
Participation in another clinical trial within the past 30 days
Current use of immunosuppressive medications (medicines that lower the body’s immune response)
History of serious infections requiring hospitalization in the past 3 months
Active tuberculosis or positive tuberculosis test
History of blood disorders that significantly affect blood cell counts
Inability to perform required breathing tests for the study
Mental conditions that could interfere with following study procedures
Drug or alcohol abuse within the past year

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Universitaetsklinikum Heidelberg AöR	Heidelberg	Germany

Other Sites

Site Name	City	Country	Status
Ruhrlandklinik Westdeutsches Lungenzentrum Am Universitaetsklinikum Essen gGmbH	Essen	Germany
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany

Trial status

Country	Status	Recruitment Start
Germany	Recruiting	28.06.2022

Trial locations

Investigated drugs:

Anakinra

Anakinra is a medication that works by blocking a protein in the body that causes inflammation. It is given as an injection under the skin (subcutaneous injection). In this trial, it is being studied to see if it can help people with cystic fibrosis by reducing inflammation in their lungs and improving their lung function. The medication aims to help make breathing easier for patients with cystic fibrosis by potentially decreasing inflammation in their airways.

Investigated diseases:

Cystic fibrosis

Cystic Fibrosis – A genetic disorder that affects the cells producing mucus, sweat, and digestive fluids. In this condition, these normally thin and slippery fluids become thick and sticky, causing them to build up in various organs, particularly the lungs and digestive system. The disease primarily affects the lungs, where the thick mucus clogs the airways and traps bacteria, leading to repeated lung infections. It also affects the pancreas, preventing natural enzymes from helping the body break down and absorb food. The condition is present from birth and is caused by mutations in the CFTR gene, which controls salt and water balance in cells.

Trial ID:

2023-510376-31-00

Protocol code:

ANAKIN

NCT ID:

NCT03925194

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of anakinra safety and effectiveness given by injection under the skin in patients with cystic fibrosis

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?