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Study on the Safety and Tolerability of Tamoxifen in Cystic Fibrosis Patients Not Eligible for CFTR Modulator Therapy

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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 the safety and tolerability of a medication called tamoxifen citrate in individuals with cystic fibrosis. Cystic fibrosis is a genetic condition that affects the lungs and digestive system, leading to severe respiratory and digestive problems. The study is specifically for patients who do not have mutations that are currently eligible for treatment with CFTR modulator drugs, which are a type of medication used to treat certain forms of cystic fibrosis.

The purpose of the study is to evaluate how safe and well-tolerated tamoxifen citrate is for these patients. Participants in the study will take tamoxifen citrate in the form of a film-coated tablet, which is taken orally. The study will last for 24 weeks, during which time the participants will be monitored for any side effects or adverse events. The study will also look at changes in lung function, the number of lung infections, hospitalizations, and other health measures related to cystic fibrosis.

Throughout the study, researchers will collect data on various health indicators, such as lung function tests and the frequency of lung infections. They will also monitor the use of antibiotics and any changes in body weight and sweat chloride levels, which are important in understanding the impact of cystic fibrosis. The goal is to gather information that could help improve treatment options for patients with cystic fibrosis who are not currently eligible for CFTR modulator therapies.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, participants will be required to sign an informed consent form. This form confirms understanding of the study's purpose and procedures, and agreement to participate.

    Participants must meet specific criteria, such as being 18 years or older, having cystic fibrosis, and not having mutations eligible for CFTR modulator drugs.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to evaluate the participant's health status. This includes a pulmonary function test to measure lung capacity and function.

    Female participants will undergo a serum pregnancy test to ensure eligibility.

  3. Step 3

    Medication administration

    Participants will begin taking the study medication, tamoxifen citrate, in the form of a film-coated tablet called TAMOXENE 20 mg. The medication is taken orally.

    The dosage is one tablet per day, and the treatment will continue for a duration of 24 weeks.

  4. Step 4

    Regular monitoring

    Throughout the study, participants will attend regular check-ups to monitor health and any side effects. This includes tracking any adverse events and changes in health status.

    Participants will be asked to report any new symptoms or health changes to the study team.

  5. Step 5

    Mid-study evaluation

    At the midpoint of the study, participants will undergo a comprehensive evaluation to assess the impact of the medication on their health.

    This evaluation will include a repeat of the pulmonary function test and other health assessments.

  6. Step 6

    Final assessment

    At the end of the 24-week period, a final assessment will be conducted. This will include a thorough evaluation of the participant's health and any changes observed during the study.

    Participants will be asked to provide feedback on their experience and any side effects encountered.

  7. Step 7

    Post-study follow-up

    After completing the study, participants will have a follow-up period to monitor any long-term effects of the medication.

    Participants are advised to continue using non-hormonal contraception for two months after the study ends.

Who can join the trial?

11 criteria

  • Must have cystic fibrosis.
  • Must be able to perform reliable and repeatable lung function tests.
  • Must be able to communicate well with the study team, understand, and follow the study requirements.
  • Female participants must have a negative blood pregnancy test.
  • If sexually active, must use non-hormonal birth control methods during the study and for 2 months after the study ends.
  • Must sign a consent form agreeing to participate in the study and allow the use of personal data.
  • Must not have genetic mutations that are currently eligible for treatment with CFTR modulator drugs.
  • Must be 18 years old or older.
  • Must have a lung function test result called ppFEV1 between 40% and 90% of the expected value for their age, sex, and height before using a bronchodilator (a medicine that helps open the airways).
  • Must have a stable routine treatment for cystic fibrosis, including doses and medications like inhaled antibiotics, bronchodilators, anti-inflammatory drugs, inhaled steroids, and physiotherapy, for at least 28 days before starting the study.
  • Must have stable respiratory health for at least 3 weeks before starting the study medication.

Who cannot join the trial?

7 criteria

  • Patients who do not have cystic fibrosis cannot participate. Cystic fibrosis is a genetic condition that affects the lungs and digestive system.
  • Patients who have mutations that are currently eligible for therapy with modulator drugs cannot participate. Modulator drugs are medications designed to treat specific genetic mutations in cystic fibrosis.
  • Patients who are not within the age range of 3 to 18 years old cannot participate.
  • Patients who are not able to understand and comply with the study procedures cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have participated in another clinical trial within the last 30 days cannot participate.
  • Patients who have any other medical condition that the study doctors think might make it unsafe for them to participate cannot participate.
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Investigated drugs

Tamoxifen Citrate is a medication being studied for its potential use in treating cystic fibrosis. Cystic fibrosis is a condition that affects the lungs and other organs, leading to thick, sticky mucus buildup. This medication is not typically used for cystic fibrosis, but researchers are exploring whether it can help patients who do not have specific genetic mutations that are currently treatable with other drugs. The main goal of the study is to see if tamoxifen citrate is safe and well-tolerated by these patients.

What is already known about the treatment

Tamoxifen Citrate – Tamoxifen citrate is administered orally in the form of a tablet. It is currently being studied for its safety and tolerability in treating cystic fibrosis, particularly in patients who cannot use existing CFTR modulator drugs due to their specific genetic mutations. The main therapeutic indication for this study is cystic fibrosis. At the molecular level, tamoxifen works by binding to estrogen receptors, which can influence various cellular processes, although its exact mechanism in cystic fibrosis is still under investigation. It is classified pharmacologically as a selective estrogen receptor modulator (SERM).

Investigated diseases

Cystic Fibrosis – Cystic fibrosis is a genetic disorder that affects the respiratory, digestive, and reproductive systems. It is characterized by the production of thick and sticky mucus that can clog the airways and lead to respiratory 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. As cystic fibrosis progresses, individuals may experience frequent lung infections, persistent coughing, and difficulty breathing. The condition is chronic and requires ongoing management to address symptoms and complications.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2024-519657-11-00Protocol codeCRCFC-TAMOXI063Estimated enrolment35 patientsSponsorAzienda Ospedaliera Universitaria Integrata Verona

sourced from the EU Clinical Trials Register and site verification

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