A Phase 2 interventional study of Tamoxifen Citrate 20Mg Tab in Cystic Fibrosis - Complete, sponsored by Azienda Ospedaliera Universitaria Integrata Verona. Recruiting at 1 site in Italy. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-11-05.
Sponsored by Azienda Ospedaliera Universitaria Integrata Verona · Phase 2, Interventional, and Treatment
Among 86 studies in Cystic Fibrosis with a site in Italy — 8 open to participants now, 11 under way, 2 of them phase 3. Cystic Fibrosis · Italy.
Among the 86 studies in Cystic Fibrosis with a site in Italy, this one is the 61st largest by planned enrollment of the 73 interventional ones, is one of 3 that started in 2026, is one of 14 in phase 2, reaches primary completion later than 5 of the 10 other live ones, and was the 82nd registered.
1,619 studies on the registry are indexed under Cystic Fibrosis; 199 are open to participants now.
This study's planned enrollment of 35 is close to the median of 36 across 1,051 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →8 of the 86 studies in Cystic Fibrosis with a site in Italy are open now, in 12 countries. Each count lists the studies.
Of the 154 Cystic Fibrosis studies recruiting now, 95 (62%) set no upper age limit and 43 (28%) stop at 75 or younger; 84 take children under 18; 41 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →86 studies; the count open now beside each. This study's row is marked.
First in 1997; this study’s year is marked. 3 started in 2026.
Median enrollment 96 across the 73 interventional studies, 80 across the 13 observational. Of the 69 that completed or were terminated, 40 (58%) have results posted.
51 of 86 have an industry lead sponsor. The 10 most frequent:
Azienda Ospedaliera Universitaria Integrata Verona is the lead sponsor of 95 studies on the registry; 18 are open to participants now.
Azienda Ospedaliera Universitaria Integrata Verona has 1 study filed under Cystic Fibrosis — fewer than ten, so there is no comparison to draw.
6 of the studies in Cystic Fibrosis with a site in Italy that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05331183Phase 3Vertex Pharmaceuticals IncorporatedStudy to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del | 2027-04-06 |
| NCT07889973Istituto Giannina GasliniA Precision Medicine Approach to Assess Protein Function Loss and the Potential for CFTR Function Restoration in Cystic Fibrosis and Related Disorders | 2026-12-31 |
| NCT06413459Ospedale San RaffaeleIdentification of New Biological Markers for the Progression of Mycobacterium Abscessus-induced Lung Disease in Cystic Fibrosis | 2027-03-30 |
| NCT07559175Aimmune Nestlé Health Science US R&D, LLCA Study to Evaluate Safety and Explore Efficacy of New Lipase NHS7108 in Adult Participants With Exocrine Pancreatic Insufficiency. | 2027-06 |
| NCT07289035Azienda Ospedaliera Universitaria Integrata VeronaEarly Study on Tamoxifen Safety/Tolerability in Cystic Fibrosis Patients Unable to Use CFTR Modulators. | 2027-06-30 |
Among the 86 studies in Cystic Fibrosis with a site in Italy, from their registry updates:
The goal of this clinical trial is to evaluate the safety and tolerability of TMX in adults patients with cystic fibrosis who do not have mutations currently eligible for therapy with modulator drugs.
The main questions it aims to answer is:
. What medical problems do participants have when taking drug TMX?
Participants will:
Normal airways regulate the volume of airway surface liquid (ASL) through the activation of both cyclic adenosine monophosphate (cAMP) and Ca2+-dependent ion channels. In cystic fibrosis, the genetic defect causes a deficiency of cAMP-dependent CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) activity, leading to reduced Cl- and water secretion from airway epithelial cells and the consequent accumulation of mucus, facilitating bacterial and fungal infections. Women with CF have reduced survival compared with men with CF, although the mechanisms underlying this sex-related disadvantage are unknown. Despite the lack of CFTR, CF airways maintain a limited ability to regulate ASL volume, as ATP release, induced by breathing, activates purinergic pathways that increase intracellular Ca2+ concentration to stimulate an alternative Cl- secretion pathway. It has been hypothesized that estrogen might influence this pathway by reducing the ability of airway epithelia to adequately respond to nucleotides. They found that UTP-mediated Cl- secretion was reduced during periovulatory estrogen peaks in both CF and healthy women. Estrogen also inhibited Ca2+ signaling and ASL volume homeostasis in both non-CF and CF airway epithelia by attenuating Ca2+ influx. 17ß-estradiol inhibits the intracellular Ca2+ signaling, thus impairing the activity of calcium-activated chloride channels (CaCC). This suggests that antiestrogens, such as tamoxifen, could be beneficial in the treatment of CF lung diseases because they may increase Cl- secretion in the airways.
It has been demonstrated that TMX can restore CaCC function by inhibiting estrogen signaling. Furthermore, authors showed that TMX can directly activate CaCC regardless of estrogen signaling, therefore generating a significant amount of chloride current.
The results of these experiments indicate that:
TMX can increase CaCC currents through two mechanisms:
In conclusion, although effective therapies for CF have been already authorized in the European Union, the use of tamoxifen citrate is justified by the current scientific literature and preclinical data. Patients with CF should benefit from this treatment. Importantly, given its mechanism of action, TMX is expected to be beneficial for patients both with F508del CFTR mutation and other rare variants that still remain orphan of therapies. It follows that the European Commission has considered TMX treatment as a possible clinically relevant advantage for patients with CF.
Based on the results emerged from different studies, the European Commission has granted orphan designation (EU/3/17/1877) to GB Pharma S.r.l. for tamoxifen citrate for the treatment of cystic fibrosis.
Primary Objective The study aims to evaluate the safety and tolerability of TMX in patients with cystic fibrosis who do not have mutations currently eligible for therapy with modulator drugs.
Secondary Objectives
The study also aims to evaluate the effects of TMX on:
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patients will be included if they meet all the following inclusion criteria at Visit 1:
Exclusion Criteria:
Patients will be excluded if one or more of the following criteria are met at Visit 1:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
FEV1
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients will receive TMX 20 mg/day, in the morning. The choice of this dosage is based on pharmacokinetic and pharmacodynamic data in the literature in patients with breast cancer. Based on this scientific evidence, the dose of 20 mg/day is the minimum dose to ensure safety and efficacy of treatment. Women should start treatment with TMX between 8 and 12 days after the onset of menstruation. A longer screening period to allow for this timing will not be considered a protocol violation. Men will start treatment at V2 (day 1) and continue at home for 24 weeks. After screening, visits will be performed on the first day of TMX administration (baseline) and after 4, 8, 12, 16, and 24 weeks. Patients will discontinue TMX treatment at week 24 and will be followed for an additional 4 weeks for safety evaluation.
Drug: Tamoxifen Citrate 20Mg Tab
One tablet per day (20 mg/day), in the morning.
Also known as: TMX
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
The primary endpoint of this study will be evaluated by calculating between baseline and week 24 the incidence of serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) and treatment discontinuation due to AEs. In addition, frequency of specific, indication-relevant adverse events (e.g., thromboembolic events, elevation of liver enzymes, pulmonary exacerbations) and cumulative AE analyses, such as the number of AEs per patient, will be also evaluated.
Time frame: Calculating between baseline and week 24
Changes in respiratory function values
The relative change from baseline to week 24 in ppFEV1
Time frame: From baseline to week 24
Changes in pulmonary exacerbation
The number of pulmonary exacerbations up to week 24 and the time to the first pulmonary exacerbation up to week 24
Time frame: Up to week 24
Changes in hospitalizations
The number of hospitalizations for cystic fibrosis lasting \> 24 hours and the time to the first hospitalization for cystic fibrosis
Time frame: From baseline to week 24
Changes in quality of life
The absolute change in the respiratory domain score of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) (the more the value increases, the greater the perceived well-being)
Time frame: From baseline to week 24
Changes in antibiotic use
* the use of intravenous antibiotics during the study (total number of days of intravenous antibiotics for sino-pulmonary signs and symptoms up to week 24) * the use of oral antibiotics during the study (total number of days of oral antibiotics for sino-pulmonary signs and symptoms up to week 24)
Time frame: Up to week 24
Changes in BMI
Changes in BMI from baseline to week 24
Time frame: From baseline to week 24
Changes in sputum microbiology
Changes in sputum microbiology from screening to week 24
Time frame: From screening to week 24
Sweat test - Changes in amount of chloride
Changes in the amount of chloride measured with the sweat test
Time frame: At the beginning and end of the study
Plan to share: No
No publications or documents are linked to this record.
Started Jan 2026 (actual). Primary completion is estimated for Jun 2027, 17 months after the start.
Of 57 phase 2 interventional studies under Cystic Fibrosis, half reached primary completion within 21 months of starting (the median), counting those still running; a quarter within 9 and three quarters within 34. This study's planned 17 months is below that.
Since we started tracking on Sep 25, 2026, the dates moved once, on Nov 5, 2026: primary completion Apr 2026 → Jun 2027 and completion Feb 2027 → Dec 2027.
Earlier changes are on the record history on ClinicalTrials.gov ↗
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
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Studies of the drug tested here: Tamoxifen
Azienda Ospedaliera Universitaria Integrata Verona