Exploratory study to evaluate the safety and tolerability of tamoxifen citrate in the treatment of cystic fibrosis in patients without mutations currently eligible for therapy with CFTR modulator drugs
EU CTIS ID: 2024-519657-11-00
What this study is testing
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.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Subjects of both sexes, affected by cystic fibrosis, attending the CF Center in Verona
- Subjects able to perform reliable and reproducible pulmonary function test maneuvers
- Subjects able to communicate well with the investigator, understand, and comply with the study requirements
- Female subjects must have a negative serum pregnancy test
- Sexually active subjects able to follow the contraceptive methods defined within the protocol (non-hormonal contraception) during the study and for 2 months after study discontinuation
- Signed informed consent for participation in the study and for the processing of personal data
You likely can't join if
- Patients on any CFTR modulator therapy
- Hemoglobin levels < 9.0 g/dl
- Any surgical or medical condition that may significantly alter the absorption, distribution, metabolism, or excretion of drugs, or that may jeopardize the subject in case of participation in the study.
- History of immunodeficiency diseases
- History of drug or alcohol abuse
- History of any disease or condition that, in the investigator’s opinion, could confound the study results.
See the full eligibility criteria
Who can join
- Subjects of both sexes, affected by cystic fibrosis, attending the CF Center in Verona
- Subjects able to perform reliable and reproducible pulmonary function test maneuvers
- Subjects able to communicate well with the investigator, understand, and comply with the study requirements
- Female subjects must have a negative serum pregnancy test
- Sexually active subjects able to follow the contraceptive methods defined within the protocol (non-hormonal contraception) during the study and for 2 months after study discontinuation
- Signed informed consent for participation in the study and for the processing of personal data
- Patients who do not have mutations currently eligible for therapy with CFTR modulator drugs
- Age 18 years or older
- Predicted forced expiratory volume in 1 second (ppFEV1) ≥ 40% and ≤ 90% (of the predicted value for people of their age, sex, and height) before bronchodilator administration
- Stable routine CF therapy in terms of dose and medication (inhaled antibiotic cycles, bronchodilator, anti-inflammatory, inhaled corticosteroid, physiotherapy technique/schedule) within 28 days prior to Day 1
- Clinically stable respiratory disease within 3 weeks before Day 1 (first dose of study drug)
What rules you out
- Patients on any CFTR modulator therapy
- Hemoglobin levels < 9.0 g/dl
- Any surgical or medical condition that may significantly alter the absorption, distribution, metabolism, or excretion of drugs, or that may jeopardize the subject in case of participation in the study.
- History of immunodeficiency diseases
- History of drug or alcohol abuse
- History of any disease or condition that, in the investigator’s opinion, could confound the study results.
- Abnormal liver function, defined as ≥ 3 times the upper limit of normal (ULN) for any of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), total bilirubin
- Presence at baseline visit of endometrial polyps or vaginal symptoms (e.g., blood discharge, spotting, staining).
- Participation in a clinical study where an investigational drug was administered within 30 days prior to enrollment in the study or 5 half-lives of the study drug, whichever is longer
- Female patients who are pregnant or breastfeeding or who wish to become pregnant during the clinical study period and within one month after the end of the study.
- Female patients of childbearing potential who do not use adequate contraception. A woman is considered of childbearing potential (WOCBP), i.e., fertile, after menarche and until reaching post-menopause, unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy, and bilateral oophorectomy. A postmenopausal state is defined as the absence of menstruation for 12 months without an alternative medical cause. An elevated follicle-stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a postmenopausal state in women not using hormonal contraception or hormone replacement therapy. However, in the absence of 12 months of menstrual cycle, a single FSH measurement is not sufficient.
- Pulmonary exacerbations within 3 weeks before Day 1 (first dose of study drug)
- Changes in therapy for lung disease within 3 weeks before Day 1 (first dose of study drug).
- Family and/or personal history of thromboembolism and thromboembolic conditions up to 1st-degree relatives
- Documented hereditary thrombophilia (hypercoagulability), e.g., protein C, protein S, and antithrombin deficiency; factor V G169A Leiden, prothrombin G20210A (PT20210A), elevated factor VIII levels, hereditary dysfibrinogenemia.
- History of solid organ or hematopoietic transplant
- History of hypersensitivity to the study drug or drugs of similar chemical classes or any excipients
- History or presence of prolonged QT interval (QTcB > 450 msec)
- History of malignancy in any organ system (other than localized basal cell carcinoma of the skin) in the last 5 years
The study team makes the final eligibility decision.
Where it's taking place
- Italy
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Italy. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from EU CTIS. The study team decides eligibility, and your own doctor gives medical advice.