2024-519657-11-00招募中2 期
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 Protocol Code: CRCFC-TAMOXI063
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 35
- 试验地点
- 1
- 主要终点
- 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.
研究概览
简要总结
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.
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •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)
排除标准
- •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
结局指标
主要结局
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.
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.
次要结局
- The secondary endpoints will evaluate the relative change from baseline to week 24 in ppFEV1
- The secondary endpoints will evaluate the number of pulmonary exacerbations up to week 24
- The secondary endpoints will evaluate the time to the first pulmonary exacerbation up to week 24
- The secondary endpoints will evaluate the number of hospitalizations for cystic fibrosis lasting > 24 hours
- The secondary endpoints will evaluate the time to the first hospitalization for cystic fibrosis
- the absolute change in the respiratory domain score of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) from baseline to week 24
- 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)
- changes in BMI from baseline to week 24
- changes in sputum microbiology at the beginning and end of the study
- changes in the amount of chloride measured with the sweat test at the beginning and end of the study
研究者
Marco Cipolli
Scientific
Azienda Ospedaliera Universitaria Integrata Verona
研究点 (1)
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