EUCTR2013-004595-35-BE进行中（未招募）1 期
Multi-center phase 2 study to assess the safety, tolerability and early signs of efficacy of tid orally administered BAY63-2521 in adult deltaF508 homozygous Cystic Fibrosis patients - Early signs of efficacy study with BAY63-2521 in adult homozygous deltaF508 Cystic Fibrosis patients
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Bayer AG
- 入组人数
- 63
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Signed informed consent available before any study specific tests or procedures are performed
- •2. Patients must be at least 18 years of age at time of inclusion (i.e. upon signature of informed consent)
- •3. Patient diagnosed with Cystic Fibrosis according to standard criteria (i.e. either elevated sweat chloride content above 60 mmol/ L and/ or genetic testing)
- •4. Patient is homozygous for the deltaF508 mutation
- •5. Patient has a mild-to-moderate stage of lung disease as determined by FEV1 (FEV1 between 40 and 100% predicted)
- •6. Patient has a stable condition of lung disease (no ongoing or recent pulmonary exacerbation and no change in current treatment) within the last 4 weeks prior to screening
- •7. Ability and willingness to understand and follow study procedures for the entire study
- •8. Patients do not smoke. Patients with a history of smoking can be included, if they have refrained from smoking for the last 3 months. If a patients starts smoking during the study participation, he/ she needs to be excluded and considered to be a drop out
- •9. Body mass index (BMI): = 16 kg/ m²
- •Inclusion criterion valid for study part 1 only: 10. Women of childbearing potential must agree to use adequate contraception when sexually active. ‘Adequate contraception’ is defined as one highly effective form of contraception (intrauterine devices [IUD], contraceptive implants or tubal sterilization) or a combination of methods (hormone method with a barrier method). If a partner’s vasectomy is the chosen method of contraception or if a partner has documented azoospermia, a hormone or barrier method must be used in combination. Adequate contraception is required from the signing of the informed consent form up until 4 weeks after the last study drug administration.
- •Inclusion criterion valid for study part 2 only: 11. Women of childbearing
- •potential must agree to use adequate contraception when sexually
- •active. 'Adequate contraception' is defined as one highly effective form
- •of contraception (intrauterine devices [IUD], contraceptive implants or
- •tubal sterilization) or a combination of methods (hormone method with a
- •barrier method). For patients on Orkambi hormonal methods (including
- •hormonal oral contraceptives) cannot be accepted in this study. They
- •need to choose non-hormonal methods. If a partner's vasectomy is the
- •chosen method of contraception or if a partner has documented
- •azoospermia, a hormone or barrier method must be used in combination.
- •Adequate contraception is required from the signing of the informed
- •consent form up until 4 weeks after the last study drug administration.
- •Inclusion criterion valid for study part 2 only: 12. Patients receiving
- •Orkambi (Lumcaftor + Ivacaftor) as part of their standard care need to
- •be on stable Orkambi treatment for at least 3 months prior to screening
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 61
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •1. Patients with Cystic Fibrosis with any background other than homozygous deltaF508 mutation
- •Exclusion criterion 2 only valid for study part 1: Patients receiving treatment with Lumacaftor and/or Ivacaftor
- •3. Active state of hemoptysis or pulmonary hemorrhage, including those events managed by bronchial artery embolization. Also any history of moderate hemoptysis within the 3 months prior to inclusion
- •4. Any history of pneumothorax, bronchial artery embolization or massive hemoptysis. Massive hemoptysis being defined as acute bleeding >240 mL in a 24-hour period or recurrent bleeding >100 mL/ d over several days
- •5. A positive sputum culture for Burkholderia cenocepacia, Burkholderia dolosa, and/ or Mycobacterium absessus either currently or within the previous year.
- •6. Active allergic broncho-pulmonary aspergillosis
- •7. Current pulmonary exacerbation
- •8. nown history of solid organ transplantation
- •9. Known history of any form of pulmonary hypertension
- •10. Known or suspected malignant tumors or a history of malignant tumors
- •11. Unstable liver disease as indicated by
- •a.bilirubin >2 times upper limit normal (ULN) and/ or hepatic transaminases >5 times ULN
- •b.signs of severe hepatic insufficiency (e.g. impaired albumin synthesis with an albumin < 32g/ L, hepatic encephalopathy > Grade 1a)
- •12. Patients with severe hepatic impairment (Child Pugh C) should be excluded
- •13. Recent evidence (within 12 months prior to inclusion) of distal intestinal obstruction syndrome.
- •14. Patients with creatinine clearance <15 mL/ min or on dialysis need to be excluded.
- •15. Known history of cardiovascular disease unless stable and without therapy changes in the previous 3 months
- •16. Known history of clinically relevant arterial hypotension or clinically relevant orthostatic reactions (e.g. as indicated by syncopes, dizziness)
- •17. enous/ arterial thromboembolic diseases (particularly deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction)
- •18. Known current thyroid disorders which require treatment (patients with an euthyroid struma who do not need any treatment can participate)
- •19. Known hypersensitivity to the study medication (active substances or excipients of the preparations)
- •20. Documented severe or clinically significant allergic reactions including anaphylaxis or hives
- •21. Intolerance to lactose requiring strictly lactose-free diet and restriction to lactose-free oral medicines (hereditary galactose intolerance, galactose-glucose malabsorption, lactase deficiency)
- •22. Recent history (i.e. in the last 12 months prior to screening) of severe hypoglycemic events in patients with severe Cystic Fibrosis diabetes
- •23. Any medical disorder, condition, or history of such that would impair the patient's ability to participate or complete this study in the opinion of the investigator
- •24. Smoking (former smokers who have stopped smoking at least 3 months prior to the first screening visit may be included)
- •25. Suspicion of drug or alcohol abuse or recent (i.e. within 2 years) history of drug, medicine or alcohol abuse
- •26. Donation of blood or plasmapheresis after or within 4 weeks of signing the informed consent form
- •27. Concomitant use of the following medication: nitrates or nitric oxide donors (such as amyl nitrite) in any form, PDE 5 inhibitors (such as sildenafil, tadalafil, vardenafil), strong multi pathway CYP and p-gp/ BCRP inhibitors such as azole antimycotics (e.g. ketoconazole, itraconazole) or HI
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