NL-OMON42435已完成2 期
Multi-center, randomized, double-blind, placebo-controlled 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 Riociguat in homozyg. deltaF508 CF pat.
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
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
- •9. Body mass index (BMI): * 16 and * 32 kg/ m² (calculated by dividing the patient*s weight by the square of his/ her height [kg/ m2])
- •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.
排除标准
- •1. Patients with Cystic Fibrosis with any background other than homozygous deltaF508
- •2. Patients receiving treatment with 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. Known history of solid organ transplantation
- •9. Known history of any form of pulmonary arterial hypertension;11. Known or suspected malignant tumors or a history of malignant tumors
- •12. Unstable liver disease as indicated by
- •a. bilirubin >2 times upper limit normal (ULN) and/ or hepatic transaminases >5
- •b. signs of severe hepatic insufficiency (e.g. impaired albumin synthesis with an
- •albumin < 32g/ L, hepatic encephalopathy > Grade 1a)
- •13. Patients with severe hepatic impairment (Child Pugh C) should be excluded
- •14. Recent evidence (within 12 months prior to inclusion) of distal intestinal obstruction
- •15. Patients with creatinine clearance <15 mL/ min or on dialysis need to be excluded.
- •16. Known history of cardiovascular disease unless stable and without therapy changes in
- •the previous 3 months
- •17. Known history of clinically relevant arterial hypotension or clinically relevant
- •orthostatic reactions (e.g. as indicated by syncopes, dizziness)
- •18. Venous/ arterial thromboembolic diseases (particularly deep vein thrombosis,
- •pulmonary embolism, stroke, myocardial infarction)
- •19. Known current thyroid disorders which require treatment (patients with an euthyroid
- •struma who do not need any treatment can participate)
- •20. Known hypersensitivity to the study medication (active substances or excipients of
- •the preparations)
- •21. Documented severe or clinically significant allergic reactions including anaphylaxis
- •22. Intolerance to lactose requiring strictly lactose-free diet and restriction to lactose-free
- •oral medicines (hereditary galactose intolerance, galactose-glucose malabsorption,
- •lactase deficiency)
- •23. Recent history (i.e. in the last 12 months prior to screening) of severe hypoglycemic
- •events in patients with severe Cystic Fibrosis diabetes
- •24. 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
- •25. Smoking (former smokers who have stopped smoking at least 3 months prior to the
- •first screening visit may be included)
- •26. Suspicion of drug or alcohol abuse or recent (i.e. within 2 years) history of drug,
- •medicine or alcohol abuse
- •27. Donation of blood or plasmapheresis after or within 4 weeks of signing the informed
- •consent form
- •28. 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),
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