Trial Condition(s):
Early signs of efficacy study with Riociguat in adult homozygous delta F508 Cystic Fibrosis patients
Assessment of the safety, tolerability and early signs of efficacy of three times a day orally administered BAY63-2521 in adult delta F508 homozygous Cystic Fibrosis patients not on treatment with Orkambi
- Signed informed consent available before any study specific tests or procedures are performed - Patients must be at least 18 years of age at time of inclusion (i.e. upon signature of informed consent) - Patient diagnosed with Cystic Fibrosis according to standard criteria (i.e. either elevated sweat chloride content above 60 mmol/ L and/ or genetic testing) - Patient is homozygous for the deltaF508 mutation - Patient has a mild-to-moderate stage of lung disease as determined by FEV1 (FEV1 between 40 and 100% predicted) - 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 - Ability and willingness to understand and follow study procedures for the entire study - 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 - Body mass index (BMI): ≥ 16 kg/ m² (calculated by dividing the patient’s weight by the square of his/ her height [kg/ m2]) Inclusion criterion valid for study part 1 only: - 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 criteria valid for study part 2 only: - 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 - 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 (patients on Lumacaftor and/or Ivacaftor are excluded in part 1)
- Patients with Cystic Fibrosis with any background other than homozygous deltaF508 mutation - Exclusion criterion only valid for study part 1: Patients receiving treatment with Lumacaftor and/ or Ivacaftor - 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 - 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 - A positive sputum culture for Burkholderia cenocepacia, Burkholderia dolosa, and/ or Mycobacterium abscessus either currently or within the previous year - Active allergic broncho-pulmonary aspergillosis - Current pulmonary exacerbation - Known history of solid organ transplantation - Known history of any form of pulmonary hypertension - Clinically relevant deviations of the screened laboratory parameters from reference ranges outside of expected changes for Cystic Fibrosis patients, especially a hemoglobin value below 110 g/L or a creatinine clearance based on the Cockcroft-Gault formula < 15 ml/ min
Locations | Status | ||
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Locations Investigative Site Toronto, Canada, M5B 1W8 | Status Terminated | Contact Us: E-mail: [email protected] Phone: +49 30 300139003 | |
Locations Investigative Site Berlin, Germany, 13353 | Status Terminated | Contact Us: E-mail: [email protected] Phone: +49 30 300139003 | |
Locations Investigative Site London, United Kingdom, SW3 6NP | Status Terminated | Contact Us: E-mail: [email protected] Phone: +49 30 300139003 | |
Locations Investigative Site Belfast, United Kingdom, BT12 7AB | Status Terminated | Contact Us: E-mail: [email protected] Phone: +49 30 300139003 | |
Locations Investigative Site Birmingham, United States, 35233-1711 | Status Completed | Contact Us: E-mail: [email protected] Phone: +49 30 300139003 | |
Locations Investigative Site St. Louis, United States, 63110 | Status Completed | Contact Us: E-mail: [email protected] Phone: +49 30 300139003 | |
Locations Investigative Site Denver, United States, 80206 | Status Completed | Contact Us: E-mail: [email protected] Phone: +49 30 300139003 | |
Locations Investigative Site PARIS, France, 75674 | Status Terminated | Contact Us: E-mail: [email protected] Phone: +49 30 300139003 | |
Locations Investigative Site ROTTERDAM, Netherlands, 3015 CE | Status Terminated | Contact Us: E-mail: [email protected] Phone: +49 30 300139003 | |
Locations Investigative Site BRUXELLES - BRUSSEL, Belgium, 1090 | Status Terminated | Contact Us: E-mail: [email protected] Phone: +49 30 300139003 |
Multi-center phase 2 study to assess the safety, tolerability and early signs of efficacy of tid orally administered BAY63-2521 in adult delta F508 homozygous Cystic Fibrosis patients
Trial Type:
Interventional
Intervention Type:
Drug
Trial Purpose:
Treatment
Allocation:
Randomized
Blinding:
Double Blind
Assignment:
Parallel Assignment
Trial Arms:
2