Trial Condition(s):

Cystic Fibrosis

Early signs of efficacy study with Riociguat in adult homozygous delta F508 Cystic Fibrosis patients

Bayer Identifier:

17020

ClinicalTrials.gov Identifier:

NCT02170025

EudraCT Number:

2013-004595-35

Terminated/Withdrawn

Trial Purpose

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

Inclusion Criteria
- 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)
Exclusion Criteria
- 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

Trial Summary

Enrollment Goal
21
Trial Dates
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Phase
2
Could I receive a placebo?
Yes
Products
Adempas (Riociguat, BAY63-2521)
Accepts Healthy Volunteers
No

Where to Participate

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Locations
Status
LocationsStatus
Locations

Investigative Site

Toronto, Canada, M5B 1W8

Status
Terminated
 
Locations

Investigative Site

Berlin, Germany, 13353

Status
Terminated
 
Locations

Investigative Site

London, United Kingdom, SW3 6NP

Status
Terminated
 
Locations

Investigative Site

Belfast, United Kingdom, BT12 7AB

Status
Terminated
 
Locations

Investigative Site

Birmingham, United States, 35233-1711

Status
Completed
 
Locations

Investigative Site

St. Louis, United States, 63110

Status
Completed
 
Locations

Investigative Site

Denver, United States, 80206

Status
Completed
 
Locations

Investigative Site

PARIS, France, 75674

Status
Terminated
 
Locations

Investigative Site

ROTTERDAM, Netherlands, 3015 CE

Status
Terminated
 
Locations

Investigative Site

BRUXELLES - BRUSSEL, Belgium, 1090

Status
Terminated
 

Trial Design