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

Bayer AG0 sites63 target enrollmentMay 16, 2014

ConditionsCystic Fibrosis MedDRA version: 19.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

DrugsAdempas

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Cystic Fibrosis
Sponsor: Bayer AG
Enrollment: 63
Status: Active, not recruiting
Last Updated: 7 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

May 16, 2014

End Date

TBD

Last Updated

7 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Bayer AG

Eligibility Criteria

Inclusion Criteria

•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.

Exclusion Criteria

•1\. Patients with Cystic Fibrosis with any background other than homozygous delta F508 mutation
•2\. 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\. Known 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