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This Study Collects Information on the Safety of Inhaled Pegylated Adrenomedullin (PEG-ADM), How the Drug is Tolerated and How it Affects Patients Suffering From a Type of Lung Failure That Cause Fluid to Build up in the Lungs Making Breathing Difficult (ARDS)

Phase 2
Terminated
Conditions
Acute Respiratory Distress Syndrome
Interventions
Drug: BAY1097761 Active Dose 1
Drug: BAY1097761 Active Dose 2
Other: Placebo to BAY1097761
Registration Number
NCT04417036
Lead Sponsor
Bayer
Brief Summary

The study is composed of two parts. In part A of the study two active doses of inhaled pegylated adrenomedullin (PEG-ADM) will be compared regarding safety and efficacy to a substance that has no therapeutic effect (placebo) in order to find an optimal and safe of the study drug. In part B of the study the highest dose that is considered safe and has demonstrated efficacy will be taken forward to collect information how well patients suffering from Acute Respiratory Distress Syndrome (ARDS) respond to treatment with inhaled pegylated adrenomedullin (PEG-ADM) compared to treatment with placebo. ARDS is a type of lung failure that cause fluid to build up in the lungs making breathing difficult or impossible.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
90
Inclusion Criteria
  • ≥18 years of age at the time of inclusion into study.

  • Invasively mechanically ventilated acute respiratory distress syndrome [ARDS] patients (diagnosed according to Berlin definition of ARDS, including positive end-expiratory pressure [PEEP] of ≥5 cm H2O, X-ray (or CT scan) indicative of ARDS: bilateral opacities not fully explained by cardiac failure, fluid overload, lobar/lung collapse, effusions or nodules).

  • Initial diagnosis of mild, moderate or severe ARDS prior to study inclusion, with acute onset of ARDS within 1 week after suspected trigger factor of

    • Pneumonia
    • Aspiration
    • Sepsis
    • Pancreatitis
  • Prior to randomization, hypoxemia with PaO2:FiO2 ≤300 mmHg continuously observed for a period of ≥4 hours (with values of ≥2 arterial blood gas [ABG] analyses during that time, with the last value obtained timely (generally ≤3 hours) prior to randomization), under ventilation with minimum PEEP ≥8 cm H2O.

  • Time from first meeting the last diagnostic ARDS criterion (Berlin criteria) to randomization must be ≤48 hours.

  • For Study Part A: ARDS patients for whom measurements of extra-vascular lung water are regarded as medically indicated by the treating physician, and these measurements are planned as part of their clinical care, from Study Day 1 up to Study Day 7 (if then still intubated).

Exclusion Criteria
  • Any value of a PaO2:FiO2 ratio >300 mmHg within a time interval of 4 hours before randomization
  • Rescue therapy (e.g. inhalation of nitric oxide gas and/or inhalation of prostacyclin analogues, or extra corporeal membrane oxygenation [ECMO] / extra corporeal CO2 removal [ECCO2R]) already initiated at screening and/or Study Day 1 (prior to first dose of the study intervention)
  • Moribund participants not expected to survive 24 hours (clinical decision)
  • Expected duration of invasive mechanical ventilation less than 48 hours (clinical decision)
  • History of co-morbidities requiring long-term/home oxygen use (e.g. severe chronic obstructive pulmonary disease [COPD], pulmonary fibrosis) or non-invasive ventilation (except for sleep apnea management), or making weaning per se improbable (e.g. ALS, muscular dystrophy)
  • Smoke inhalation injury, extensive burns or trauma/head injury as concomitant condition
  • History of pneumectomy, lung lobectomy or lung transplant
  • Diffuse alveolar hemorrhage from vasculitis
  • Current lung malignancy (incl. lung metastasis), or other malignancy requiring chemotherapy or radiation within the last month
  • Chronic kidney disease with a history of renal replacement therapy (e.g. dialysis)
  • Chronic liver disease Child-Pugh Class C
  • Chronic heart failure NYHA IV
  • Known hypersensitivity to polyethyleneglycol (PEG, Macrogol)
  • Participation in other interventional studies involving pharmacological interventions, or biological or cell therapy interventions
  • Diagnosis of COVID-19 pneumonia within 6 weeks prior to study inclusion. History of SARS-CoV-2 infection (positive test based on nucleic acid amplification technology or positive antigen test) without COVID-19 pneumonia does not exclude patients

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Part A - Active Drug Dose 1BAY1097761 Active Dose 1Participants will receive Active Drug Dose 1 for a maximum of 14 days in study phase Part A
Part A - Active Drug Dose 2BAY1097761 Active Dose 2Participants will receive Active Drug Dose 2 for a maximum of 14 days in study phase Part A
Part A - PlaceboPlacebo to BAY1097761Participants will receive Placebo for a maximum of 14 days in study phase Part A
Part B - Active Drug DoseBAY1097761 Active Dose 1Participants will receive Active Drug 1 or 2 for a maximum of 14 days in study phase Part B
Part B - Active Drug DoseBAY1097761 Active Dose 2Participants will receive Active Drug 1 or 2 for a maximum of 14 days in study phase Part B
Part B - PlaceboPlacebo to BAY1097761Participants will receive Placebo for a maximum of 14 days in study phase Part B
Primary Outcome Measures
NameTimeMethod
VFS in Part B participantsAt Day 28

Ventilator-free survival (VFS, participants alive and not on invasive mechanical ventilation)

Secondary Outcome Measures
NameTimeMethod
Proportion of participants who still require invasive mechanical ventilation support in Part A and Part B participantsAt Day 28 and Day 60
Ventilator-free days (VFDs) in Part A and Part B participantsWithin Day 28 and Day 60
VFS in Part A and Part B participantsAt Day 60

Ventilator-free survival (VFS, participants alive and not on invasive mechanical ventilation)

All-cause mortality in Part A and Part B participantsAt Day 28, Day 60 and Day 90
VFS in Part A participantsAt Day 28

Ventilator-free survival (VFS, participants alive and not on invasive mechanical ventilation)

CUI in Part A participantsUp to 7 days

Clinical Utility Index (CUI) is a summary measure used to compare different treatments, the index score will range between 0 and 1.

Integrated analysis on VFS invoving all participants from Part A and Part BAt Day 28 and Day 60

Ventilator-free survival (VFS, participants alive and not on invasive mechanical ventilation)

Trial Locations

Locations (22)

Corporació Sanitària Parc Taulí

🇪🇸

Sabadell, Barcelona, Spain

Hospital de la Santa Creu i de Sant Pau

🇪🇸

Barcelona, Spain

Fakultni nemocnice v Motole

🇨🇿

Praha 5, Czechia

Center Hospitalier Michallon - Grenoble

🇫🇷

La Tronche, France

ASST Santi Paolo e Carlo

🇮🇹

Milano, Lombardia, Italy

Masaryk Hospital Usti n/L

🇨🇿

Usti nad Labem, Czechia

Klinikum der Stadt Köln gGmbH - Krankenhaus Merheim

🇩🇪

Köln, Nordrhein-Westfalen, Germany

Hôpital du Nord - Marseille

🇫🇷

Marseille Cedex 20, France

Cochin - Paris

🇫🇷

Paris, France

Centre Hospitalier Universitaire - Angers

🇫🇷

Angers Cedex 09, France

Hôpital Civil - Strasbourg

🇫🇷

Strasbourg, France

Istituto Clinico Humanitas - Humanitas Mirasole S.p.A.

🇮🇹

Milano, Lombardia, Italy

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

🇮🇹

Milano, Lombardia, Italy

Ciutat Sanitaria i Universitaria de la Vall d'Hebron

🇪🇸

Barcelona, Spain

Universitätsklinikum Schleswig-Holstein (UKSH)

🇩🇪

Kiel, Schleswig-Holstein, Germany

Universitätsklinikum AKH Wien

🇦🇹

Wien, Austria

Hôpital de la Pitié-Salpétrière

🇫🇷

Paris, France

Fakultni nemocnice Kralovske Vinohrady

🇨🇿

Praha 10, Czechia

Klinikum Oldenburg AöR

🇩🇪

Oldenburg, Niedersachsen, Germany

University Hospital of Wales

🇬🇧

Cardiff, United Kingdom

Guy's Hospital

🇬🇧

London, United Kingdom

Medizinische Universität Innsbruck

🇦🇹

Innsbruck, Tirol, Austria

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