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Immunosuppressive Treatment in Chronic Virus-Negative Inflammatory Cardiomyopathy

Registration Number
NCT05570409
Lead Sponsor
LMU Klinikum
Brief Summary

Evaluating Immunosuppressive treatment (Mycophenolate mofetil and prednisolon compared to placebo) for 6 months in patients with chronic virus- Negative Inflammatory cardiomyopathy - a multicenter, randomized, double-blind, placebo-controlled trial.

Detailed Description

Inflammatory cardiomyopathy constitutes a relevant part of the cohort of non-dilated left ventricular cardiomyopathy / dilated cardiomyopathy (DCM) and is associated with adverse outcome. Urgent medical needs remain with respect to the therapeutic options for inflammatory cardiomyopathy. So far, no specific therapy for patients with inflammatory cardiomyopathy is available. Existing data on immunosuppression for inflammatory cardiomyopathy is preliminary and needs further validation by larger randomized, controlled, multicenter trials.

Patients with biopsy-proven virus-negative inflammatory dilated or non-dilated left ventricular cardiomyopathy and moderate to severe deterioration of cardiac function despite optimal medical treatment (OMT) for heart failure (HF) will be randomized (1:1) in a double-blinded way to Mycophenolate mofetil (MMF) 1g bid and prednisolone at initially 1mg/kg in a step-down regime for 6 months or placebo. The clinical benefit will be measured with respect to absolute increase in LVEF (metric and binary co-primary endpoints assessed by MRI core lab) of immunosuppressive treatment with MMF and prednisolone compared to placebo at 12 months follow-up.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
130
Inclusion Criteria
  1. Age ≥18 years
  2. Medical therapy for HF for ≥3 months and <10 years according to current guideline recommendations
  3. Persistent reduction of LVEF <50% on a routine echocardiographic evaluation (Simpson's biplane) not older than 1 month at time of inclusion
  4. EMB with immunohistochemical evidence of lymphocytic myocarditis defined as ≥14 leukocytes/mm2 including up to 4 monocytes/mm2 with the presence of CD3 positive T-lymphocytes ≥7 cells/mm2 and increased MHC-II expression as approved by the histopathology core lab
  5. Absence of established cardiotropic virus infection in EMBs (i.e. enteroviruses, HHV-6, EBV, CMV, adenoviruses, parvovirus B19 >500 copies) as approved by the histopathology core lab
  6. Negative pregnancy test and the use of a highly effective contraceptive measure in women with child-bearing potential (according to CTFG recommendations)
  7. Written informed consent.
Exclusion Criteria
  1. Histopathological (as approved by the histopathology core lab) and/ or clinical evidence of acute lymphocytic myocarditis, sarcoidosis, GCM or eosinophilic myocarditis,
  2. Known systemic inflammatory disease,
  3. Recent major surgery within <6 weeks, recent ICD implantation within <6 weeks or recent CRT implantation within <3 months prior to,
  4. Known coronary artery disease responsible for cardiac dysfunction (i.e., prior myocardial infarction, persistent stenosis ≥ 70%),
  5. Pregnancy or lactation,
  6. Contraindications to immunosuppressive treatment with MMF + corticosteroids,
  7. Inability to provide informed consent.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboMycophenolate Mofetil PlaceboMycophenolate mofetil (MMF) and prednisolone Placebo
PlaceboPrednisolone PlaceboMycophenolate mofetil (MMF) and prednisolone Placebo
Immunosuppressive treatmentMycophenolate MofetilMycophenolate mofetil (MMF) 1g bid and prednisolone at initially 1mg/kg in a step-down regime
Immunosuppressive treatmentPrednisoloneMycophenolate mofetil (MMF) 1g bid and prednisolone at initially 1mg/kg in a step-down regime
Primary Outcome Measures
NameTimeMethod
LVEF increase (metric)12 months follow-up

Absolute increase in LVEF at 12 months follow-up as assessed by blinded investigators of the MRI core lab (metric endpoint)lab) of immunosuppressive treatment with MMF and prednisolone compared to placebo

LVEF increase (binary)12 months follow-up

Proportion of patients with an absolute increase in LVEF ≥10% at 12 months follow-up as assessed by blinded investigators of the MRI core lab (binary endpoint).

Secondary Outcome Measures
NameTimeMethod
LVEF increase 6 months (MRI)6 months follow-up

Absolute increase in LVEF and rate of increase by ≥10% at 6 months follow-up (MRI, metric, and binary endpoint).

Ventricular remodelling (MRI)6 and 12 months follow-up

Absolute decrease of left ventricular diameters, volumes, mass, and sphericity from baseline to 6 and 12 months follow-up (MRI).

Strain (MRI)6 and 12 months follow-up

Changes in global longitudinal, radial, and circumferential strain from baseline to 6 and 12 months follow-up (MRI).

Composite clinical outcome12 months

Composite clinical outcome: cardiac death, heart transplantation or a heart failure event (hospitalization for heart failure or the equivalent, i.e., an urgent HF visit) within 12 months from randomization, analyzed as time to first event.physical capacity, cardiac autonomic function, transplant-free survival and hospitalization rate, biomarkers and adverse events

LVEF increase (echo)6 and 12 months follow-up

Absolute increase in LVEF and rate of increase by ≥10% at 6 and 12 months follow-up (echo, metric, and binary).

Ventricular remodelling (echo)6 and 12 months follow-up

Decrease of left ventricular diameters and volumes by ≥10% at 6 and 12 months follow-up (echo).

Strain (echo)6 and 12 months follow-up

Changes in global longitudinal, radial, circumferential, early, and late diastolic strain (LV), free wall and septal strain (RV), left atrial strain (LA) from baseline to 6 and 12 months follow-up (echo).

Diastolic parameters (echo)6 and 12 months follow-up

Changes in diastolic parameters from baseline to 6 and 12 months follow-up (echo).

Mitral and tricuspid regurgitation (echo)6 and 12 months follow-up

Presence of MR/TR \>2 at baseline and at 6 and 12 months followup (echo).

Cardiopulmonary exercise capacity6 and 12 months follow-up

Changes in cardiopulmonary exercise capacity: Distance in the sixminute walk test (6MWT) from baseline to 6 and 12 months followup and (optionally) VO2max, anaerobic threshold and VE/VCO2 on spiroergometry.

NYHA6 and 12 months follow-up

Changes in NYHA functional class from baseline to 6 and 12 months follow-up.

QoL12 months

Changes in patient-reported outcome (quality of life; QOL) from baseline to follow-up as assessed by the Kansas City Cardiomyopathy Questionnaire (KCCQ).

Cardiac autonomic function6 and 12 months follow-up

Changes in cardiac autonomic function from baseline to 6 and 12 months follow-up.

Composite safety outcome12 months

Time to the first occurrence of any of the components of the composite safety outcome: death of any cause, arrhythmias requiring intervention, severe adverse events requiring hospitalization.

Biomarker12 months

Time-averaged proportional change in NT-proBNP

Trial Locations

Locations (14)

Kerckhoff-Klinik GmbH

🇩🇪

Bad Nauheim, Germany

Charité - University Hospital Berlin

🇩🇪

Berlin, Germany

UHF- Universitäres Herz- und Gefässzentrum

🇩🇪

Frankfurt, Germany

University Hospital Essen

🇩🇪

Essen, Germany

University Hospital Freiburg - Bad Krozingen

🇩🇪

Freiburg, Germany

Universitätsmedizin Göttingen

🇩🇪

Göttingen, Germany

UKE Hamburg

🇩🇪

Hamburg, Germany

Klinikum rechts der Isar

🇩🇪

Munich, Germany

LMU Klinikum Standort Innenstadt

🇩🇪

München, Germany

University Hospital Regensburg

🇩🇪

Regensburg, Germany

University Hospital Greifswald

🇩🇪

Greifswald, Germany

Universitäres Herzzentrum Lübeck

🇩🇪

Lübeck, Germany

University Hospital Heidelberg

🇩🇪

Heidelberg, Germany

LMU Klinikum

🇩🇪

Munich, Germany

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