ANAKINRA for Inflammatory dilated cardiomyopathy

Inclusion criteria

• {"criterion_text":"- Age: 18 Years to 75 years;"}
• {"criterion_text":"- Diagnosis of DCM according to current guidelines;"}
• {"criterion_text":"- Symptoms of HF not improved or worsened despite at least 3 months of optimal therapy;"}
• {"criterion_text":"- LVEF<50% at echocardiography (TTE), not improved or worsened despite at least 3 months of optimal therapy;"}
• {"criterion_text":"- Increased high-sensitive troponin T (hs-TnT), and/or findings suggestive for actual or prior myocardial inflammation at cardiac MRI (within 6 months);"}
• {"criterion_text":"- Absence of coronary artery disease (coronary artery stenosis > 50% at angiography or coronary CT Scan, acceptable if performed during the last 12 months)."}
• {"criterion_text":"- Ability to sign an informed consent;"}
• {"criterion_text":"- Presence of CD3+ >7/mm2 cells on EMB, in addiction to all the aformentioned inclusion criteria, will be needed exclusively to be enrollend in the Phase IIa Randomized Double Blind monocentric Clinical Trial."}

Exclusion criteria

• {"criterion_text":"- Genetic DCM;"}
• {"criterion_text":"- Hepatic impairment = Child-Pugh Class C;"}
• {"criterion_text":"- Mechanical ventilation circulatory assistance;"}
• {"criterion_text":"- Pregnancy, breastfeeding. Female patients of childbearing potential may participate if adequate contraception is used during the study. (For the purposes of this trial, women of childbearing potential are defined as “All female subjects after puberty unless they are post-menopausal for at least 2 years or are surgically sterile.”)"}
• {"criterion_text":"- Contra-indication to ANAKINRA (known hypersensitivity to the active substance or to any of the excipients or to Escherichia coli-derived proteins)."}
• {"criterion_text":"- Presence of neutropenia < 1,5.109/L), or thrombocytopenia < 50.000/mm3;"}
• {"criterion_text":"- Any comorbidity limiting survival or conditions predicting inability to complete the study;"}
• {"criterion_text":"- Any concomitant immune-suppressive medications (i.e. azathioprine, methotrexate, cyclosporine, mycophenolate, cyclophosphamide, rituximab, tacrolimus);"}
• {"criterion_text":"- Therapy with prednisone >10 mg daily and/or any immuno-suppressive agents within 3 months before the enrolment;"}
• {"criterion_text":"- Congenital and/or acquired valvular disease or any other heart disease that could justify the severity of cardiac dysfunction; Major surgery within 2 weeks prior to randomization, or unhealed operation wounds."}
• {"criterion_text":"- Toxin abuse/exposure (Alcohol, amphetamines, cocaine, anthracyclines [e.g., doxycycline], trastuzumab, clozapine, chloroquine, carbon monoxide, cobalt, lead, mercury;"}
• {"criterion_text":"- Clinical suspicion or proven underlying active, chronic or recurrent bacterial, fungal or viral infections, including tuberculosis, or HIV infection or epatitis B virus (HBV) or hepatitis C virus (HCV) infection, Lyme disease, Chagas disease or any other bacterial/fungeal/protozoal disease possibly responsible for DCM;"}
• {"criterion_text":"- Endocrine, infiltrative (Cushing’s disease, acromegaly not clinically controlled hypo/hyperthyroidism, pheochromocytoma) or neuromuscular diseases (Dystrophinopathies [Duchenne/Becker muscular dystrophy/X-linked DCM], Limb-girdle muscular dystrophies, Facioscapulohumeral muscular dystrophy, Emery-Dreifuss muscular dystrophy, Friedreich’s ataxia, Myotonic dystrophy);"}
• {"criterion_text":"- Contraindications to EMB;"}
• {"criterion_text":"- Contraindications to PET/MRI (i.e. gadolinium hypersensitivity, renal failure, claustrophobia, pacemaker or ICD device, blood glucose>12.5 mmol/L);"}
• {"criterion_text":"- History of malignancy in the previous 5 years. Exceptions are basal cell skin cancer, carcinoma-in-situ of the cervix or low-risk prostate cancer after curative therapy;"}
• {"criterion_text":"- Any other concomitant or previous biological anti-cytokine treatment administered within 5 -half lives of the specific drug;"}
• {"criterion_text":"- Renal failure as defined by estimated glomerular filtration rate (eGFR) <30 ml/min, according to Cockcroft-Gault;"}

Primary endpoints

• {"endpoint_text":"- Improvement in left-ventricular function, based on the increase of left ventricular (LV) ejection fraction (EF) assessed by transthoracic echocardiography at 4-weeks.","definition_or_measurement_approach":"LV ejection fraction (EF) assessed by transthoracic echocardiography (TTE) at 4 weeks."}

Italy

Earliest CTIS Part Ii Submission Date

04-10-2024

Latest Decision Or Authorization Date

16-10-2024

Processing Time Days

12

Number Of Sites

1

Number Of Participants

24

Primary sponsor

Full Name

Ospedale San Raffaele S.r.l.

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Italy