Heart Failure Due to Dilated Cardiomyopathy
Conditions
Interventions
Experimental Group:For JV001 injection, two dose slope climbing tests were planned, 2×10^11vg/kg and 6×10^11vg/kg, respectively
Sponsors
Shanghai East Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (1) diagnosed DCM>=1 year, and current NYHA class II-IV; (2) Left ventricular ejection fraction (LVEF) =3 months; Or unable to tolerate standard guideline-recommended drug therapy (clinical evidence required), but investigator assessed subjects who might benefit from the study drug; Or more than 2 hospitalizations due to heart failure within 1 year requiring intravenous diuretics, and the condition was stable for more than 2 weeks; 4. Have the ability to understand and voluntarily sign the informed consent before the trial, and be able to complete the study in accordance with the requirements of the trial protocol; 5. Male or female: (1) Male subjects must agree to use contraception for at least 6 months after the treatment visit; (2) The female subjects were not pregnant or breastfeeding; And (3) women of childbearing potential agree to follow contraceptive instructions for at least 6 months after dosing.
Exclusion criteria
Exclusion criteria: 1.Patients with heart failure caused by heart diseases other than dilated cardiomyopathy (including but not limited to severe valvular heart disease, hyperthyroidism, congenital heart disease, acute viral myocarditis, acute coronary syndrome, hypertrophic obstructive cardiomyopathy, pericardial disease, myocardial amyloidosis, infiltrative cardiomyopathy,uncorrected thyroid disease, or left ventricularaneurysm); 2.Coronary angiography has a degree of narrowing of the left main coronary artery (LM), left anteriordescending branch (LAD), left circumflex branch(LCX) or right coronary artery (RCA) > 50%; 3.Uncontrolled arrhythmias that the investigatorbelieves would affect this trial; 4.Acute myocardial infarction within 6 monthsbefore screening; 5.Previous presence of subjects with immunological abnormalities that the investigators believe could affect this trial (including but not limited to congenital immunodeficiency, lupusery thematosus, primary vasculitis, systemic sclerosis, antiphospholipid syndrome,autoimmune liver disease, autoimmune thyroiditis); 6.Those who have a history of tumor for less than 5 years or currently have a tumor, or those who have precancerous lesions confirmed by pathological examination (including but not limited to breast ductal carcinoma in situ, cervicaldysplasia, etc.); 7.Known progress liver disease (active hepatitis A, chronic hepatitis B or C virus infection, non-cardiogenic cirrhosis, etc.); 8.Acute infection developed within 2 weeks prior to screening requiring intravenous antibiotic therapy, or current infection requiring anti-infective therapy; 9.Those who have a history of disseminated herpes simplex infection or recurrent (> 1 times) or disseminated herpes zoster; 10.Percutaneous coronary intervention (PCI), coronary bypass grafting (CABG), Implantable Cardioverter Defibrillator (ICD)/ permanentpacemaker/ Cardiac resynchronisation therapy (CRT) implantation, radiofrequency ablation,ventricular volume reduction, valve repair orplasty, intra-aortic balloon counterpulsation, passive restraint devices (e.g., CorCap™ cardiacsupport devices), cardiac assist device implantation, heart transplantation, or other cardiac surgery may be received within 3 months prior to screening or within 3 months after administration; 11.Those who donated >= 400 mL of blood within 4 weeks prior to screening, or who had significant blood loss equivalent to at least 400 mL, or who received blood transfusions within 8 weeks; 12.Subjects with contraindications for coronary angiography; 13.Contraindications for Magnetic Resonance Imaging (MRI) detection, including but not limited to: pacemaker, defibrillator, artificial heart valve,metal clip after aneurysm surgery, drug perfusiondevice implanted in the body, any electronic device implanted in the body (neurostimulator, bone growth stimulator), intravascular embolization steel ring, filter, ECG recording monitor, shrapnel or iron sand in the body, fixed steel plate and nails after fracture surgery, cochlear implant, intraocular metal foreign body,etc. Claustrophobia, etc. 14.Those who have a history of substance abuse within the past five years or have used drugs in the 3 months prior to the test; 15.Patients are participating in other clinical trials, or have been completed less than 3 months after the end of other clinical trials; 16.Abnormal liver function: ALT or AST > 3 times the upper limit of normal value (ULN); 17.Abnormal renal function: glomerular filtration rate< 30 m
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Dose-limiting toxicity (DLT) within 28 days of a single intracoronary infusion of JV001;Incidence of adverse events and serious adverse events within 1 year of administration; | — |
Secondary
| Measure | Time frame |
|---|---|
| 6-minute Walk Test (6MWT);Myocardial remodeling;N-terminal Prohormone Brain Natriuretic Peptide Tests;Left ventricular ejection fraction;AAV9 Vector, Target Gene (TERT) Payload;ADA/NAb; | — |
Countries
China
Contacts
Public ContactWei Han
Shanghai East Hospital
Outcome results
None listed