Non-Ischemic Heart Failure Clinical Trial
Official title:
A Phase IIa Randomized Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Ischemia-tolerant Allogeneic Mesenchymal Bone Marrow Cells to Subjects With Non-ischemic Heart Failure
| Verified date | December 2019 |
| Source | CardioCell LLC |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
A phase IIa study to assess the safety and preliminary efficacy of intravenous dose of ischemia-tolerant Allogeneic Mesenchymal Bone Marrow Cells in subjects with non-ischemic heart failure.
| Status | Completed |
| Enrollment | 23 |
| Est. completion date | May 11, 2017 |
| Est. primary completion date | May 11, 2017 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years and older |
| Eligibility |
Inclusion Criteria: 1. Males and females =18 years of age 2. LVEF =35% on echocardiogram within 12 months of randomization to undergo MRI 3. Screening cardiac MRI at baseline with: Ejection fraction =40% No significant hyper enhancement on MRI scan in the opinion of the central imaging lab reviewer 4. Patients with non-ischemic heart failure etiology, as documented by absent or non-obstructive coronary artery disease on x-ray angiography or coronary computed tomography 5. Patients with history of heart failure and treated for at least three months with GDMT 6. NYHA class II-III symptoms 7. Ability to understand and provide signed informed consent 8. Reasonable expectation that patient will receive standard post-treatment care and attend all scheduled safety follow-up visits Exclusion Criteria: 1. Pregnant or nursing women or those of childbearing age and not using an effective method of contraception 2. History of stroke within 3 months 3. Cardiac surgery within 3 months prior to randomization or the likelihood of a requirement for such procedures during the study period 4. Current ICD or CRT or implantation planned within 6 months of infusion 5. Presence of clinically significant, uncorrected valvular heart disease, hypertrophic or restrictive cardiomyopathy, active myocarditis, or uncontrolled hypertension 6. History of cardiac arrest or life-threatening arrhythmias within 3 months 7. Treatment with parenteral inotropic agents within 1 month of randomization 8. Anticipated cardiac transplantation within 1 year 9. Illness other than heart failure with life expectancy less than 1 year 10. Received an experimental drug or device within 30 days of randomization 11. Left ventricular assist device or implantation planned in the next 6 months 12. Patients with complex congenital heart disease 13. Uncontrolled seizure disorder 14. Presence of immune deficiency 15. Clinically significant hematologic, hepatic, or renal impairment as determined by screening clinical laboratory tests: - Liver disease = ALT or AST > 3x normal, alkaline phosphatase or bilirubin >2x normal) - Renal disease = estimated glomerular filtration rate as assessed by the MDRD formula <30 ml/min - Hematologic = Unexplained leukocytosis >10 or hemoglobin < 9gm/dl 16. Presence of any other clinically-significant medical condition, psychiatric condition, or laboratory abnormality, that in the judgment of the investigator or sponsor for which participation in the study would pose a safety risk to the subject 17. Inability to comply with the conditions of the protocol 18. Malignancy within the previous five years, except adequately treated basal cell carcinoma, provided that it is neither infiltrating nor sclerosing, and carcinoma in situ of the cervix 19. Active myocarditis or early postpartum cardiomyopathy (within six months). 20. Systemic corticosteroids, cytostatics, immunosuppressive drug therapy (cyclophosphamide, methotrexate, cyclosporine, azathioprine, etc.), and DNA depleting or cytotoxic drugs taken within four weeks prior to study treatment 21. Porphyria 22. Allergy to sodium citrate or any "caine" type of local anesthetic 23. Any contraindication for gadolinium use for MRI 24. Patient scheduled for hospice care 25. Clinically relevant abnormal findings in the clinical history, physical examination, ECG, or laboratory tests at the screening assessment that would interfere with the objectives of the study or would preclude safe completion of the study. Abnormal findings could include: known HIV infection or other immunodeficiency state, chronic active viral infection (such as hepatitis B or C), acute systemic infections (defined as patients undergoing treatment with antibiotics), gastrointestinal tract bleeding, or any severe or acute concomitant illness or injury 26. Any other medical, social, or geographical factor that would make it unlikely that the patient could comply with study procedures (e.g., alcohol abuse, lack of permanent residence, severe depression, disorientation, distant location, or noncompliance) |
| Country | Name | City | State |
|---|---|---|---|
| United States | Emory University Hospital | Atlanta | Georgia |
| United States | Northwestern University Centers for Heart Failure Therapy | Chicago | Illinois |
| United States | Hospital of the University of Pennsylvania, Heart Failure and Transplant Program | Philadelphia | Pennsylvania |
| United States | Stony Brook Heart Institute | Stony Brook | New York |
| United States | MedStar Washington Hospital Center | Washington | District of Columbia |
| Lead Sponsor | Collaborator |
|---|---|
| CardioCell LLC | Stemedica Cell Technologies, Inc. |
United States,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Safety Will be Evaluated by Number of AE | As identified in the SAP, the safety analysis was the primary objective and was evaluated by the number of AEs | Total AEs and SAEs within 450 days post-infusion | |
| Secondary | Change in LVEF From Baseline to Day 90 Post-initial Infusion. | The secondary efficacy endpoint was the change in LVEF from baseline to Day 90 post-initial infusion. Participants with data available at each time point. | Baseline to Day 90 |