Stem cell thrapy in patients with cardiomyopathia not related to ischemicheart disease: a pilot study

Phase 1

• Conditions: The overall aim of the project is to test the feasibility and safety of allogeneic adipose-derived stromal cells (CSCC_ASC) investigational medicinal product, to improve myocardial function in patients with nonischemic dilated cardiomyopathies (NIDCM) and heart failure.; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

• Registration Number: EUCTR2018-002538-19-SI
• Lead Sponsor: Rigshospitalet

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-10-04
• Last Update Posted: 20 February 2023

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

1. 30 to 80 years of age
2. Signed informed consent
3. Patients with non-ischemic dilated cardiomyopathy
4. NYHA = II in spite of optimal heart failure treatment and have no other treatment options
5. Heart failure medication unchanged two months prior to inclusion/signature of informed consent. Changes in diuretics accepted
6. LVEF = 405%
7. Plasma NT-pro-BNP > 300 pg/ml (> 35 pmol/L)
8. Patients cannot be included until three months after implantation of a cardiac resynchronisation therapy device (CRTD) and until 1 month after an ICD unit
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 15
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 15

Exclusion Criteria

1. Heart Failure NYHA I
2. Moderate to severe aortic stenosis (valve area < 1.3 cm2) or valvular disease with option for surgery or interventional therapy.
3. Heart failure caused by cardiac valve disease or untreated hypertension.
4. If the patient is expected to be candidate for MitraClip therapy of mitral regurgitation in the 12 months follow-up period.
5. Cardiomyopathy with a reversible cause that has not been treated e.g. thyroid disease, alcohol abuse, hypophosphataemia, hypocalcaemia, cocaine abuse, selenium toxicity & chronic uncontrolled tachycardia
6. Cardiomyopathy in association with a neuromuscular disorder e.g. Duchenne's progressive muscular dystrophy
7. Previous cardiac surgery
8. Diminished functional capacity for other reasons such as: obstructive pulmonary disease (COPD) with forced expiratory volume (FEV) < 1L/min, moderate to severe claudication or mobid obesity
9. Clinical significant anaemia (haemoglobin < 6 mmol/L), leukopenia (leucocytes < 2 109/L), leucocytosis (leucocytes > 14 109/L) or thrombocytopenia (thrombocytes < 50 109/L)
10. Reduced kidney function (eGFR < 30 ml/min)
11. Left ventricular thrombus
12. Anticoagulation treatment that cannot be paused during cell injections.
13. Patients with reduced immune response
14. History with malignant disease within five years of inclusion or suspected malignity – except treated skin cancer other than melanoma
15. Pregnant women
16. Woman of childbearing potential unless ßHCG negative and they should be on contraception during the trial
17. Other experimental treatment within four weeks of baseline tests
18. Participation in another intervention trial
19. Life expectancy less than one year

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of the study is to investigate safety and regenerative capacity of direct intra-myocardial injection of 100 million allogeneic CSCC_ASCs in NIDCM patients with reduced left ventricular EF (= 40%) and heart failure.;Secondary Objective: Allogeneic antibodies, left ventricular ejection fraction, end-systolic volume and myocardial mass. Development of allogeneic antibodies and laboratory safety measurements 1, 3 and 6 months after treatment and changes in left ventricular ejection fraction (LVEF), end-diastolic volume and myocardial mass at 6 months follow-up. Additional secondary endpoints are changes in NYHA, Kansas City Cardiomyopathy Questionnaire, EQ-5D3L Questionnaire, 6 min walking test, additional echocardiographic measures (Global strain %) and NT-pro-BNP.;Primary end point(s): 1. Left ventricle end-systolic volume Measured using echocardiography;Timepoint(s) of evaluation of this end point: 6 months after treatment <br>

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Allogeneic antibodies, left ventricular ejection fraction, end-systolic<br>volume and myocardial mass.<br>Additional secondary endpoints are changes in NYHA, Kansas City<br>Cardiomyopathy Questionnaire, EQ-5D3L Questionnaire, 6 min walking<br>test, additional echocardiographic measures (Global strain %) and NTpro-BNP.<br>;Timepoint(s) of evaluation of this end point: 1, 3, 6 and 12 months after treatment