Stem Cell Therapy for Cardiomyopathy (CATO Trial)
Recruiting in Palo Alto (17 mi)
+2 other locations
Age: 18+
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: Roberto Bolli
Prior Safety Data
Approved in 3 jurisdictions
Trial Summary
What is the purpose of this trial?This is a Phase IIA, randomized, double blind, placebo controlled, multicenter study designed to assess the safety, feasibility, and efficacy of umbilical cord derived mesenchymal stromal cells (UC MSCs), administered intravenously (IV) as a single dose or repeated doses, in patients with ischemic cardiomyopathy (ICM).
Is stem cell therapy using umbilical cord-derived mesenchymal stromal cells (UC-MSCs) safe for humans?
Studies have shown that using umbilical cord-derived mesenchymal stromal cells (UC-MSCs) in humans is generally safe, with no serious adverse effects reported in patients with heart failure or rheumatoid arthritis.
23468How is the treatment with Umbilical Cord-derived Mesenchymal Stromal Cells (UC-MSCs) different from other treatments for cardiomyopathy?
This treatment is unique because it uses stem cells from umbilical cords, which are easily accessible and can be expanded in the lab. Unlike other treatments, UC-MSCs are administered intravenously and have shown potential to improve heart function by helping repair heart tissue in experimental models.
56789What data supports the effectiveness of the treatment Umbilical Cord-derived Mesenchymal Stromal Cells (UC-MSCs) for cardiomyopathy?
Research shows that UC-MSCs can improve heart function and repair heart tissue in animal models of heart disease, suggesting they might help in treating cardiomyopathy. These cells have been shown to improve heart function in rat models of heart failure and have regenerative potential in heart conditions.
16789Will I have to stop taking my current medications?
The trial requires participants to be on stable, maximally tolerated doses of heart failure medications for at least one month before joining. This means you should not stop taking your current medications, but they need to be stable without changes for 30 days.
Eligibility Criteria
Adults aged 21-85 with heart failure symptoms, documented coronary artery disease, and left ventricular dysfunction are eligible for this trial. They must be on stable heart medications for at least a month and women of childbearing age should use birth control. Exclusions include severe liver issues, certain allergies, recent cancer history, cognitive barriers to consent, or any condition that might interfere with the study.Inclusion Criteria
I've been on stable heart failure medication for at least a month.
I am between 21 and 85 years old.
I experience moderate to moderately severe heart failure symptoms.
I have severe heart artery blockage, heart muscle damage, and signs of heart failure.
Exclusion Criteria
My liver enzymes are more than three times the normal limit.
Participant Groups
The trial is testing whether injecting umbilical cord-derived mesenchymal stromal cells into patients with ischemic cardiomyopathy can be safe and effective. Participants will receive either one dose or repeated doses intravenously in a randomized double-blind setup against a placebo.
3Treatment groups
Experimental Treatment
Placebo Group
Group I: single-dose groupExperimental Treatment1 Intervention
One dose of UC-MSCs (100 x 106 cells) will be infused IV at a rate of 2 ml/min for a total of 60 ml over 30 minutes (3.3 million cells/ml/min). This will be followed by three IV infusions of placebo (same volume and rate) 2, 4, and 6 months later.
Group II: repeated-dose groupExperimental Treatment1 Intervention
Four doses of UC-MSCs (100 x 106 cells each) will be given 2 months apart. Each dose will be infused IV at a rate of 2 ml/min for a total of 60 ml over 30 minutes (3.3 million cells/ml/min).
Group III: control groupPlacebo Group1 Intervention
Four doses of vehicle (Plasma-Lyte A supplemented with 1% HSA) will be given 2 months apart. Each dose will be infused IV at a rate of 2 ml/min for a total of 60 ml over 30 minutes.
Umbilical Cord-derived Mesenchymal Stromal Cells (UC-MSCs) is already approved in China, European Union, United States for the following indications:
π¨π³ Approved in China as UC-MSCs for:
- Type 1 Diabetes
- Ischemic Cardiomyopathy
- Graft Versus Host Disease
- Sepsis
- Inflammatory Bowel Disease
- Cerebral Palsy
πͺπΊ Approved in European Union as UC-MSCs for:
- Graft Versus Host Disease
- Sepsis
- Inflammatory Bowel Disease
- Cerebral Palsy
πΊπΈ Approved in United States as UC-MSCs for:
- Graft Versus Host Disease
- Sepsis
- Inflammatory Bowel Disease
Find A Clinic Near You
Research locations nearbySelect from list below to view details:
University of Louisville School of Medicine, Institute of Molecular CardiologyLouisville, KY
University of Miami Miller School of MedicineMiami, FL
The Texas Heart Institute Houston TexasHouston, TX
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Who is running the clinical trial?
Roberto BolliLead Sponsor
University of MiamiCollaborator
United States Department of DefenseCollaborator
University of TexasCollaborator
References
Isolation of mesenchymal stem cells using the total length of umbilical cord for transplantation purposes. [2022]Umbilical cord (UC) mesenchymal cells have the ability to differentiate into various cell types, which make them an easily obtainable source for therapeutic uses. Different approaches have been used to isolate mesenchymal stem cells (MSC).
Banking human umbilical cord-derived mesenchymal stromal cells for clinical use. [2022]A great deal of interest has arisen recently with respect to human mesenchymal stem cells (MSCs), due to their broad therapeutic potential. However, the safety and efficacy of MSCs expanded ex vivo for clinical applications remain a concern. In this article, we establish a standardized process for manufacture of human umbilical cord-derived MSCs (UC-MSCs), which encompasses donor screening and testing, recovery, two-stage expansion, and administration. The biological properties and safety of UC-MSCs were then characterized and tested. The safety data from use in human patients have also been reported. After clinical-scale expansion, a yield of 1.03-3.78 Γ 10(8) MSCs was achieved in 10 batch manufacturing runs. The biological properties, such as plastic adherence, morphology, specific surface antigen (CD105, CD73, CD90, positive β₯ 95%; CD45, CD34, CD31, CD11b, CD19, HLA-DR, negative β€2%), and multipotent differentiation potential (osteogenesis and adipogenesis) were retained. Bacterial and mycoplasma tests were negative and endotoxin levels were lower than 2 EU/ml. No adverse events were noted in two patients treated with intravenously and/or intrathecally administered MSCs. The data obtained indicate that banking UC-MSCs for clinical use is feasible.
Human umbilical cord mesenchymal stem cell therapy for patients with active rheumatoid arthritis: safety and efficacy. [2022]This study was designed to assess the safety and efficacy of human umbilical cord mesenchymal stem cells (UC-MSCs) in the treatment of rheumatoid arthritis (RA). In this ongoing cohort, 172 patients with active RA who had inadequate responses to traditional medication were enrolled. Patients were divided into two groups for different treatment: disease-modifying anti-rheumatic drugs (DMARDs) plus medium without UC-MSCs, or DMARDs plus UC-MSCs group (4Γ10(7) cells per time) via intravenous injection. Adverse events and the clinical information were recorded. Tests for serological markers to assess safety and disease activity were conducted. Serum levels of inflammatory chemokines/cytokines were measured, and lymphocyte subsets in peripheral blood were analyzed. No serious adverse effects were observed during or after infusion. The serum levels of tumor necrosis factor-alpha and interleukin-6 decreased after the first UC-MSCs treatment (P
Role of VEGF-A in angiogenesis promoted by umbilical cord-derived mesenchymal stromal/stem cells: in vitro study. [2022]Mesenchymal stromal/stem cells derived from human umbilical cord (UC-MSCs) uniquely combine properties of embryonic and postnatal MSCs and may be the most acceptable, safe, and effective source for allogeneic cell therapy e.g. for therapeutic angiogenesis. In this report we describe pro-angiogenic properties of UC-MSCs as manifested in vitro.
Intramuscular injection of human umbilical cord-derived mesenchymal stem cells improves cardiac function in dilated cardiomyopathy rats. [2022]Stem cells provide a promising candidate for the treatment of the fatal pediatric dilated cardiomyopathy (DCM). This study aimed to investigate the effects of intramuscular injection of human umbilical cord-derived mesenchymal stem cells (hUCMSCs) on the cardiac function of a DCM rat model.
Safety and Efficacy of the Intravenous Infusion of Umbilical Cord Mesenchymal Stem Cells in Patients With Heart Failure: A Phase 1/2 Randomized Controlled Trial (RIMECARD Trial [Randomized Clinical Trial of Intravenous Infusion Umbilical Cord Mesenchymal Stem Cells on Cardiopathy]). [2022]Umbilical cord-derived mesenchymal stem cells (UC-MSC) are easily accessible and expanded in vitro, possess distinct properties, and improve myocardial remodeling and function in experimental models of cardiovascular disease. Although bone marrow-derived mesenchymal stem cells have been previously assessed for their therapeutic potential in individuals with heart failure and reduced ejection fraction, no clinical trial has evaluated intravenous infusion of UC-MSCs in these patients.
Umbilical cord mesenchymal stromal cells engraft and transdifferentiate into cardiomyocyte-like cells following acute myocardial ischemiaβ. [2021]Human umbilical cord-derived mesenchymal stromal cells (hUC-MSCs) gained importance in acute/chronic ischemic cardiomyopathy because of their outstanding regenerative potential in various pathologic conditions. The present study was designed to determine to what extent hUC-MSCs contribute to myocardial regeneration in acute experimental myocardial infarction (MI) in rats.
Human umbilical cord-derived stem cell sheets improve left ventricular function in rat models of ischemic heart failure. [2022]Human umbilical cord-derived mesenchymal stem cells (UC-MSCs) are among the most promising cell therapy sources used to treat ischemic heart disease. Cell sheet engineering has been used to transplant stem cells and improve their therapeutic effectiveness. We aimed to evaluate the effectiveness of UC-MSC sheets in the treatment of chronic ischemic heart failure.
Chorion-derived perinatal mesenchymal stem cells improve cardiac function and vascular regeneration: Preferential treatment for ischemic heart disease. [2022]Stem cell therapy has emerged as a novel treatment for heart failure after myocardial infarction (Ml). Bone marrow-derived mesenchymal stem cells (BM-MSCs) are commonly considered because of their accessibility and usability. However, their therapeutic potential remains controversial. In our previous in vitro study, chorion-derived mesenchymal stem cells (C-MSCs) and umbilical cord-derived mesenchymal stem cells (UC-MSCs) demonstrated an ability to differentiate into cardiomyocytes and neural cells, respectively. Thus, we examined whether C-MSCs had a better differentiation potential in an MI animal model.