Clinical Trials

Cell Therapy



Cardiovascular Cell Therapy Research Network (CCTRN)

CONCERT

Combination Of meseNchymal and c-kit+ Cardiac stEm cells as Regenerative Therapy for Heart Failure (CONCERT-HF)

Heart failure is a serious and common condition in which the heart muscle does not pump blood throughout the body as well as it should. Over a period of years, the heart’s pumping ability continues to get worse and a heart transplant may become necessary. Treatment options are extremely limited.

Stem cells are cells that do not yet have a specific function in the body. Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown from bone marrow (the spongy tissue inside of bones). C-kit+ Cardiac Stem Cells (CSCs) are stem cells that can be grown from a sample of tissue collected from the heart. Stem cells can develop into other types of more mature cells, such as blood and muscle cells. It is hoped that by placing these cells in the heart, they can give rise to new muscle that will allow the heart to work better.

The purpose of this research study is to determine whether giving MSCs and/or CSCs to patients with heart muscle damage is safe. It will also help us learn whether these treatments improve heart function for people who are not ideal candidates for other forms of standard therapy such as surgery or techniques used to widen or unclog an artery.

SENECA

Stem Cell Injection in Cancer Survivors (SENECA)

Common use of a group of cancer medications called anthracyclines has dramatically improved cancer survival numbers over the past 50 years. Anthracycline-based cancer medications remain common and effective treatments for breast cancer, lymphomas, leukemias, and sarcomas. Unfortunately, the use of anthracyclines is limited due to their poisonous effects on the heart, including the development of a form of heart failure called anthracycline-induced cardiomyopathy (AIC). These effects can be seen as late as 20 years after the cancer treatment. Current treatments for AIC reduce the symptoms but there is no cure for this disease. While studies suggest that the usual medications used to treat heart failure (e.g. ACE inhibitors, angiotensin receptor blockers, beta-blockers, and statins) may help treat AIC, there continues to be a group of patients that will develop worsening symptoms and end-stage heart failure despite the best medical therapy, with many individuals worsening to the point of requiring a heart transplant.

Stem cells are cells that do not yet have a specific function in the body. Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown from bone marrow (the spongy tissue inside of bones). Stem cells can develop into other types of more mature (specific) cells, such as blood and muscle cells. It is hoped that by placing these cells into the heart, they will allow the heart to work better and reduce the scarred heart tissue associated with heart failure.

Rather than taking these cells from the patient’s own bone marrow (which has been exposed to the cancer medications in the past), MSCs can be taken from a healthy donor (who has never had chemotherapy). This type of stem cell is called allogeneic (or allo for short).

The purpose of this research study is to determine whether giving allo-MSCs to patients with AIC is safe and whether these treatments improve heart function.

MI-NSTEMI

Non-ST Elevation Myocardial Infarction

This is a trial for patients coming to the emergency room with a heart attack and who are enrolled at the time of going to the catheterization laboratory. The trial is evaluating the effect of donor stem cells to minimize the loss of heart function over time. Patients will be enrolled for one year into this trial.

DREAM-HF

Efficacy and Safety of Allogeneic Mesenchymal Precursor Cells (Rexlemestrocel-L) for the Treatment of Heart Failure (DREAM HF-1)

This is a trial for patients with heart failure (ejection fraction <40%) regardless of reason (with or without coronary artery disease). This trial will evaluate the effect of donor MSC’s compared to placebo on heart function over time. Patients will be enrolled for one year into this trial.

CardiAMP

Randomized Controlled Pivotal Trial of Autologous Bone Marrow Mononuclear Cells Using the CardiAMP Cell Therapy in Patients With Post Myocardial Infarction Heart Failure (CardiAMP Heart Failure Trial)

This is a trial for patients who have had a heart attack in the past and have heart failure as a result (ejection fraction <20%-<40%). This trial will evaluate the effect administering the patient’s own bone marrow stem cells directly into the most affected parts of the heart compared to placebo on heart function over time. Patients will be enrolled for one year into this trial.

Please contact our Clinical Trials Office for information: (352) 273-8933  ufcardiovascularct@medicine.ufl.edu

Pediatric Brain Tumor Immunotherapy Trials

ReMATCH

Phase II study to evaluate the effectiveness and progression free survival of Adoptive T Cell Therapy During Recovery from Myeloablative and Non-Myeloablative Chemotherapy with Hematopoietic Stem Cell Transplantation in first recurrence of medulloblastoma and supratentorial primitive neuroectodermal tumors (PNET)

This multi-institutional trial will consist of two groups A and B, and three parts: 1) Biopsy or Resection, 2) Induction or Salvage Chemotherapy 3) Consolidation or NMA Chemotherapy followed by Stem Cell infusion (HSC) and Immunotherapy (TTRNA -ALT and DC vaccinations).

ACTION

Phase I study to determine the safety of adoptive cellular therapy in pediatric patients with newly diagnosed High Grade Glioma receiving dose intensified TMZ and Immunotherapy therapy (TTRNA ALT-DC’s) with and without human stem cells (HSC).

This clinical trial will consist of three parts: Surgery, Chemoradiation, and adjuvant TMZ with Immunotherapy (Five TMZ cycles with TTRNA-DCs and TMZ Cycle 6 followed by DCs + ALT +/- HSCs.

 

BRAVO

Phase I study to determine the feasibility and safety of adoptive cellular therapy in pediatric patients with newly diagnosed DIPG with or without dose-intensified TMZ during cycles of Dendritic cell vaccination.

This clinical trial will consist of two Groups, A and B, and three parts: 1) Biopsy, 2) focal radiotherapy + concurrent TMZ, and 3) immunotherapy with human stem cell infusion (HSC) during and after maintenance cycles DI TMZ (Group A); or 1) Biopsy, 2) focal radiotherapy alone, and 3) immunotherapy consisting of DC vaccines prior to cyclophosphamide + fludarabine lymphodepletive conditioning and adoptive cellular therapy and human stem cells (HSC) afterward (Group B).


Adult Immunotherapy Clinical Trials

ATTAC-II

A Phase II Randomized, Blinded, and Placebo-controlled Trial of CMV RNA-Pulsed Dendritic Cells with Tetanus-Diphtheria Toxoid Vaccine in Patients with Newly-Diagnosed Glioblastoma

The purpose of this research study is to determine if an investigational dendritic cell vaccine, called pp65 DC, is effective for the treatment of a specific type of brain tumor called glioblastoma (GBM) when given with stronger doses of routine chemotherapy.

It is a multi-site trial. Patients are randomized to 3 treatment arms: pp65 short LAMP DC vaccine; pp65 Full-Length LAMP DCs vaccine; Autologous unpulsed Peripheral Blood Mononuclear Cells (control group).

The vaccine is given in conjuction with dose-intense chemotherapy (Temozolomide).