Clinical Trials

The purpose of this study is to evaluate the safety of using unlicensed cord blood units from the National Cord Blood Program in unrelated  patients needing stem cell transplants, by carefully documenting all infusion-related problems.

The purpose of this study is to assess the effectiveness and safety of Cx601, adult allogeneic expanded adipose-derived stem cells (eASC), for the treatment of complex perianal fistula(s) in patients with Crohn's disease over a period of 24 weeks and a follow-up period up to 52 weeks.

The purpose of this study is to collect fat and blood vessel wall tissue for processing into adult stem cells and then test those cells for specific biological markings.

The purpose of this study is to collect human menstrual blood at the time of gynecology visits in order to conduct future studies on the isolation and characterization of human menstrual blood and endometrial stem cells to better individualize treatment for abnormal uterine bleeding (AUB) and study the therapeutic properties of human menstrual-derived Mesenchymal Stem Cells (MSCs).

The purpose of this study is to assess optimal dosing frequency, effectiveness and safety of adipose-derived autologous mesenchymal stem cells delivered into the spinal fluid of patients with multipe system atrophy (MSA).

Multiple system atrophy (MSA) is a rare, rapidly progressive, and invariably fatal neurological condition characterized by autonomic failure, parkinsonism, and/or ataxia. There is no available treatment to slow or halt disease progression. 

The purpose of this study is to explore patients’ perceptions using educational interventions to debunk or prebunk misinformation of advertisements about unproven stem cell interventions (SCIs). 

The purpose of this study is to assess the safety and tolerability of intravenously delivered mesenchymal steml cells (MSC) in one of two fixed dosing regimens at two time points in patients with chronic kidney disease.

The purpose of this study to test the feasibility and safety for autologous (from your own body) skin cells that are manufactured into stem cells of cardiac lineage to be delivered into the heart muscle to determine if those stem cells will strengthen the heart muscle and can be used as an additional treatment for the management of  congenital heart disease. 

The purpose of this study is to assess the effectiveness and safety of Cx601, adult allogeneic expanded adipose-derived stem cells (eASC), for the treatment of complex perianal fistula(s) in patients with Crohn's disease over a period of 24 weeks and a follow-up period up to 52 weeks.

The purpose of this study is to evaluate the long-term safety of a single dose of darvadstrocel in participants with Crohn's disease (CD) and complex perianal fistula by evaluation of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).

The aim of this study is to measure the differences in quality of life and mood of hematopoietic stem cell transplant (HCT) patients and their caregivers staying at a hospital hospitality house (HHH), such as the Gift of Life Transplant House, the Help in Healing Home, and the Gabriel House of Care versus staying at a hotel/rental apartment or house. The goal is to investigate if staying in a HHH, with its different environment and support systems and programs, has a positive impact on the quality of life (QOL) and mood of patients undergoing a HCT and their caregivers.

The purpose of this study is to evaluate the pharmacokinetics (PK), safety and tolerability of pegcetacoplan in patients with TA-TMA.

The purpose of this study is to assess the safety and feasibility of mesenchymal stem cells therapy in patients with advanced chronic obstructive pulmonary disease.

The purpose of this study is to produce, using current Good Manufacturing Practices (cGMPs), a bank of 50 primary fibroblast cell lines from skin biopsies obtained by consenting donors who meet 21 CFR 1271 donor eligibility criteria, and to use fibroblasts in the cell bank generated in aim 1 to produce new induced pluripotent stem cell lines using Good Manufacturing Practices (cGMPs). These iPSC lines will then be screened to identify those with optimal characteristics for treatment purposes, as well as for the potential generation of transplantable tissues and therapeutics for chronic disease.

The purpose of this study is to determine the safety and feasibility of allogeneic, culture-expanded BM-MSCs in subjects with painful facet joint arthropathy.

The purpose of this study is to determine determine the safety of intraspinal delivery of mesenchymal stem cells (MSCs) to the cerebral spinal fluid of patients with Amyotrophic Lateral Sclerosis (ALS) using a dose-escalation study.

This study aims to evaluate the safety of local delivery of AMSCs for recurrent GBM by noting the incidence of adverse events, as well as radiological and clinical progression.

To assess the preliminary efficacy of local delivery of AMSCs for recurrent GBM by comparing the clinical, survival, progression, and radiographic outcomes from patients enrolled in our study to historical controls from our institution.

The purpose of the present study is to investigate the safety and efficacy of a single intrathecal injection of autologous, culture expanded AD-MSCs specifically in subjects with severe traumatic SCI when compared to patients undergoing physical therapy.

The overall goal of this study is to determine the safety and feasibility of infusing adipose-derived mesenchymal stem cells directly into the artery of renal allografts with biopsy-proven rejection in order to reduce inflammation detected in the graft.   We contend that future studies will show that administering immunomodulatory cells directly into the allograft will be more effective and safer than the current approaches of delivering massive doses of systemic immunosuppression.

Study participation involves receiving mesenchymal stem cells (MSC), created from the adipose tissue (body fat) of a donor, and infused into the main artery of a transplanted ...

The purpose of this study is to assess the safety of autologous mesenchymal stromal (stem) cell transfer using a biomatrix (the Gore Fistula Plug) to treat perianal fistula.

To determine the safety and toxicity of intra-arterial infused autologous adipose derived mesenchymal stromal (stem) cells in patients with vascular occlusive disease of the kidney.

The purpose of this study is to evaluate the long-term safety of a single dose of darvadstrocel in participants with Crohn's disease (CD) and complex perianal fistula by evaluation of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).

The purpose of this trial is to compare the treatment strategy of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) to the treatment strategy of Best Available Therapy (BAT) for treatment-resistant relapsing multiple sclerosis (MS). Participants will be randomized at a 1 to 1 (1:1) ratio. All participants will be followed for 72 months after randomization (Day 0, Visit 0).

The purpose of this study is to evaluate the effectiveness of ibrutinib in reducing the incidence of NIH moderate/severe chronic GVHD.

The purpose of this study is to collect, convert and bank blood cells from healthy volunteers into stem cells (iPSCs) at a current good manufacturing practice (cGMP) facility within the Discovery and Innovation building on the Mayo FLorida campus. After comprehensive validation, we will bank those cGMP-iPSCs as a resource available to Mayo Clinic investigators and also to outside investigators as appropriate. Those bio-specimens could be unique resources to develop new protocols for production of clinical grade iPSC-derived cells, cell-derived products such as extracellular vesicles, and tissues to support Investigational New Drug (IND) and related clinical trials.

The objective of this study is to evaluate the safety and feasibility of autologous mononuclear cells (MNC) collected from bone marrow (BM) delivered into the myocardium of the right ventricle of subjects with Ebstein anomaly undergoing surgical Ebstein repair. Additionally, the potential cardiovascular benefits will also be evaluated. This add-on procedure is anticipated to pose little risk to the subject and has the potential to foster a new strategy that leverages the regenerative capacity of individuals with congenital heart disease during the surgically mandated Ebstein repair.

To assess the safety and feasibility of mesenchymal stem cells therapy in patients with transplant related bronchiolitis obliteran syndrome (BOS).

The purpose of this study is to assess the safety and tolerability of intra-arterially delivered mesenchymal stem/stromal cells (MSC) to a single kidney in one of two fixed doses at two time points in patients with progressive diabetic kidney disease. 

Diabetic kidney disease, also known as diabetic nephropathy, is the most common cause of chronic kidney disease and end-stage kidney failure requiring dialysis or kidney transplantation.  Regenerative, cell-based therapy applying MSCs holds promise to delay the progression of kidney disease in individuals with diabetes mellitus.  Our clinical trial will use MSCs processed from each study participant to test the ...

This phase I/II trial studies the side effects and best dose of oncolytic measles virus encoding thyroidal sodium iodide symporter (MV-NIS) infected mesenchymal stem cells and to see how well it works in treating patients with recurrent ovarian cancer. Mesenchymal stem cells may be able to carry tumor-killing substances directly to ovarian cancer cells.

The purpose of this study is to engage a cohort of patients who are avid information seekers about stem cells to assess their beliefs, online information sources and their credibility, and views on the credibility and persuasiveness of advertisements and warning messages available on the internet; we will use this data along with health behavior theories to develop communication messages aimed at inoculating patients against misinformation, correcting misconceptions, and providing evidence-based information about stem cell procedures.

This study aims to evaluate the safety of intramyocardial delivery of autologous umbilical cord blood-derived mononuclear cells during Fontan surgical palliation and measure surrogate markers of myocardial protection within a non-randomized study design to obtain prospective data from treatment and control populations.

The purpose of this study is to determine the safety and efficacy of intrathecal treatment delivered to the cerebrospinal fluid (CSF) of mesenchymal stem cells in ALS patients every 3 months for a total of 4 injections over 12 months. Mesenchymal stem cells (MSCs) are a type of stem cell that can be grown into a number of different kinds of cells. In this study, MSCs will be taken from the subject's body fat and grown. CSF is the fluid surrounding the spine. The use of mesenchymal stem cells is considered investigational, which means it has not been approved by ...

Group 1: The primary purpose of this study is to evaluate the safety and tolerability of an autologous dendritic cells (DC) vaccine delivered by intra-tumoral injection in patients with primary liver cancer treated with high-dose conformal external beam radiotherapy (EBRT).

Group 2: The primary purpose of this study is to estimate the progression-free survival rate at 2 years post-registration to see if treatment is efficacious compared to historical data

This study is an extension to re-treat partial and non-responders from the previously approved Phase 1 MCS-AFP protocols IRB #12-009716 (Crohn's Disease perianal fistulas) and 15-003200 (cryptoglandular perianal fistulas).

To compare the effect of senolytic drugs on cellular senescence, physical ability or frailty, and adipose tissue-derived MSC functionality in patients with chronic kidney disease.

Primary Objectives:
To assess the efficacy of a single 3-day treatment regimen with dasatinib and quercetin (senolytic drugs) on clearing senescent adipose-derived MSC in patients with CKD.

To assess the efficacy of a single 3-day treatment regimen with dasatinib and quercetin (senolytic drugs) on improving adipose-derived MSC functionality in patients with CKD.

Secondary Objective:
To assess the short-term effect of a single 3-day treatment regimen with dasatinib and quercetin (senolytic drugs) on ...

The investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore Bio-A Fistula Plug) in a Phase I study using a single dose of 20 million cells. 20 patients (age 12 to 17 years) with Crohns perianal fistulas will be enrolled. Subjects will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore fistula plug as per current clinical practice. The subjects will be subsequently followed for fistula response and closure ...

The purpose of this study is to test the safety of this novel cell, combination- based regenerative therapy for use in patients with symptomatic focal cartilage defects of the knee.

In this proposal, we will generate hiPSCs from AA patients and use our TREE-based approaches to introduce AA-associated variants into isogenic hiPSCs. In turn, we will use these isogenic hiPSC lines in a 3-D cortical model to address the following hypothesis-testing questions: (1) Does the presence of specific ABCA7 variants modulate disease-related phenotypes in a hiPSC-based system? (2) Are the risk modifying effects of the ABCA7 variants mediated through cell-autonomous or non-autonomous mechanisms? (3) Do these ABCA7 variants exert their effects through modulation of Aβ processing, secretion, and uptake? (4) What is the effect of these ABCA7 variants ...

The investigators propose to study the safety of autologous mesenchymal stromal cell transfer using a biomatrix (the Gore® Bio-A®; Fistula Plug) in a Phase I study using a single dose of 20 million cells. Twenty adult patients (age 18 years or older) with refractory, complicated perianal fistulizing Crohn's disease will be enrolled. Subjects will undergo standard adjuvant therapy including drainage of infection and placement of a draining seton with continuation of pre-existing anti-Crohn's therapy. Six weeks post placement of the draining seton, the seton will be replaced with the MSC loaded Gore® Bio-A® fistula plug as per current clinical practice. ...

The purpose of this study is to assess neurodevelopmental and psychosocial outcomes (i.e., executive function, social cognition, psychosocial adjustment, adaptive skills) in children with hypoplastic left heart syndrome (HLHS) who underwent right-ventricle-directed delivery of autologous umbilical cord derived mononuclear cells during staged cardiac surgical palliation, and to compare their outcomes to a matched sample of children with HLHS who did not receive autologous umbilical cord derived mononuclear cells during surgery.

The purpose of this study is to assess the safety, tolerability, optimal dosing, effectiveness signals reflecting kidney repair, and markers of mesenchymal stem cells (MSC) function that relate to response to allogenenic adipose tissue-derived MSC in patients with Chronic Kidney Disease (CKD).

Will injection(s) of autologous culture-expanded AMSCs be safe and efficacious for treatment of painful Hip OA, and if so, which dosing regimen is most effective?

The purpose of this study is to determine the safety of using an autologous mesenchymal stromal cell (MSC) coated fistula plug in people with fistulizing Crohn's disease. Autologous means these cells to coat the plug come from the patient.

This study will evaluate the safety of intramuscular administration of PLX-R18 (allogenetic ex-vivo explanded placental adherent stromal cells) in subjects who have with incomplete hematopoietic recovery after hematopoietic stem cell transplantation.

The objective of this study is to generate a panel of iPSCs from 30 subjects who do not have a personal history of major neuropsychiatric disorders.  

State-of-the-art induced pluripotent stem cells (iPSC) technology has become a powerful biomedical research tool and it clearly holds great potential for application to neuropsychiatric research.

The purpose of this study is to determine the success of mesenchymal stem cells, developed from the patient's own fat tissue, for reducing hemodialysis arteriovenous fistula failure when applied during the time of surgical creation.

The purpose of this study is to evaluate the safety and effectiveness of CD34+ cell intracoronary injections for treating coronary endothelial dysfunction (CED).

The purpose of this study is to collect adipose tissue from patients undergoing elective surgery, or from healthy volunteers, test the donors to assure that they comply with all regulatory aspects required of healthy donors, expand and test mesenchymal stromal cells (MSC), and bank them for future use.

Ulcerative Colitis (UC) is a chronic inflammatory disease affecting the mucosal lining of the colon and rectum and the incidence is increasing, but the etiology remains unknown. Patients may require a proctocolectomy due to refractory disease. Prior to an operation, UC is treated with antibiotic therapy, immunomodulatory therapy and immunosuppressive agents. While there is an increasing number of approved biologics for the treatment of UC, there are many patients that still suffer from refractory disease. Thus, alternative mechanisms of therapy are desperately needed.

Treatments that have the potential to reduce mucosal inflammation could alleviate the pathology of luminal UC. This trial ...

The purpose of this study is to assess the safety and effectiveness of a Stem cell transfer using a biomatrix (The Gore Fistula Plug) in patients with persistent symptoms of post-surgical gastrointestinal leaks despite current standard radiologic and endoscopic treatments.  The subjects will be followed for fistula response and closure for 18 months. This is an autologous product (derived from the patient) and used only for the same patient.

The purpose of this study is to evaluate the safety and effectiveness of CD34+ cell intracoronary injections for treating coronary endothelial dysfunction (CED).

The purpose of this study is to collect adipose tissue from patients undergoing elective surgery, or from healthy volunteers, test the donors to assure that they comply with all regulatory aspects required of healthy donors, expand and test mesenchymal stromal cells (MSC), and bank them for future use.

Ulcerative Colitis (UC) is a chronic inflammatory disease affecting the mucosal lining of the colon and rectum and the incidence is increasing, but the etiology remains unknown. Patients may require a proctocolectomy due to refractory disease. Prior to an operation, UC is treated with antibiotic therapy, immunomodulatory therapy and immunosuppressive agents. While there is an increasing number of approved biologics for the treatment of UC, there are many patients that still suffer from refractory disease. Thus, alternative mechanisms of therapy are desperately needed.

Treatments that have the potential to reduce mucosal inflammation could alleviate the pathology of luminal UC. This trial ...

The purpose of this study is to evaluate quality of life over time in patients treated with CAR-T therapy compared with autologous and allogeneic stem cell transplant.

The current proposal aims to test the feasibility of immune function analysis for Tai Chi Easy (TCE) intervention in multiple myeloma (MM) patients undergoing autologous stem cell transplantation (ASCT) with concurrent exploration of health related quality of life (HRQOL).

The purpose of this study is to determine the effectiveness of MB-CART2019.1 cells administered following a conditioning lymphodepletion regimen in diffuse large B cell lymphoma (DLBCL) subjects who failed at least two lines of therapy as measured by objective response rate (ORR) at one month.

This is a double-blind, sham-controlled clinical study to evaluate the safety and feasibility of AMI MultiStem therapy in subjects who have had a heart attack (Non-ST elevation MI).

The purpose of this study is to compare the efficacy and safety of maribavir to valganciclovir for the treatment of cytomegalovirus (CMV) infection in asymptomatic hematopoietic stem cell transplant recipients.

The purpose of this study is to evaluate safety, tolerability, pharmacokinetics, and effectiveness of SER-155 in adults undergoing hematopoietic stem cell transplantation to reduce the risk of infection and graft vs. host disease.

This phase Ib/II trial studies how well dendritic cell therapy after cryosurgery in combination with pembrolizumab works in treating patients with stage III-IV melanoma that cannot be removed by surgery. Vaccines made from a person's white blood cells mixed with tumor proteins may help the body build an effective immune response to kill tumor cells. Cryosurgery, also known as cryoablation or cryotherapy, kills tumor cells by freezing them. Monoclonal antibodies, such as pembrolizumab, may block tumor growth in different ways by targeting certain cells. Giving dendritic cell therapy after cryosurgery in combination with pembrolizumab may work better in treating patients ...

The study aims to characterize patient factors, such as pre-existing comorbidities, cancer type and treatment, and demographic factors, associated with short- and long-term outcomes of COVID-19, including severity and fatality, in cancer patients undergoing treatment. The study also is aimed to describe cancer treatment modifications made in response to COVID-19, including dose adjustments, changes in symptom management, or temporary or permanent cessation. Lastely, evaluate the association of COVID-19 with cancer outcomes in patient subgroups defined by clinico-pathologic characteristics.

The purpose of this study is to determine the safety of using an autologous mesenchymal stromal cell (MSC) coated fistula plug in people with rectovaginal fistulizing Crohn's disease. Autologous means that these cells that coat the plug come from you. You will be in this study for two years. There is potential to continue to monitor your progress with lifelong regular visits as part of your standard of care. All study visits take place at Mayo Clinic and Rochester, MN. The study visit schedule is as follows: Visit 1 (Week -6) - Screening visit: exam under anesthesia and surgery to ...

The purpose of this study is to evaluate the cellular composition of PRP produced by the Arthrex Angel System.

The purpose of this study is evaluate the safety of allogeneic adipose derived mesenchymal stem cell (AMSC) use during hemodialysis arteriovenous fistula and arterial bypass creation and its effectiveness on improving access maturation and primary anastomotic patency.

Although survivorship recommendations have been developed in areas such as lymphoma and stem cell transplant, the long-term effects of CAR-T therapy are unknown. In addition, relatively little is known about the psychosocial impact of CAR-T on survivors and their caregivers. Due to the intensive nature of CAR-T treatment and its unique side effects, including neurotoxicity in the acute setting and infections and financial burden in the long-term setting, a longitudinal study that assesses these issues in a quantitative and qualitative fashion is required. Consideration of both patient and caregiver needs is important for the provision of appropriate and ...

The purpose of this study is to evaluate the side effects of vaccine therapy in treating patients with glioblastoma that has come back. Vaccines made from a person's white blood cells mixed with tumor proteins from another person's glioblastoma tumors may help the body build an effective immune response to kill tumor cells. Giving vaccine therapy may work better in treating patients with glioblastoma.

The purpose of this trial is to evaluate the cosmetic role of novel anti-aging regenerative skin care product, human platelet extract (HPE), on skin rejuvenation. 

Skin aging is a natural part of human aging process caused by intrinsic and extrinsic factors, such as genetics, cellular metabolism, chronic light exposure and other toxins.  Cosmetological care for facial skin aging includes daily skin care, correct sun protection and aesthetic non-invasive procedures. 80 participants over the age of 40 years with moderate photoaging (dyschromic facial skin with fine lines and wrinkles) will be recruited from Mayo Clinic Center for Aesthetic Medicine and ...

The purpose of this study is to collect adiopose tissue to derive mesenchymal stem cells.

The purpose of this study is to compare standard-dose combination chemotherapy to high-dose combination chemotherapy and stem cell transplant in treating patients with germ cell tumors that have returned after a period of improvement or did not respond to treatment. Drugs used in chemotherapy, such as paclitaxel, ifosfamide, cisplatin, carboplatin, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Giving ...

The purpose of this study is to assess the feasibility and safety of delivering adipose mesenchymal stem cells (AMSCs) to kidney allografts.

The purpose of this study is to assess the safety, effectiveness, and overall benefit of FCR001 cell therapy in de novo living donor renal transplantation.

This randomized phase III trial studies rituximab after stem cell transplant and to see how well it works compared with rituximab alone in treating patients with in minimal residual disease-negative mantle cell lymphoma in first complete remission. Monoclonal antibodies, such as rituximab, may interfere with the ability of cancer cells to grow and spread. Giving chemotherapy before a stem cell transplant helps kill any cancer cells that are in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. After treatment, stem cells are collected from the patient's blood and stored. ...

The primary objective of the United States Food and Drug Administration (FDA) for this study is to demonstrate non-inferiority in subjects who received an allogeneic BMT for subjects randomized to Rezafungin for Injection compared to subjects randomized to the standard antimicrobial regimen (SAR) for fungal-free survival at Day 90 (±7 days).

The primary objective of the European Medicines Agency (EMA) for this study is to demonstrate superiority in subjects who received an allogeneic BMT randomized to Rezafungin for Injection compared to subjects randomized to the SAR for fungal-free survival at Day 90 (±7 days).

This randomized phase III trial studies ibrutinib to see how well it works compared to placebo when given before and after stem cell transplant in treating patients with diffuse large B-cell lymphoma that has returned after a period of improvement (relapsed) or does not respond to treatment (refractory). Before transplant, stem cells are taken from patients and stored. Patients then receive high doses of chemotherapy to kill cancer cells and make room for healthy cells. After treatment, the stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy. Ibrutinib is a ...

The purpose of this research study is to evaluate a treatment regimen called IRD which will be given to participants after their stem cell transplant in an effort to help prolong the amount of time the participants are disease-free after transplant. IRD is a three-drug regimen consisting of ixazomib, lenalidomide (also called Revlimid), and dexamethasone. After 4 cycles of IRD, the participants will be randomized to receive maintenance therapy either with ixazomib or lenalidomide. 09/23/2019: Upon review of the interim analysis that suggested inferior progression-free survival in the ixazomib maintenance arm, there will be no further randomizations into the ...

The primary purpose of this study is to identify the therapeutic effect of Adipose-Induced Regeneration (AIR) in radiation-induced skin injury of post-mastectomy breast cancer patients.