Clinical Trials

The study purpose is to understand patients’ with the diagnosis of Diabetes Mellitus type 1 or 2 perception of the care they receive in the Diabetes clinic or Diabetes technology clinic at Mayo Clinic and to explore and to identify the healthcare system components patients consider important to be part of the comprehensive regenerative care in the clinical setting.

However, before we can implement structural changes or design interventions to promote comprehensive regenerative care in clinical practice, we first need to characterize those regenerative practices occurring today, patients expectations, perceptions and experiences about comprehensive regenerative care and determine the ...

The purpose of this study is to obtain adipose tissue from patients undergoing vascular surgery where EVs can be isolated from the fat and used for research purposes to examine their effect on inflammation and remodeling in culture and in animal models. The samples will immediately be processed to obtain EVs for research purposes - this is not a biorepository. 

The primary objective of the study is to utilize comprehensive, family-centered genomic screening strategies - chromosomal microarray (CMA) and whole genome sequencing (WGS) - to discover the genetic bases for Hypoplastic Left Heart Syndrome (HLHS), a severe type of congenital heart disease characterized by underdevelopment of left heart structures.

The purpose of this study is to produce and characterize patient-specific cardiac tissue for cardiomyopathy using reprogrammed cell technology. 

As part of the long-term goal of successfully implementing tissue regeneration strategies in an individualized manner for patients with thoracic diseases including, but not limited to: cystic fibrosis, pulmonary fibrosis and pulmonary hypertension, the investigators will assess the feasibility of collecting skin biopsies from patients undergoing surgery for thoracic disease, culturing skin fibroblasts from the biopsy, and reprogramming these skin fibroblasts into induced pluripotent cells.

The purpose of this research is to develop stem cells from cells within patients’ skin in order to better understand peripheral neuropathy and help develop treatments.

The purpose of this study is to create a protocol that can be used for collecting and studying umbilical cord tissue cells from healthy donors, and using these cells and their derivatives in clinical trials.

This randomized, double-blind, placebo-controlled, multicenter, confirmatory study will evaluate the efficacy and safety of Cook MyoSite Incorporated Autologous Muscle Derived Cells for Urinary Sphincter Repair (AMDC-USR) compared to a placebo (vehicle) control dose in the treatment of stress urinary incontinence (SUI) in adult female patients.

The purpose of this study is to determine the safety of sc-rAAV2.5IL-1Ra, a gene therapy carried by a virus and delivered by injection into the knee joint of patients with moderate osteoarthritis of the knee.

The goal of this study is to create a repository of skin fibroblasts from patients with inherited myopathies or suspected inherited myopathies. The study will optimize the development of skeletal muscle cells from patient induced pluripotent stem cells (iPSCs) and to characterize their morphological, biochemical, electrophysiological and molecular properties. The study will also use skin fibroblasts as source of DNA to investigate for research purpose the genetic defect causing the myopathy, and test in future potentially therapeutic drugs in the iPSC-derived muscle cells.

The objective of this early feasibility study is to assess the feasibility and preliminary safety of the Endogenex Divice for endoscopic duodenal mucosal regeneration in patients with type 2 diabetes (T2D) inadequately controlled on 2-3 non-insulin glucose-lowering medications. 

The purpose of this study is to determine the safety and feasibility of autologous, adipose derived stromal vascular fraction injections in the treatment of knee osteoarthritis.

The purpose of this study is to determine the receptivity of the Mayo Clinic’s Regenerative Medicine Consultation Service (RMCS) patients to participating in future research and sharing their ongoing experiences engaging in stem cell therapies (SCTs).

The purpose of this study is to determine if mesenchymal stem cells (MSC) derived from the fat tissue can be safely administered into the cerebrospinal fluid (CSF) of patients with spinal cord injury. Adipose-derived mesenchymal stem cells (AD-MSCs) have been used in previous research studies at the Mayo Clinic. All subjects enrolled in this study will receive AD-MSC treatment, which is still experimental and is not approved by the U.S. Food and Drug Administration (FDA) for large scale use. However, the FDA has allowed the use of this agent in this research study.

The purpose of this study is to develop a salivary repository protocol to boost salivary regenerative medicine research.

This study is being done to collect skin samples from people with and without neurodegenerative and vascular disorders including Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS), stroke and many others. We will use these skin samples to make and bank (store) a group of cells (cell line) called inducible pluripotent stem (iPS) cells.

The purpose of this study is to determine the safety and feasibility of injections of autologous umbilical cord blood (UCB) cells into the right ventricle of Hypoplastic Left Heart Syndrome (HLHS) children undergoing a scheduled Glenn surgical procedure. The investigators are doing this research study to find out if autologous stem cells from the individual's own umbilical cord blood can be used to strengthen the muscle of the right side of their heart. This will help determine the safety and feasibility of using cell-based regenerative therapy as an additional treatment for the management of HLHS.

The purpose of this study is to produce and characterize patient-specific cardiac tissue for Hypoplastic Left Heart Syndrome (HLHS) using reprogrammed cell technology.

The purpose of this study is to derive and characterize patient-specific disease models for sudden death-predisposing heritable channelopathies and cardiomyopathies using iPS-cell technology.  It is hoped that the molecular, cellular, and electrophysiological phenotypes of these in-vitro disease models will further elucidate the pathophysiologic mechanisms underlying these sudden death-associated conditions.

The purpose of this study is to develop patient-derived organoid cultures of urothelial carcinoma from non-invasively obtained urine samples and to characterize the histological, molecular and functional features of patient-derived organoids.

The objective of this study is the application of regenerative medicine techniques for advanced and individualized nasal reconstruction. Using pre-operative 3D facial scanning, the patient can choose the desired nose shape. The data can then be used to create patient-specific templates for both the nasal tip reconstruction mold and the skin/soft tissue reconstruction template.

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.

Primary Goal: To determine the toxicity of in-house, manufactured MUC1-activated T cells in patients with relapsed/refractory MUC1-expressing multiple myeloma.

The rationale for using MUC1-stimulated T-cells to treat multiple myeloma is twofold. The first is that T-cell therapies have been shown to be active in myeloma, making it an attractive disease model for the proposed study. The other is that we are expanding and using naturally occurring myeloma-fighting T-cells which may offer benefits, particularly with respect to longevity, as compared to the methods currently being employed using CAR-T and bispecific antibodies. This is highly significant as one of the ...

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 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.

The purpose of this study is to determine if cells from a patient's own adipose tissue is safe and capable of helping regenerate the femoral head in patients with osteonecrosis. The standard of care is known as hip decompression which simply removes dead tissue from the femoral head and creates a new cavity to be filled in by healthy bone. This trial will use hip decompression plus saline injection in one hip and hip decompression supplemented with adipose derived regenerative cells in patients with osteonecrosis in both of their hips.

The purpose of this study is to develop and implement an innovative treatment for erectile dysfunction (ED).

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. ...

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 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.

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).

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 determine the safety, feasibility, and efficacy of use of Platelet Rich Plasma (PRP) treatment in female breast cancer survivors with genitourinary syndrome of menopause (GSM).

The overall goal of this study is to develop regenerative cell therapy for use in female patients with aging skin. The primary objective of this proposal is to conduct a pilot study on the efficacy and safety of using platelet rich plasma (PRP) to treat this condition in females.

The purposes of this study are to determine the safety and feasibility of autologous platelet-rich plasma for the treatment of vulvar lichen sclerosus, and to determine the efficacy of autologous platelet-rich plasma for the treatment of vulvar lichen sclerosus.

The overall goal of this study is to develop regenerative cell therapy for use in patients with osteoarthritis (OA). The primary objective of this proposal is to conduct a pilot study that assesses the safety and feasibility of using concentrated bone marrow aspirate containing MSC to treat patients with painful knee OA.

The purpose of this study is to investigate the correlation between a patient’s complete blood count with differential (CBC w/diff), platelet count, platelet-rich plasma (PRP) blood and platelet counts with a patient’s clinical outcome from a PRP injection for the treatment of knee osteoarthritis (OA).

The purpose of this study is to determine whether AVB-114 compared to standard of care treatment is effective in inducing remission of the treated complex perianal fistula in subjects with Crohn’s Disease. It also aims to assess clinical and radiologic components of fistula remission, safety of treatment, disease activity, patient Quality of Life, and patient care journey, between AVB-114 and standard of care treatment.

The purpose of this study is to determine the safety and practical treatment use of STEM cells collected from a patient's own fat tissue, expanded in laboratory culture, and injected to treat symptoms of mild to severe knee osteoarthritis.

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.

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.

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 ...

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 ...

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 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.

The purpose of this research study is to gather information on the safety and effectiveness of Autologous Chondrocyte Transplantation system, Novocart® 3D, in comparison to standard microfracture treatment for injured knee.

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 ...

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).

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.

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 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).

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.

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 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 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.

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 utilize fresh tumor tissue to aid the development of future therapies for brain cancer.  

The primary objective of this registry is to obtain long-term survivorship data for the use of fresh OCAs in cartilage repair and reconstruction procedures.

The objectives of this study are to determine the effect of patient factors and manufacturing procedures on OBA product characteristics, to assess the clinical outcomes of patients affected by multiple painful musculoskeletal disorders and tissue regeneration needs that are treated with guided procedures in the Mayo Clinic Pain practice, to assess genetic and epigenetic factors associated to multiple painful musculoskeletal disorders and tissue regeneration needs, to determine the effect of OBA characteristics on patient outcomes for patients treated with OBA procedures, including a reference group of patients not receiving OBAs as part of their medical care.

This confirmatory study is a prospective randomized trial comparing the efficacy and safety of an autologous chondrocyte tissue implant (NeoCart) to the surgical intervention microfracture in the treatment of cartilage defects in the knee.

The purpose of this study is to assess the safety of the Angel® Concentrated Platelet Rich Plasma System and Angel® CPRP Processing Set to process Platelet Rich Plasma for intra-articular injection into four lumbar facet joints (2 levels) causing chronic low back pain.

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 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 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 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 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 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.

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.

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

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 assess the feasibility and safety of delivering adipose mesenchymal stem cells (AMSCs) to kidney allografts.

The purpose of this study is to characterize the readily available (circulating in peripheral blood) mediators of cell proliferation in individuals undergoing living liver donation.

The purpose of this study is to assess and compare regenerative injection therapies such as bone marrow aspiration and concentration (BMAC) and platelet rich plasma (PRP) for osteoarthritis.

The objectives of this study are to produce an allogeneic cartilage bank whereby cartilage tissue is obtained from consented donors who meet 21 CFR 1271 donor eligibility criteria, and to simluate clinical implantation of living donor osteochondral allografts banked from Aim 1, demonstrated to meet both viability and sterility requirements, in patients undergoing osteochondral transplantation in the Mayo clinical practice. Actual grafting using collected cartilage will not occur. This is a process validation study.

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.