ClinicalTrials in Regenerative Medicine

Regenerative medicine encompasses stem cell therapies, gene therapies, tissue engineering, and extracellular vesicle-based treatments aimed at repairing or regenerating damaged tissues and organs. Below is a curated list of clinical trials, categorized by source, focusing on ongoing, recruiting, or recently completed studies for conditions such as cardiovascular diseases, neurodegenerative disorders, diabetes, and cancers. Where possible, links to the studies have been added based on available sources like CIRM’s website, Mayo Clinic pages, ClinicalTrials.gov, and PubMed. All links are set to open in a new window.

Trials from Mayo Clinic Research

Mayo Clinic conducts trials involving stem cells, gene vectors, or tissue engineering for conditions like heart disease, diabetes, and musculoskeletal issues. Selected trials include links to Mayo Clinic pages or ClinicalTrials.gov:

1. Title: Patient-Centered Exploration to Understand their Perception of Comprehensive Regenerative Care

   Phase: N/A

   Status: Not specified

   Condition/Disease: Diabetes Mellitus (Type 1 or 2)

   Intervention/Description: Survey-based study on patient perceptions of regenerative care in diabetes clinics.

   Link: Mayo Clinic Trial Page

2. Title: A Study to Evaluate Adipose Tissue for Regenerative Medicine Research in Cardiovascular Disease

   Phase: N/A

   Status: Not specified

   Condition/Disease: Cardiovascular Disease

   Intervention/Description: Collection of adipose tissue-derived extracellular vesicles (EVs) to study inflammation and remodeling.

   Link: Mayo Clinic Trial Page

3. Title: Genetic Investigations in Hypoplastic Left Heart Syndrome

   Phase: N/A

   Status: Not specified

   Condition/Disease: Hypoplastic Left Heart Syndrome

   Intervention/Description: Genomic screening (chromosomal microarray and whole genome sequencing) for genetic causes.

   Link: Mayo Clinic Trial Page

4. Title: Bioengineered Cardiac Tissue for Cardiomyopathy Analysis

   Phase: N/A

   Status: Not specified

   Condition/Disease: Cardiomyopathy

   Intervention/Description: Reprogrammed cells to create patient-specific cardiac tissue for analysis.

   Link: Mayo Clinic Trial Page

5. Title: A Phase I Study of iPS Cell Generation From Patients With COPD

   Phase: Phase 1

   Status: Not specified

   Condition/Disease: Chronic Obstructive Pulmonary Disease (COPD)

   Intervention/Description: Induced pluripotent stem cells (iPSCs) from skin fibroblasts for thoracic disease research.

   Link: ClinicalTrials.gov (NCT01860898)

6. Title: Autologous Muscle Derived Cells for Female Urinary Sphincter Repair

   Phase: N/A (Confirmatory)

   Status: Not specified

   Condition/Disease: Stress Urinary Incontinence

   Intervention/Description: Autologous muscle-derived cells injected for sphincter repair in females.

   Link: ClinicalTrials.gov (NCT01893138)

7. Title: Safety and Feasibility of Novel Therapy for Duodenal Mucosal Regeneration for Type II Diabetes (REGENT-1-US)

   Phase: Early Feasibility

   Status: Not specified

   Condition/Disease: Type 2 Diabetes

   Intervention/Description: Endoscopic device for duodenal mucosal regeneration to improve glucose control.

   Link: Veeva Clinical Trial Page

8. Title: Development of Skeletal Muscle Fibers from Patient-Derived Induced Pluripotent Stem Cells (iPSCs)

   Phase: N/A

   Status: Not specified

   Condition/Disease: Inherited Myopathies

   Intervention/Description: iPSCs to develop and characterize skeletal muscle cells.

   Link: Mayo Clinic Trial Page

9. Title: A Study of the Safety of Joint Injected Sc-rAAV2.5IL-1Ra in Patients with Moderate Knee Osteoarthritis

   Phase: N/A

   Status: Not specified

   Condition/Disease: Knee Osteoarthritis

   Intervention/Description: Gene therapy (virus-carried IL-1Ra) injected into the knee for safety assessment.

   Link: ClinicalTrials.gov (NCT02790723)

Key Trials from a Literature Review on Regenerative Medicine Applications

A 2022 review article summarizes clinical trials using cellular therapies (e.g., MSCs), extracellular vesicles (EVs), and tissue-engineered constructs for diseases like COVID-19, kidney disease, and heart failure. Notable studies include links to PubMed where available:

1. Study/Reference: Saad et al. (2017)

   Phase: Not specified (IND #15082)

   Status/Outcomes: No side effects; improved renal perfusion and oxygenation

   Condition/Disease: Atherosclerotic Renovascular Disease

   Intervention/Description: Autologous MSCs to stimulate angiogenesis and immune modulation.

   Link: PubMed

2. Study/Reference: Ye et al. (2017)

   Phase: Not specified

   Status/Outcomes: Improved (amino acides) secretion; reduced insulin needs

   Condition/Disease: Type 1 Diabetes

   Intervention/Description: MSCs to modulate immune response in newly diagnosed patients.

   Link: Not found in search; related trial: ClinicalTrials.gov (NCT03484741)

3. Study/Reference: Bartunek et al. (2017)

   Phase: Phase 3 (CHART-1)

   Status/Outcomes: Safe but neutral results; suggests better patient selection

   Condition/Disease: Ischemic Heart Failure

   Intervention/Description: Cardiopoietic stem cells for symptomatic heart failure.

   Link: PubMed

4. Study/Reference: Butler et al. (2017)

   Phase: Phase 2A

   Status/Outcomes: Safe; improved health status and function

   Condition/Disease: Nonischemic Cardiomyopathy

   Intervention/Description: MSCs with immunomodulatory effects.

   Link: PubMed

5. Study/Reference: Nassar et al. (2016)

   Phase: Not specified

   Status/Outcomes: Safe; improved kidney function

   Condition/Disease: Chronic Kidney Disease (Grade III-IV)

   Intervention/Description: Umbilical cord MSC-derived EVs to reduce inflammation.

   Link: PubMed

6. Study/Reference: Meng et al. (2020)

   Phase: Phase 1

   Status/Outcomes: Safe; reduced IL-6 levels

   Condition/Disease: Moderate to Severe COVID-19

   Intervention/Description: MSCs for anti-inflammatory effects.

   Link: PubMed

7. Study/Reference: Shi et al. (2021)

   Phase: Phase 2 (Randomized, Double-Blind)

   Status/Outcomes: Improvement in lung lesion volume

   Condition/Disease: Severe COVID-19

   Intervention/Description: Human umbilical cord MSCs for lung damage repair.

   Link: PubMed

8. Study/Reference: Sengupta et al. (2020)

   Phase: Not specified

   Status/Outcomes: Safe; restored oxygenation and immunity

   Condition/Disease: Severe COVID-19

   Intervention/Description: Bone marrow MSC-derived exosomes to downregulate cytokine storm.

   Link: PubMed

9. Study/Reference: Wu et al. (2020)

   Phase: Phase 1

   Status/Outcomes: Safe; reduced pulmonary fibrosis

   Condition/Disease: COVID-19 Pulmonary Fibrosis

   Intervention/Description: Human embryonic stem cell-derived immunity- and matrix-regulatory cells (hESC-IMRCs).

   Link: PubMed

10. Study/Reference: Ram-Liebig et al. (2017)

    Phase: Multicenter Observational

    Status/Outcomes: Not detailed; focused on reconstruction

    Condition/Disease: Urethral Stricture

    Intervention/Description: Tissue-engineered autologous oral mucosa graft.

    Link: PubMed

Trials Funded by the California Institute for Regenerative Medicine (CIRM)

CIRM supports innovative stem cell and gene therapies for rare and treatment-resistant diseases. The following trials highlight their efforts, with links to CIRM trial pages:

1. Disease/Target: Age-related macular degeneration

   Therapy Type: Stem cell therapy

   Phase: Phase 1/2

   Status: Launching

   Intervention/Description: Stem cells to restore retinal function in dry AMD.

   Link: RPESC RPE-4W Therapy for Dry Age-Related Macular Degeneration

2. Disease/Target: Amyotrophic Lateral Sclerosis (ALS)

   Therapy Type: Stem cell therapy

   Phase: Phase 3

   Status: Completed

   Intervention/Description: Neural stem cells injected into the spinal cord to slow ALS progression.

   Link: Phase 3 Study of NurOwn in ALS

3. Disease/Target: B cell cancers, Leukemia

   Therapy Type: Gene therapy

   Phase: Phase 1

   Status: Recruiting

   Intervention/Description: CAR-T cell therapy targeting CD19 for blood cancers.

   Link: Phase 1 Study of CD19/CD22 CAR T Cells

4. Disease/Target: Beta Thalassemia

   Therapy Type: Gene therapy

   Phase: Phase 1/2

   Status: Completed

   Intervention/Description: Gene-edited hematopoietic stem cells to correct beta-globin deficiency.

5. Disease/Target: Brain Cancer

   Therapy Type: Gene therapy

   Phase: Phase 1

   Status: Recruiting

   Intervention/Description: Oncolytic virus or CAR-T cells for glioblastoma.

6. Disease/Target: COVID-19, Respiratory Disorders

   Therapy Type: Stem cell therapy

   Phase: Phase 1/2

   Status: Closed

   Intervention/Description: Mesenchymal stem cells (MSCs) to reduce inflammation in severe cases.

7. Disease/Target: Cystinosis, Kidney Failure

   Therapy Type: Gene therapy

   Phase: Phase 1/2

   Status: Active, not recruiting

   Intervention/Description: Gene-corrected autologous stem cells for cystinosis treatment.

8. Disease/Target: Epilepsy

   Therapy Type: Stem cell therapy

   Phase: Phase 1

   Status: Recruiting

   Intervention/Description: GABAergic interneuron progenitors to control seizures.

9. Disease/Target: Heart Disease

   Therapy Type: Stem cell therapy

   Phase: Phase 1

   Status: Completed

   Intervention/Description: Cardiac progenitor cells for myocardial repair post-heart attack.

10. Disease/Target: HIV/AIDS

    Therapy Type: Gene therapy

    Phase: Phase 1/2

    Status: Recruiting

    Intervention/Description: CCR5-edited stem cells for HIV resistance.

11. Disease/Target: Sickle Cell Disease

    Therapy Type: Gene therapy

    Phase: Phase 1

    Status: Recruiting

    Intervention/Description: CRISPR-edited hematopoietic stem cells to produce fetal hemoglobin.

12. Disease/Target: Spinal Cord Injury

    Therapy Type: Stem cell therapy

    Phase: Phase 1/2

    Status: Active, not recruiting

    Intervention/Description: Neural stem cells for functional recovery in chronic injury.