A Phase I Single Site Open Label Study of Intra-arterial Delivery of Mesenchymal Stem Cells for Luminal Ulcerative Colitis
Overview
- Phase
- Phase 1
- Intervention
- Adipose derived, autologous mesenchymal stem cells
- Conditions
- Ulcerative Colitis
- Sponsor
- Mayo Clinic
- Enrollment
- 20
- Locations
- 1
- Primary Endpoint
- Number of participants with treatment-related adverse events
- Status
- Active, Not Recruiting
- Last Updated
- 3 months ago
Overview
Brief Summary
Researchers are trying to determine the safety and feasibility of using an adipose derived mesenchymal stem cell (MSC) to treat people with Ulcerative Colitis.
Detailed Description
Participants will undergo screening for study, if eligible, participants will be dosed with 15 million or 30 million cells will be administered via IA delivery with interventional radiology. Participant study visits after study intervention includes visits on: Day 1, Week 1, Week 2, Week 8, Week 24, Week 52, and Week 104.
Investigators
William A. Faubion, M.D.
Principal Investigator
Mayo Clinic
Eligibility Criteria
Inclusion Criteria
- •Males and Females 18-65 years of age.
- •Moderate to Severe medically refractory inflammatory ulcerative colitis:
- •as defined by a an Adapted Mayo Score of 5to 9 points
- •including an endoscopic sub-score of 2 or 3
- •Concurrent therapies with corticosteroids, 5-ASA drugs, thiopurines, MTX, antibiotics, anti-TNF, and anti-integrin therapy are permitted.
- •To meet the definition of refractory UC, all patients must have failed at least 2 standard FDA approved medications for the treatment of UC
- •Current standard therapy includes 5-ASA products, thiopurines, anti-TNF therapy, ustekinemab, vedolizumab, and tofacitinib (i.e. all FDA approved therapies for UC).
- •Refractory and failure to response is defined as continued symptoms despite 12 weeks of therapy at FDA approved doses by product necessitating change in medical strategy or referral for colectomy.
- •All patients should have undergone a colonoscopy in last 12 months to rule out malignant or premalignant condition
- •Female subjects that are of child bearing potential must to agree to use effective contraception method(s) for the duration of the study
Exclusion Criteria
- •Inability to give informed consent.
- •Clinically significant medical conditions within the six months before administration of MSCs: e.g. myocardial infarction, active angina, congestive heart failure or other conditions that would, in the opinion of the investigators, compromise the safety of the patient.
- •Specific exclusions; Known history of hepatitis B, C, or HIV
- •Patients that have had a partial colectomy
- •Patients that have underlying vasculitis or have been diagnosed with an underlying condition that predisposes to developing blood clots.
- •History of cancer including melanoma (with the exception of localized skin cancers)
- •Investigational drug within thirty (30) days of baseline
- •History of clinically significant auto-immunity (other than UC) or any previous example of fat-directed autoimmunity. Note that auto-immmunity is defined as a systemic immune mediated disease for which the antigen is known or unknown. Autoimmune diseases other than UC are excluded. Extraintestinal manifestations of UC (specifically joint inflammation, eye inflammation, PSC, skin manifestations- i.e. pyoderma gangrenosum, erythema nodosum) will be allowable.
- •Allergic to local anesthetics
- •Pregnant patients or trying to become pregnant or breast feeding.
Arms & Interventions
Autologous mesenchymal stem cells
Adipose derived, autologous mesenchymal stem cells (AD-MSCs) at a dose of 15 million or 30 million cells will be administered via intra-arterial delivery with interventional radiology to the inferior mesenteric artery in subjects with medically refractory ulcerative colitis.
Intervention: Adipose derived, autologous mesenchymal stem cells
Outcomes
Primary Outcomes
Number of participants with treatment-related adverse events
Time Frame: 24 months
Evaluate safety by assessment of adverse events defined as worsening (change in nature, severity, or frequency of bowel movements, bleeding per rectum, or tenesmus) of UC present at the time of the study, intercurrent illnesses, abnormal laboratory values (this includes clinically significant shifts from baseline within the range of normal that the investigator considers to be clinically significant) or clinically significant abnormalities in physical examination, vital signs, weight, frequency of bloody stools or change in stools.
Secondary Outcomes
- Number of participants with improved healing on pathology(24 months)
- Number of participants with mucosal healing(6 months)
- Number of participants with clinical symptom response(24 months)