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Angiographic Delivery of AD-MSC for Ulcerative Colitis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04312113
Recruitment Status : Recruiting
First Posted : March 18, 2020
Last Update Posted : September 30, 2021
Sponsor:
Information provided by (Responsible Party):
William A. Faubion, M.D., Mayo Clinic

Tracking Information
First Submitted Date  ICMJE March 14, 2020
First Posted Date  ICMJE March 18, 2020
Last Update Posted Date September 30, 2021
Actual Study Start Date  ICMJE November 16, 2020
Estimated Primary Completion Date December 31, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 14, 2020)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 14, 2020)
  • Number of participants with mucosal healing [ Time Frame: 6 months ]
    Mucosal appearance at endoscopy via Adapted Mayo Score (defined as score of 0 or 1)
  • Number of participants with clinical symptom response [ Time Frame: 24 months ]
    To assess the clinical symptom response of luminal healing induced by the intra-arterial delivery of autologous AD-MSCs for the treatment of UC. Using the validated via Adapted Mayo Score (decrease from Baseline ≥ 2 points and ≥ 30%, including a decrease in rectal bleeding sub-score ≥ 1 or an absolute rectal bleeding sub-score ≤ 1)
  • Number of participants with improved healing on pathology [ Time Frame: 24 months ]
    Histopathology: Improved healing on surgical pathology (colectomy specimen or post-intervention colonic biopsies) as compared to pre-operative endoscopic biopsies
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Angiographic Delivery of AD-MSC for Ulcerative Colitis
Official Title  ICMJE A Phase I Single Site Open Label Study of Intra-arterial Delivery of Mesenchymal Stem Cells for Luminal Ulcerative Colitis
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Ulcerative Colitis
Intervention  ICMJE Drug: Adipose derived, autologous mesenchymal stem cells
Fat tissue will be enzymatically treated and cells will be cultured until a sufficient number are obtained for the treatment protocol.
Other Name: AD-MSC
Study Arms  ICMJE Experimental: 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: Drug: Adipose derived, autologous mesenchymal stem cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: March 14, 2020)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2022
Estimated Primary Completion Date December 31, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
  • Hemoglobin must be greater than 8
  • INR must be less than 1.5
  • Ability to comply with protocol
  • Competent and able to provide written informed consent

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.
  • Neoplasia of the colon and preoperative biopsy
  • C. Difficile infection within 30 days of study injection
  • Diagnosis of indeterminate colitis or suspicion of CD
  • Subjects with fulminant colitis, toxic megacolon, with ostomy, or ileoanal pouch
  • History or demonstration of pathology related to adipose tissue
  • Any other indication determined by the PI to be counter indicated for participation on this trial.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Jessica Friton (507) 284-0495 friton.jessica@mayo.edu
Contact: Erin Kammer 507-538-0678 kammer.erin@mayo.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04312113
Other Study ID Numbers  ICMJE 19-000826
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party William A. Faubion, M.D., Mayo Clinic
Study Sponsor  ICMJE Mayo Clinic
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: William Faubion, MD Mayo Clinic
PRS Account Mayo Clinic
Verification Date September 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP