Ulcerative ColitisPhase 1NCT04312113
Angiographic Delivery of AD-MSC for Ulcerative Colitis
A Phase 1 study testing Adipose derived, autologous mesenchymal stem cells in people with Ulcerative Colitis, run by Mayo Clinic at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Ulcerative Colitis
- What is being tested?
- Adipose derived, autologous mesenchymal stem cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Nov 2020to Dec 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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)
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
About this study
Official title: A Phase I Single Site Open Label Study of Intra-arterial Delivery of Mesenchymal Stem Cells for Luminal Ulcerative Colitis
Researchers are trying to determine the safety and feasibility of using an adipose derived mesenchymal stem cell (MSC) to treat people with Ulcerative Colitis.
Read the 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.
- Conditions
- Ulcerative Colitis
- Being tested
- Adipose derived, autologous mesenchymal stem cells
- Medical subject headings
- Colitis, Ulcerative
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Adipose derived, autologous mesenchymal stem cellsDrug
Also called AD-MSC
Fat tissue will be enzymatically treated and cells will be cultured until a sufficient number are obtained for the treatment protocol.
| Study group | Role | What they receive |
|---|---|---|
| Autologous mesenchymal stem cells | Experimental | 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. Receives: Drug: Adipose derived, autologous mesenchymal stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events
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.
Measured: 24 months
Secondary outcomes (3)
Number of participants with mucosal healing
Mucosal appearance at endoscopy via Adapted Mayo Score (defined as score of 0 or 1)
Measured: 6 months
Number of participants with clinical symptom response
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)
Measured: 24 months
Number of participants with improved healing on pathology
Histopathology: Improved healing on surgical pathology (colectomy specimen or post-intervention colonic biopsies) as compared to pre-operative endoscopic biopsies
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2020
- Study start16 Nov 2020
- Primary completionOct 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ulcerative ColitisStudies of the same condition, open and under way.
- More trials run by Mayo ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mayo Clinic
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, William A. Faubion, M.D., Mayo Clinic
- Investigators
- William Faubion, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04312113
- Sponsor's protocol ID
- 19-000826
- Status last verified
- Jan 2026
- First submitted
- 14 Mar 2020
- Last updated
- 21 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Single Site Open Label Study of Intra-arterial Delivery of Mesenchymal Stem Cells for Luminal Ulcerative Colitis
- ClinicalTrials.gov ID
- NCT04312113
- Acronym / sponsor ID
- 19-000826
- Conditions
- Ulcerative Colitis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Mayo ClinicUniversity, hospital or other
- Study start
- 16 Nov 2020 (actual)
- Primary completion
- Oct 2026 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 18 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous mesenchymal stem cells | Experimental | Drug: Adipose derived, autologous mesenchymal stem cellsAdipose 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. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Males and Females 18-65 years of age.
- Moderate to Severe medically refractory inflammatory ulcerative colitis:
- 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
- All patients should have undergone a colonoscopy in last 12 months to rule out malignant or premalignant condition
Key exclusion criteria (16 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Adipose derived, autologous mesenchymal stem cellsAlso: AD-MSCFat tissue will be enzymatically treated and cells will be cultured until a sufficient number are obtained for the treatment protocol. | Drug | Autologous mesenchymal stem cells |
Primary outcome
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.
3 secondary outcomes
Number of participants with mucosal healing
Time frame: 6 months
Number of participants with clinical symptom response
Time frame: 24 months
Number of participants with improved healing on pathology
Time frame: 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jan 2026.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
William Faubion, MD principal investigator
Mayo Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Adipose derived, autologous mesenchymal stem cells in people with Ulcerative Colitis, run by Mayo Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 21 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-related adverse events (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jan 2026.