Stem Cells for the Treatment of Pouchitis

NCT05578508 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: CCF-Pouchitis
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine the safety and feasibility of using allogeneic bone marrow derived mesenchymal stem cells (MSCs) to treat people with medically refractory Pouchitis.

Conditions

Pouchitis; Crohn's Disease; Ulcerative Colitis Chronic; Inflammatory Bowel Diseases; Pouch, Ileal

Keywords

Mesenchymal Stem Cells; Crohn's Disease; Inflammatory Bowel Diseases; Pouchitis; Ulcerative Colitis

Outcome Measures

Primary Outcomes (2)

• Adverse Events

  Number of adverse events that occur throughout the study.

  Change from Baseline over 12 months after the MSC injection

• Healing

  PDAI endoscopic activity less than or equal to 1, Clinical PDAI score less than or equal to 2, and total PDAI less than or equal to 4

  Change from Baseline over 12 months after the MSC Injection

Secondary Outcomes (9)

• Endoscopic Remission

  Change from Baseline over 12 months after the MSC Injection

• Clinical Remission

  Change from Baseline over 12 months after the MSC Injection

• Endoscopic Improvement

  Change from Baseline over 12 months after the MSC Injection

• Clinical Improvement

  Change from Baseline over 12 months after the MSC Injection

• Partial Clinical Healing measured with the Pouchitis Disease Activity Index No No response

  Baseline, 1 week, 2 weeks, 1 month, 2 months, 3 months, 6 months, 12 months after MSC injection

Study Arms (1)

Treatment Arm

EXPERIMENTAL

Submucosal endoscopic injection of 60 million allogeneic bone marrow derived mesenchymal stem cells (MSCs) into ileal pouch at baseline and possibly again after 3 months if not completely healed.

Drug: Mesenchymal Stem Cells (MSCs)

Interventions

Mesenchymal Stem Cells (MSCs) DRUG

Endoscopic injection of allogeneic bone marrow derived mesenchymal stem cells (MSCs) to the ileal pouch.

Also known as: Allogeneic Bone Marrow Derived Mesenchymal Stem Cells (MSCs)

Treatment Arm

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Men and women 18-75 years of age
• Residents of the United States
• Medically refractory pouchitis defined as lack of response to antibiotics, immunomodulators, and/or biologics
• Concurrent therapies with corticosteroids, 5-ASA drugs, thiopurines, MTX, antibiotics, anti-TNF therapy, anti-integrin and anti-interleukin are permitted if have been on them for at least 2 months prior to study enrollment without change
• No malignant or premalignant intestinal condition, ruled out on colonoscopy within 90 days of MSC delivery
• Ability to comply with protocol
• Competent and able to provide written informed consent
• Must have failed or have a contraindication to standard medical therapy including anti-TNF, anti-interleukin, or anti-integrin agent

You may not qualify if:

• 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
• HIV
• Hepatitis B or C
• Abnormal CBC at screening
• Abnormal AST or ALT at screening
• History of cancer including melanoma (with the exception of localized skin cancers) within 5 years of study enrollment
• Investigational drug use within thirty (30) days of baseline
• Pregnant or breastfeeding
• Multifocal proximal small bowel involvement which resembles Crohn's of the small bowel
• Evidence of pelvic sepsis and pelvic penetrating fistulizing disease
• Patients with intestinal diversion above the level of the pouch
• Neoplasia of pouch
• Change in medical regimen for pouchitis in the two months prior to study enrollment

Sponsors & Collaborators

• The Cleveland Clinic: lead
• Case Western Reserve University: collaborator

Study Sites (1)

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Related Publications (11)

• Garcia-Olmo D, Garcia-Arranz M, Garcia LG, Cuellar ES, Blanco IF, Prianes LA, Montes JA, Pinto FL, Marcos DH, Garcia-Sancho L. Autologous stem cell transplantation for treatment of rectovaginal fistula in perianal Crohn's disease: a new cell-based therapy. Int J Colorectal Dis. 2003 Sep;18(5):451-4. doi: 10.1007/s00384-003-0490-3. Epub 2003 May 20.

  PMID: 12756590 BACKGROUND

• Cho YB, Lee WY, Park KJ, Kim M, Yoo HW, Yu CS. Autologous adipose tissue-derived stem cells for the treatment of Crohn's fistula: a phase I clinical study. Cell Transplant. 2013;22(2):279-85. doi: 10.3727/096368912X656045. Epub 2012 Sep 21.

  PMID: 23006344 BACKGROUND

• Dietz AB, Dozois EJ, Fletcher JG, Butler GW, Radel D, Lightner AL, Dave M, Friton J, Nair A, Camilleri ET, Dudakovic A, van Wijnen AJ, Faubion WA. Autologous Mesenchymal Stem Cells, Applied in a Bioabsorbable Matrix, for Treatment of Perianal Fistulas in Patients With Crohn's Disease. Gastroenterology. 2017 Jul;153(1):59-62.e2. doi: 10.1053/j.gastro.2017.04.001. Epub 2017 Apr 9.

  PMID: 28400193 BACKGROUND

• Garcia-Olmo D, Garcia-Arranz M, Herreros D, Pascual I, Peiro C, Rodriguez-Montes JA. A phase I clinical trial of the treatment of Crohn's fistula by adipose mesenchymal stem cell transplantation. Dis Colon Rectum. 2005 Jul;48(7):1416-23. doi: 10.1007/s10350-005-0052-6.

  PMID: 15933795 BACKGROUND

• Garcia-Olmo D, Herreros D, Pascual I, Pascual JA, Del-Valle E, Zorrilla J, De-La-Quintana P, Garcia-Arranz M, Pascual M. Expanded adipose-derived stem cells for the treatment of complex perianal fistula: a phase II clinical trial. Dis Colon Rectum. 2009 Jan;52(1):79-86. doi: 10.1007/DCR.0b013e3181973487.

  PMID: 19273960 BACKGROUND

• Molendijk I, Bonsing BA, Roelofs H, Peeters KC, Wasser MN, Dijkstra G, van der Woude CJ, Duijvestein M, Veenendaal RA, Zwaginga JJ, Verspaget HW, Fibbe WE, van der Meulen-de Jong AE, Hommes DW. Allogeneic Bone Marrow-Derived Mesenchymal Stromal Cells Promote Healing of Refractory Perianal Fistulas in Patients With Crohn's Disease. Gastroenterology. 2015 Oct;149(4):918-27.e6. doi: 10.1053/j.gastro.2015.06.014. Epub 2015 Jun 25.

  PMID: 26116801 BACKGROUND

• Cho YB, Park KJ, Yoon SN, Song KH, Kim DS, Jung SH, Kim M, Jeong HY, Yu CS. Long-term results of adipose-derived stem cell therapy for the treatment of Crohn's fistula. Stem Cells Transl Med. 2015 May;4(5):532-7. doi: 10.5966/sctm.2014-0199. Epub 2015 Mar 31.

  PMID: 25829404 BACKGROUND

• Lee WY, Park KJ, Cho YB, Yoon SN, Song KH, Kim DS, Jung SH, Kim M, Yoo HW, Kim I, Ha H, Yu CS. Autologous adipose tissue-derived stem cells treatment demonstrated favorable and sustainable therapeutic effect for Crohn's fistula. Stem Cells. 2013 Nov;31(11):2575-81. doi: 10.1002/stem.1357.

  PMID: 23404825 BACKGROUND

• Panes J, Garcia-Olmo D, Van Assche G, Colombel JF, Reinisch W, Baumgart DC, Dignass A, Nachury M, Ferrante M, Kazemi-Shirazi L, Grimaud JC, de la Portilla F, Goldin E, Richard MP, Leselbaum A, Danese S; ADMIRE CD Study Group Collaborators. Expanded allogeneic adipose-derived mesenchymal stem cells (Cx601) for complex perianal fistulas in Crohn's disease: a phase 3 randomised, double-blind controlled trial. Lancet. 2016 Sep 24;388(10051):1281-90. doi: 10.1016/S0140-6736(16)31203-X. Epub 2016 Jul 29.

  PMID: 27477896 BACKGROUND

• Ciccocioppo R, Bernardo ME, Sgarella A, Maccario R, Avanzini MA, Ubezio C, Minelli A, Alvisi C, Vanoli A, Calliada F, Dionigi P, Perotti C, Locatelli F, Corazza GR. Autologous bone marrow-derived mesenchymal stromal cells in the treatment of fistulising Crohn's disease. Gut. 2011 Jun;60(6):788-98. doi: 10.1136/gut.2010.214841. Epub 2011 Jan 21.

  PMID: 21257987 BACKGROUND

• de la Portilla F, Alba F, Garcia-Olmo D, Herrerias JM, Gonzalez FX, Galindo A. Expanded allogeneic adipose-derived stem cells (eASCs) for the treatment of complex perianal fistula in Crohn's disease: results from a multicenter phase I/IIa clinical trial. Int J Colorectal Dis. 2013 Mar;28(3):313-23. doi: 10.1007/s00384-012-1581-9. Epub 2012 Sep 29.

  PMID: 23053677 BACKGROUND

MeSH Terms

Conditions

Pouchitis; Crohn Disease; Inflammatory Bowel Diseases; Colitis, Ulcerative

Condition Hierarchy (Ancestors)

Ileitis; Enteritis; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases; Ileal Diseases; Colitis; Colonic Diseases

Study Officials

• Amy L Lightner, MD

  The Cleveland Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Staff Colorectal Surgeon

Study Record Dates

• First Submitted: November 1, 2019
• First Posted: October 13, 2022
• Study Start: April 1, 2022
• Primary Completion: February 1, 2024
• Study Completion: February 1, 2024
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)