Mesenchymal Stem Cells for the Treatment of Perianal Fistulizing Crohn's Disease

NCT04519671 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: 20-1020
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine the safety and efficacy of using adult allogeneic bone marrow derived mesenchymal stem cells (MSCs), to treat people for medically refractory perianal fistulizing Crohn's disease.

Conditions

Perianal Crohn Disease; Perianal Fistula; Crohn Disease

Keywords

perianal fistula; perianal Crohn's disease; mesenchymal stromal cells; mesenchymal stem cells

Outcome Measures

Primary Outcomes (1)

• Treatment related adverse events

  Number of participants with treatment related adverse events post-injection of 75 million allogeneic bone marrow derived MSC's for the treatment of perianal fistula(s) in the setting of Crohn's disease as assessed by protocol CCF-Stem Cells IBD-001

  Month 6

Secondary Outcomes (1)

• Complete clinical healing

  Month 6, Month 12

Other Outcomes (3)

• Partial clinical healing

  Month 6, Month 12

• Lack of response

  Month 6, Month 12

• Worsening of disease

  Month 6, Month 12

Study Arms (2)

Mesenchymal Stem Cells

EXPERIMENTAL

Direct injection of adult allogeneic bone marrow derived mesenchymal stem cells, at a dose of 75 million cells into perianal fistula(s) at baseline with a possible repeat injection at 3 months if not completely healed from the first injection.

Drug: Mesenchymal Stem Cells

Placebo

PLACEBO COMPARATOR

Direct injection of normal saline. If not completely healed after 6 months, participants will then cross over to the treatment group to receive a direct injection of adult allogeneic bone marrow derived mesenchymal stem cells, at a dose of 75 million cells into perianal fistula(s)

Other: Placebo

Interventions

Mesenchymal Stem Cells DRUG

Direct injection of adult allogeneic bone marrow derived mesenchymal stem cell product, at a dose of 75 million cells into perianal fistula(s)

Mesenchymal Stem Cells

Placebo OTHER

Normal saline

Placebo

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 with a diagnosis of Crohn's disease for at least six months duration.
• Single and Multi-tract Perianal fistula, with or without previous failed surgical repair.
• Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia
• Ability to comply with protocol
• Competent and able to provide written informed consent
• Concurrent Crohn's-related therapies with stable doses (\>2 months) corticosteroids, 5- ASA drugs, immunomodulators, anti-TNF therapy, anti-integrin and anti-interleukin therapies are permitted

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 subject.
• Hepatitis B or C
• HIV
• Abnormal AST or ALT at screening(defined as \>/= 2x ULN)
• History of cancer including melanoma (with the exception of localized skin cancers) within one year of screening.
• History of colorectal cancer within 5 years
• Investigational drug within one month of treatment
• Pregnant or breast feeding or trying to become pregnant.
• Presence of a rectovaginal or perineal body fistula
• Change in Crohn's immunosuppressive regimen within the 2 months prior to enrollment
• Uncontrolled intestinal Crohn's disease which will require escalation for medical therapy or surgery within 2 months of enrollment
• Severe anal canal disease that is stenotic and requires dilation
• Female participant unwilling to agree to use acceptable contraception methods during participation in study

Sponsors & Collaborators

• Anthony Lembo: lead

Study Sites (1)

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Related Publications (7)

• Kasparek MS, Glatzle J, Temeltcheva T, Mueller MH, Koenigsrainer A, Kreis ME. Long-term quality of life in patients with Crohn's disease and perianal fistulas: influence of fecal diversion. Dis Colon Rectum. 2007 Dec;50(12):2067-74. doi: 10.1007/s10350-007-9006-5.

  PMID: 17680311 BACKGROUND

• Sandborn WJ, Fazio VW, Feagan BG, Hanauer SB; American Gastroenterological Association Clinical Practice Committee. AGA technical review on perianal Crohn's disease. Gastroenterology. 2003 Nov;125(5):1508-30. doi: 10.1016/j.gastro.2003.08.025. No abstract available.

  PMID: 14598268 BACKGROUND

• Schwartz DA, Loftus EV Jr, Tremaine WJ, Panaccione R, Harmsen WS, Zinsmeister AR, Sandborn WJ. The natural history of fistulizing Crohn's disease in Olmsted County, Minnesota. Gastroenterology. 2002 Apr;122(4):875-80. doi: 10.1053/gast.2002.32362.

  PMID: 11910338 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

• 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, Diez MC, Tagarro I, Leselbaum A, Danese S; ADMIRE CD Study Group Collaborators. Long-term Efficacy and Safety of Stem Cell Therapy (Cx601) for Complex Perianal Fistulas in Patients With Crohn's Disease. Gastroenterology. 2018 Apr;154(5):1334-1342.e4. doi: 10.1053/j.gastro.2017.12.020. Epub 2017 Dec 24.

  PMID: 29277560 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

• Molendijk I, van der Meulen-de Jong AE, Verspaget HW, Veenendaal RA, Hommes DW, Bonsing BA, Peeters KCMJ. Standardization of mesenchymal stromal cell therapy for perianal fistulizing Crohn's disease. Eur J Gastroenterol Hepatol. 2018 Oct;30(10):1148-1154. doi: 10.1097/MEG.0000000000001208.

  PMID: 30095479 BACKGROUND

MeSH Terms

Conditions

Crohn Disease

Condition Hierarchy (Ancestors)

Inflammatory Bowel Diseases; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Study Officials

• Amy Lightner, MD

  The Cleveland Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Masking Details: Single
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor-Investigator

Study Record Dates

• First Submitted: August 17, 2020
• First Posted: August 20, 2020
• Study Start: November 19, 2020
• Primary Completion: July 12, 2022
• Study Completion: November 16, 2022
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)