NCT04791878

Brief Summary

This study plans to enroll 10 patients aged 13-17 years of age with refractory perianal fistulizing disease. Patients will be treated by direct injection to the fistula tract(s) with 75 million allogeneic bone marrow derived mesenchymal stem cells at baseline and again after 3 months if not completely healed.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Apr 2021

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 6, 2021

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 10, 2021

Completed
22 days until next milestone

Study Start

First participant enrolled

April 1, 2021

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2023

Completed
Last Updated

April 16, 2026

Status Verified

April 1, 2026

Enrollment Period

2.6 years

First QC Date

March 6, 2021

Last Update Submit

April 13, 2026

Conditions

Perianal Fistula Due to Crohn's Disease (Disorder)

Keywords

perianalfistulapediatriccrohn's disease

Outcome Measures

Primary Outcomes (2)

  • 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

    Month 3

  • 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

    Month 12

Secondary Outcomes (8)

  • Complete clinical healing

    Month 3

  • Complete clinical healing

    Month 12

  • Partial clinical healing

    Month 3

  • Partial clinical healing

    Month 12

  • Lack of response

    Month 3

  • +3 more secondary outcomes

Study Arms (1)

Mesenchymal stem cells

EXPERIMENTAL

allogeneic bone marrow derived mesenchymal stem cells

Drug: Mesenchymal stem cells

Interventions

Mesenchymal stem cellsDRUG

Direct injection of 75 million allogeneic bone marrow derived mesenchymal stem cells at baseline and again after 3 months if not completely healed

Mesenchymal stem cells

Eligibility Criteria

Age13 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Males and females aged 13-17 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 corticosteroids, 5- ASA drugs, immunomodulators, anti-TNF therapy, anti-integrin and anti-interleukin therapies are permitted.
  • Agree to use birth control or abstinence to avoid pregnancy during the study

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 colon cancer in the past two years, or treatment for other cancers within the last 6 months.
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Related Publications (12)

  • Molodecky NA, Soon IS, Rabi DM, Ghali WA, Ferris M, Chernoff G, Benchimol EI, Panaccione R, Ghosh S, Barkema HW, Kaplan GG. Increasing incidence and prevalence of the inflammatory bowel diseases with time, based on systematic review. Gastroenterology. 2012 Jan;142(1):46-54.e42; quiz e30. doi: 10.1053/j.gastro.2011.10.001. Epub 2011 Oct 14.

    PMID: 22001864BACKGROUND

  • Hellers G, Bergstrand O, Ewerth S, Holmstrom B. Occurrence and outcome after primary treatment of anal fistulae in Crohn's disease. Gut. 1980 Jun;21(6):525-7. doi: 10.1136/gut.21.6.525.

    PMID: 7429313BACKGROUND

  • 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: 17680311BACKGROUND

  • 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: 14598268BACKGROUND

  • 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: 11910338BACKGROUND

  • Chaparro M, Zanotti C, Burgueno P, Vera I, Bermejo F, Marin-Jimenez I, Yela C, Lopez P, Martin MD, Taxonera C, Botella B, Pajares R, Ponferrada A, Calvo M, Algaba A, Perez L, Casis B, Mate J, Orofino J, Lara N, Garcia-Losa M, Badia X, Gisbert JP. Health care costs of complex perianal fistula in Crohn's disease. Dig Dis Sci. 2013 Dec;58(12):3400-6. doi: 10.1007/s10620-013-2830-7. Epub 2013 Sep 13.

    PMID: 24026400BACKGROUND

  • Aguilera-Castro L, Ferre-Aracil C, Garcia-Garcia-de-Paredes A, Rodriguez-de-Santiago E, Lopez-Sanroman A. Management of complex perianal Crohn's disease. Ann Gastroenterol. 2017;30(1):33-44. doi: 10.20524/aog.2016.0099. Epub 2016 Oct 27.

    PMID: 28042236BACKGROUND

  • Adler J, Dong S, Eder SJ, Dombkowski KJ; ImproveCareNow Pediatric IBD Learning Health System. Perianal Crohn Disease in a Large Multicenter Pediatric Collaborative. J Pediatr Gastroenterol Nutr. 2017 May;64(5):e117-e124. doi: 10.1097/MPG.0000000000001447.

    PMID: 27801750BACKGROUND

  • Kugathasan S, Judd RH, Hoffmann RG, Heikenen J, Telega G, Khan F, Weisdorf-Schindele S, San Pablo W Jr, Perrault J, Park R, Yaffe M, Brown C, Rivera-Bennett MT, Halabi I, Martinez A, Blank E, Werlin SL, Rudolph CD, Binion DG; Wisconsin Pediatric Inflammatory Bowel Disease Alliance. Epidemiologic and clinical characteristics of children with newly diagnosed inflammatory bowel disease in Wisconsin: a statewide population-based study. J Pediatr. 2003 Oct;143(4):525-31. doi: 10.1067/s0022-3476(03)00444-x.

    PMID: 14571234BACKGROUND

  • Markowitz J, Grancher K, Rosa J, Simpser E, Aiges H, Daum F. Highly destructive perianal disease in children with Crohn's disease. J Pediatr Gastroenterol Nutr. 1995 Aug;21(2):149-53. doi: 10.1097/00005176-199508000-00005.

    PMID: 7472900BACKGROUND

  • Vernier-Massouille G, Balde M, Salleron J, Turck D, Dupas JL, Mouterde O, Merle V, Salomez JL, Branche J, Marti R, Lerebours E, Cortot A, Gower-Rousseau C, Colombel JF. Natural history of pediatric Crohn's disease: a population-based cohort study. Gastroenterology. 2008 Oct;135(4):1106-13. doi: 10.1053/j.gastro.2008.06.079. Epub 2008 Jul 3.

    PMID: 18692056BACKGROUND

  • Kappelman MD, Moore KR, Allen JK, Cook SF. Recent trends in the prevalence of Crohn's disease and ulcerative colitis in a commercially insured US population. Dig Dis Sci. 2013 Feb;58(2):519-25. doi: 10.1007/s10620-012-2371-5. Epub 2012 Aug 29.

    PMID: 22926499BACKGROUND

MeSH Terms

Conditions

FistulaCrohn Disease

Condition Hierarchy (Ancestors)

Pathological Conditions, AnatomicalPathological Conditions, Signs and SymptomsInflammatory Bowel DiseasesGastroenteritisGastrointestinal DiseasesDigestive System DiseasesIntestinal Diseases

Study Officials

  • Amy 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
SPONSOR INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

March 6, 2021

First Posted

March 10, 2021

Study Start

April 1, 2021

Primary Completion

November 1, 2023

Study Completion

November 1, 2023

Last Updated

April 16, 2026

Record last verified: 2026-04

Locations

US(1)