AzTrials
AzTrials

Perianal Fistula Due to Crohn's Disease (Disorder)Phase 1NCT04791878

Study of Mesenchymal Stem Cells for Pediatric Perianal Fistulizing Crohn's Disease

A Phase 1 study testing Mesenchymal stem cells in people with Perianal Fistula Due to Crohn's Disease (Disorder), run by Anthony Lembo at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 13 to 17 years
What is being tested?
Mesenchymal stem cells
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteUnited States
Study dates
1 Apr 2021to 1 Nov 2023
Phase
Phase 1
Are there results?
None posted in the registry

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 13 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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

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 subject.
  • Specific exclusions:
  • Hepatitis B or C
  • HIV
  • Abnormal AST or ALT at screening (defined as >/+2x ULN)
Show all 14Show fewer
  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Cleveland Clinic

    Cleveland, Ohio, 44195

Other trials in United States

About this study

Official title: A Phase I Study of Adult Allogeneic Bone Marrow Derived Mesenchymal Stem Cells for Pediatric Perianal Fistulizing Crohn's Disease

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.

Read the detailed description

Crohn's disease (CD), a chronic transmural inflammatory disease of the gastrointestinal tract, continues to increase in incidence for unknown reasons. According to population based studies, at least 26% of patients with CD will develop perianal fistulas in the first two decades following diagnosis, particularly those with colonic and rectal involvement. These patients experience significant morbidity due to pain, persistent drainage, recurrent perianal sepsis, and ongoing need to access medical care resulting in increased costs and impaired quality of life. Onset of Crohn's disease in childhood is associated with even more aggressive perianal fistula development, with fistulas occurring in as many as 20-31% of children within 5-7 years after Crohn's disease diagnosis. Based on national estimates of pediatric Crohn's disease prevalence, this suggests that there are more than 10,000 children with perianal fistulas due to Crohn's disease in the United States.

This study plans to enroll 10 patients (aged 13-17 years) with refractory perianal fistulizing disease. The next step in management for these patients would be a mucosal tissue flap, temporary stoma, or proctectomy with permanent ostomy.

Patients will be treated by direct injection of 75 million allogeneic bone marrow derived mesenchymal stem cells at baseline and again after 3 months if not completely healed. Patients will be followed for a total of 12 months post initial injection.

Being tested
Mesenchymal stem cells
Keywords
perianal, fistula, pediatric, crohn's disease
Medical subject headings
Fistula, Crohn Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 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

Study groups in NCT04791878
Study groupRoleWhat they receive
Mesenchymal stem cellsExperimentalallogeneic bone marrow derived mesenchymal stem cells
Receives: Drug: Mesenchymal stem cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: Month 3

  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

    Measured: Month 12

Secondary outcomes (8)

  1. Complete clinical healing

    Number of participants with complete clinical healing 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.

    Complete Healing is defined as:

    Radiographic Healing: MRI with an absence of a fluid collection >2 cm in 3 of 3 dimensions, lack of edema, inflammation or sign of active inflammatory response. A remnant scar of a fistula tract may remain

    Clinical Healing: 100% cessation of drainage on both clinical exam with deep palpation and per patient report and epithelization of the external fistula opening

    Measured: Month 3

  2. Complete clinical healing

    Number of participants with complete clinical healing 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.

    Complete Healing is defined as:

    Radiographic Healing: MRI with an absence of a fluid collection >2 cm in 3 of 3 dimensions, lack of edema, inflammation or sign of active inflammatory response. A remnant scar of a fistula tract may remain

    Clinical Healing: 100% cessation of drainage on both clinical exam with deep palpation and per patient report and epithelization of the external fistula opening

    Measured: Month 12

  3. Partial clinical healing

    Number of participants with partial clinical healing 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

    Partial clinical healing is defined as:

    Radiographic Healing: MRI with an absence of a fluid collection >2 cm in 2 of 3 dimensions, lack of edema, inflammation or sign of active inflammatory response. A remnant scar of a fistula tract may remain

    Clinical healing: Greater than or equal to 50 % cessation of drainage on both clinical exam with deep palpation and per patient report and epithelization of the external fistula opening

    Measured: Month 3

  4. Partial clinical healing

    Number of participants with partial clinical healing 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

    Partial clinical healing is defined as:

    Radiographic Healing: MRI with an absence of a fluid collection >2 cm in 2 of 3 dimensions, lack of edema, inflammation or sign of active inflammatory response. A remnant scar of a fistula tract may remain

    Clinical healing: Greater than or equal to 50 % cessation of drainage on both clinical exam with deep palpation and per patient report and epithelization of the external fistula opening

    Measured: Month 12

  5. Lack of response

    Number of participants with lack of response 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

    Lack of response is defined as:

    Radiographic and Clinical healing which does not meet the threshold for Partial Healing

    Measured: Month 3

  6. Lack of response

    Number of participants with lack of response 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

    Lack of response is defined as:

    Radiographic and Clinical healing which does not meet the threshold for Partial Healing

    Measured: Month 12

  7. Worsening of disease

    Number of participants with worsening of disease 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

    Worsening disease is defined as:

    Radiographic: MRI with a fluid collection >2 cm in 2 of 3 dimensions, edema, inflammation or sign of active inflammatory response. An increased number of tracts may be seen, or increased branching from the primary tract,

    Clinical: Increased drainage per patient report and on clinical exam

    Measured: Month 3

  8. Worsening of disease

    Number of participants with worsening of disease 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

    Worsening disease is defined as:

    Radiographic: MRI with a fluid collection >2 cm in 2 of 3 dimensions, edema, inflammation or sign of active inflammatory response. An increased number of tracts may be seen, or increased branching from the primary tract,

    Clinical: Increased drainage per patient report and on clinical exam

    Measured: Month 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Mar 2021
  2. Study start1 Apr 2021
  3. Primary completion1 Nov 2023
  4. Study completion1 Nov 2023

Why it stopped: PI left institution

Resources

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
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04791878
Sponsor's protocol ID
21-134
Status last verified
Apr 2026
First submitted
6 Mar 2021
Last updated
16 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 22001864

  • 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.

    PubMed 7429313

  • 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.

    PubMed 17680311

  • 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.

    PubMed 14598268

  • 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.

    PubMed 11910338

  • 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.

    PubMed 24026400

  • 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.

    PubMed 28042236

  • 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.

    PubMed 27801750

  • 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.

    PubMed 14571234

  • 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.

    PubMed 7472900

  • 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.

    PubMed 18692056

  • 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.

    PubMed 22926499

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Mesenchymal stem cells in people with Perianal Fistula Due to Crohn's Disease (Disorder), run by Anthony Lembo 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 7 inclusion and 14 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: stopped early, as of the sponsor's last update on 16 Apr 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?

The study was stopped early (reason given: PI left institution). Results are not available in the source record.

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?

Treatment related adverse events (time frame: Month 3). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04791878, which also lists 12 linked publications.

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 16 Apr 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in