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

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ClinicalTrials.gov Identifier: NCT04791878
Recruitment Status : Recruiting
First Posted : March 10, 2021
Last Update Posted : April 19, 2021
Sponsor:
Information provided by (Responsible Party):
Amy Lightner, The Cleveland Clinic

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.

Condition or disease Intervention/treatment Phase
Perianal Fistula Due to Crohn's Disease (Disorder) Drug: Mesenchymal stem cells Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Study of Adult Allogeneic Bone Marrow Derived Mesenchymal Stem Cells for Pediatric Perianal Fistulizing Crohn's Disease
Actual Study Start Date : April 1, 2021
Estimated Primary Completion Date : April 1, 2023
Estimated Study Completion Date : April 1, 2023

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Mesenchymal stem cells
allogeneic bone marrow derived mesenchymal stem cells
Drug: Mesenchymal stem cells
Direct injection of 75 million allogeneic bone marrow derived mesenchymal stem cells at baseline and again after 3 months if not completely healed




Primary Outcome Measures :
  1. Treatment related adverse events [ Time Frame: Month 3 ]
    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

  2. Treatment related adverse events [ Time Frame: Month 12 ]
    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


Secondary Outcome Measures :
  1. Complete clinical healing [ Time Frame: Month 3 ]

    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


  2. Complete clinical healing [ Time Frame: Month 12 ]

    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


  3. Partial clinical healing [ Time Frame: Month 3 ]

    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


  4. Partial clinical healing [ Time Frame: Month 12 ]

    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


  5. Lack of response [ Time Frame: Month 3 ]

    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


  6. Lack of response [ Time Frame: Month 12 ]

    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


  7. Worsening of disease [ Time Frame: Month 3 ]

    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


  8. Worsening of disease [ Time Frame: Month 12 ]

    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




Information from the National Library of Medicine

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Ages Eligible for Study:   13 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  1. Males and females aged 13-17 with a diagnosis of Crohn's disease for at least six months duration.
  2. Single and Multi-tract Perianal fistula, with or without previous failed surgical repair.
  3. Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia
  4. Ability to comply with protocol
  5. Competent and able to provide written informed consent
  6. Concurrent Crohn's-related therapies with stable doses corticosteroids, 5- ASA drugs, immunomodulators, anti-TNF therapy, anti-integrin and anti-interleukin therapies are permitted.
  7. Agree to use birth control or abstinence to avoid pregnancy during the study

Exclusion Criteria

  1. Inability to give informed consent.
  2. 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.
  3. Specific exclusions:

    1. Hepatitis B or C
    2. HIV
    3. Abnormal AST or ALT at screening
  4. History of colon cancer in the past two years, or treatment for other cancers within the last 6 months.
  5. Investigational drug within one month of treatment
  6. Pregnant or breast feeding or trying to become pregnant.
  7. Presence of a rectovaginal or perineal body fistula
  8. Change in Crohn's immunosuppressive regimen within the 2 months prior to enrollment
  9. Uncontrolled intestinal Crohn's disease which will require escalation for medical therapy or surgery within 2 months of enrollment
  10. Severe anal canal disease that is stenotic and requires dilation
  11. Female participant unwilling to agree to use acceptable contraception methods during participation in study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04791878


Contacts
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Contact: Caroline Matyas, BSPH 216-212-0746 matyasc@ccf.org

Locations
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United States, Ohio
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
Contact: Caroline Matyas, BSPH    216-212-0746    matyasc@ccf.org   
Principal Investigator: Amy Lightner, MD         
Sponsors and Collaborators
Amy Lightner
Investigators
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Principal Investigator: Amy Lightner, MD The Cleveland Clinic
Publications:

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Responsible Party: Amy Lightner, MD, The Cleveland Clinic
ClinicalTrials.gov Identifier: NCT04791878    
Other Study ID Numbers: Peds-PF
First Posted: March 10, 2021    Key Record Dates
Last Update Posted: April 19, 2021
Last Verified: April 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Amy Lightner, The Cleveland Clinic:
perianal
fistula
pediatric
crohn's disease
Additional relevant MeSH terms:
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Crohn Disease
Fistula
Disease
Inflammatory Bowel Diseases
Gastroenteritis
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases
Pathological Conditions, Anatomical
Pathologic Processes