Study of Mesenchymal Stem Cells for the Treatment of Ileal Pouch Fistula's in Participants With Crohn's Disease (IPAAF)
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|ClinicalTrials.gov Identifier: NCT04519684|
Recruitment Status : Recruiting
First Posted : August 20, 2020
Last Update Posted : April 5, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Ileal Pouch Crohn Disease||Drug: Mesenchymal stem cells Other: Placebo||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 participants|
|Intervention Model:||Crossover Assignment|
|Official Title:||A Phase IB/IIA Study of Allogeneic Bone Marrow Derived Mesenchymal Stem Cells for the Treatment of Ileal Anal Anastomosis and Ileal Pouch Fistulas in the Setting of Crohn's Disease of the Pouch|
|Actual Study Start Date :||October 28, 2020|
|Estimated Primary Completion Date :||October 2022|
|Estimated Study Completion Date :||October 2022|
Experimental: Mesenchymal stem cells
Direct injection of allogeneic bone marrow derived mesenchymal stem cells at a dose of 75 million cells into the ileal pouch fistula(s) at baseline with a possible repeat injection at 3 months if not completely healed from the first injection.
Drug: Mesenchymal stem cells
Allogeneic bone marrow derived mesenchymal stem cells
Placebo Comparator: Placebo
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 allogeneic bone marrow derived mesenchymal stem cells at a dose of 75 million cells into ileal pouch fistula(s).
- Treatment related adverse events [ Time Frame: Month 6 ]Number of participants with treatment related adverse events post-injection of 75 million allogeneic bone marrow derived MSC's for the treatment of medically refractory pouch fistulizing disease as assessed by protocol CCF-Stem Cells IBD-002
- Complete clinical healing [ Time Frame: Month 6, Month 12 ]
Number of participants with complete clinical healing post-injection of 75 million allogeneic bone marrow derived MSC's for the treatment of medically refractory pouch fistulizing disease in the setting of Crohn's disease of the pouch.
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
- Partial healing [ Time Frame: Month 6, Month 12 ]
Number of participants with partial clinical healing,post-injection of 75 million allogeneic bone marrow derived MSC's for the treatment of medically refractory pouch fistulizing disease in the setting of Crohn's disease
Partial 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
- Lack of response [ Time Frame: Month 6, Month 12 ]
Number of participants with lack of response post-injection of 75 million allogeneic bone marrow derived MSC's for the treatment of medically refractory pouch fistulizing disease 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
- Worsening disease [ Time Frame: Month 6, Month 12 ]
Number of participants with worsening disease-injection of 75 million allogeneic bone marrow derived MSC's for the treatment of medically refractory pouch fistulizing disease 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
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|Ages Eligible for Study:||18 Years to 75 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
- Men and women 18-75 years of age who have undergone an ileal pouch anal anastomosis at least 6 months prior who have developed a clinical diagnosis of Crohn's disease of the pouch as determined by a combination of clinical symptoms, pouchoscopy with biopsy, enterography.
Single and multi-tract (up to 2 internal and 3 external openings) fistula tract arising from the ileal pouch, ileal anal anastomosis, or anal canal distal to anastomosis that travels to the perianal skin, perineal body, or vagina. Patients with fistulas that arise from the pouch, anastomosis, or anal canal distal to the anastomosis will both be included in enrollment.
- Acceptable internal openings and tract locations for the fistula to arise from include the ileal pouch body, the pouch anal anastomosis, and the anal canal distal to the anastomosis.
- Acceptable external openings and tract locations for the fistula to arise from include the perianal skin, perineal body, and/or the vaginal wall.
- Concurrent Crohn's related therapies with stable doses (>2 months) corticosteroids, 5-ASA drugs, immunomodulators, anti-TNF therapy, anti-integrin and anti-interleukin are permitted.
- Have failed conventional medical therapies described above, defined as a lack of response to systemic immune suppression (e.g. azathioprine, methotrexate, 6-mercaptopurine) or biologic (e.g. anti-TNF, anti-integrin, anti-interleukin) therapies to treat fistulizing CD for at least 2 months
- Have no contraindications to MR evaluations: e.g. pacemaker or magnetically active metal fragments, claustrophobia
- Competent and able to provide written informed consent
- Ability to comply with protocol.
- Inability to give informed consent.
- Severe antibiotic refractory pouchitis
- Severe cuffitis refractory to antibiotics
- Change in medical management for CD in the previous 2 months or changes anticipated in the next 2 months
- 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
- Abnormal CBC at screening
i. Platelets <50 kg/uL or greater than 1.5 million kg/uL ii. WBC <50 x kg/uL iii. Hbg <7.0 g/dL d. 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 thirty (30) days of baseline
- Pregnant or breast feeding or trying to become pregnant
Branching fistula tract that has > 2 internal openings or 3 external openings,
- Subjects with greater than 3 blind/branching tracts are excluded
- Fistula tracts on the left and/or right side are allowed
- Allergic to local anesthetics
- Unwilling to agree to use acceptable contraception methods during participation in study
- Subjects with a non-abscessed chronic cavity will not be included in enrollment
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): NCT04519684
|Contact: Allison Bayles, AAemail@example.com|
|Contact: Alex VanDenBossche, BSNfirstname.lastname@example.org|
|United States, Ohio|
|Cleveland, Ohio, United States, 44195|
|Contact: Allison Bayles|
|Principal Investigator:||Amy Lightner, MD||The Cleveland Clinic|
|Responsible Party:||Amy Lightner, Sponsor-investigator, The Cleveland Clinic|
|Other Study ID Numbers:||
|First Posted:||August 20, 2020 Key Record Dates|
|Last Update Posted:||April 5, 2022|
|Last Verified:||April 2022|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
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