AlloGeneic Human Mesenchymal Stem Cells (hMSC) in PAtients With FistuLizing Crohn's Disease Via PErifistula iNjEctions (GALENE)
To evaluate the role of allogeneic mesenchymal stem cells for treatment of perianal fistulizing Crohn disease. Twenty (20) Crohn's disease patients with complex or multiple perianal or rectovaginal fistulas will be included and will be scheduled to undergo peri-fistula injections after meeting all inclusion/exclusion criteria's at baseline. Following the Pilot Phase of four
- (4) subjects, sixteen (16) subjects are scheduled to undergo perianal injections and after meeting all inclusion/exclusion criteria's, will be evaluated at baseline.
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
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|Eligible Ages||18 Years and Over|
Inclusion Criteria:1. Provide written informed consent. 2. Male and Female subjects ≥ 18 years of age at the time of signing the Informed Consent Form. 3. Subjects with Fistulizing Crohn´s disease with complex perianal fistula, multiple perianal fistulas, or rectovaginal fistula(s). The complex perianal fistula is defined as a trans-sphincteric, supra-sphincteric or an extra-sphincteric tract. Patients with multiple fistulas, "horseshoe" fistula," or any fistula with fecal incontinence as a result of the Crohn's disease itself or because of previous anal fistula surgery that cannot have more surgery are also eligible. 4. If drainage of abscess is needed, it should be done 2 or more weeks prior to onset of therapy. 5. Have had Crohn's Disease (CD) diagnosed at least 6 months prior to enrollment based on clinical, endoscopic, anatomic/pathologic and/or radiologic criteria. 6. Have a CDAI score <350. 7. During the course of the subject's Crohn's disease (CD), subject must have received anti-Tumor Necrosis Factor (TNF) agents or immunomodulators which did not heal the CD fistulas. If anti-TNFs or immunomodulators are contraindicated or led to adverse events, patients must have failed conservative therapy with antibiotics, or setons, or surgical intervention. 8. Subject who are currently receiving anti-TNFs, antibiotics, 5-aminosalicylic acid, azathioprine, 6-mercaptopurine, methotrexate, prednisone, or any similar drugs at the time of enrollment as long as the following criteria are met: 1. The patient must have been on the anti-TNF for at least 4 months 2. The dose of 5-aminosalicylic acid (5-ASA) must have been stable for at least 4 weeks prior to enrollment. 3. The dose of steroids must have been stable for at least 2 weeks prior to enrollment. 4. The dose of antibiotics must have been stable for at least 2 weeks prior to enrollment. 5. The dose of immunomodulators (for example, azathioprine, 6-mercaptopurine, or methotrexate) must have been stable for at least 8 weeks prior to enrollment and the subject on therapy for at least three months prior to enrollment 9. Patient is, in the investigator's opinion, willing and able to comply with the protocol requirements
Exclusion Criteria:In order to participate in this study, a patient Must Not: 1. Have a known, serious radiographic contrast allergy (gadolinium in particular) 2. Have a hematologic abnormality as evidenced by hematocrit < 25%, white blood cell < 2,500/ul or platelet values < 100,000/ul without another explanation. 3. Have liver dysfunction, as evidenced by enzymes (AST and ALT) greater than three times the Upper limit normal. 4. Have a coagulopathy (International Normalized ratio (INR) > 1.3) not due to a reversible cause (i.e., Coumadin). Patients on Coumadin will be withdrawn 5 days before the procedure and confirmed to have an INR < 1.3. Patients who cannot be withdrawn from Coumadin will be excluded from enrollment. 5. Bone marrow dysfunction, as evidenced by a 20% or more deviation from normal hematocrit, white blood cell count or platelet values without another explanation. 6. Be an organ transplant recipient. 7. Clinical history of malignancy within 5 years (i.e., patients with prior malignancy must be disease free for 5 years), except curatively-treated basal cell carcinoma, squamous cell carcinoma, or cervical carcinoma. 8. Non-cardiac condition that limits lifespan to < 1 year. 9. Patients with a highly active luminal CD, i.e., if they meet any of the following criteria:
- - Presence of severe proctitis (prominent friability, spontaneous bleeding, multiple erosions, deep ulcers) or very active luminal disease that requires immediate treatment, revealed by colonoscopy.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|Joshua M Hare|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|David Kerman, MD|
|Principal Investigator Affiliation||ISCI / University of Miami|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
|Overall Status||Not yet recruiting|
The disease, disorder, syndrome, illness, or injury that is being studied.
|Crohn's Disease, Fistulizing Crohn's Disease, Stem Cells|
Twenty (20) subjects will be treated with 20 million (2 x 10^7) allogeneic MSC's total divided into 10 injections of 2 million cells/cm of tract in 0.5 ml volume (for total volume of 5 ml per visit) at 4 week intervals for a maximum of 4 treatment sessions based on the discretion of the endoscopist at the time of injection.. For patients with more than 6 fistula tracts, the largest of the tracts will be injected. The first four
- (4) subjects that receive their initial injections will not be treated less than 5 days apart.
- (4) patients will have an initial safety follow up period of one month prior to proceeding with the treatment of further patients.
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