AlloGeneic Human Mesenchymal Stem Cells (hMSC) in PAtients With FistuLizing Crohn's Disease Via PErifistula iNjEctions (GALENE)

NCT02677350 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: 20150965
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Crohn's Disease; Fistulizing Crohn's Disease; Stem Cells

Outcome Measures

Primary Outcomes (1)

• Evaluation of treatment emergent adverse events from the bone marrow-derived allogeneic mesenchymal stem cells implant

  Evaluation of treatment emergent adverse events based on viability, safety and tolerance of the bone marrow-derived allogeneic mesenchymal stem cells implant (MSCs) in fistulizing Crohn's Disease patients, defined as: * Treatment emergent adverse event (AE) rates, infections, hospitalizations or surgical interventions. * Physical examination * Vital signs * Laboratory tests (biochemistry, hematology, urinalysis)

  At each intervention and 7, 10, and 16 months after last intervention

Secondary Outcomes (8)

• Clinically: to assess changes in the Crohn's Disease Activity Index (CDAI), the Perianal Disease Activity Index (PDAI).

  At each intervention and 7, 10, and 16 months after last intervention

• Endoscopic assessment of rectum using a limited simplified endoscopic activity score for Crohn's disease (SES-CD) to evaluate extent and severity of ulcers.

  At each intervention and 7, 10, and 16 months after last intervention

• Radiologic assessment using MRI

  Baseline, Month 4, and 16 months only if fistula closure has not been achieved at month 4

• Evaluate symptomatic patients.

  Month 4 and Month 16

• Evaluate the effect of local treatment with allogeneic bmMSCs using the short Inflammatory Bowel Disease Questionnaire (sIBDQ)

  At each intervention and 7, 10, and 16 months after last intervention

Study Arms (1)

Pilot

EXPERIMENTAL

Twenty (20) subjects will be treated with 20 million (2 x 10\^7) Allogeneic Bone Marrow derived Human Mesenchymal Stem Cells (hMSCs) 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.

Drug: Allogeneic Bone Marrow derived Human Mesenchymal Stem Cells (hMSCs)

Interventions

Allogeneic Bone Marrow derived Human Mesenchymal Stem Cells (hMSCs) DRUG

Peri-fistula injections

Also known as: allo-hMSCs

Pilot

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Provide written informed consent.
• Male and Female subjects ≥ 18 years of age at the time of signing the Informed Consent Form.
• 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.
• If drainage of abscess is needed, it should be done 2 or more weeks prior to onset of therapy.
• Have had Crohn's Disease (CD) diagnosed at least 6 months prior to enrollment based on clinical, endoscopic, anatomic/pathologic and/or radiologic criteria.
• Have a CDAI score \<350.
• 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.
• 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:
• The patient must have been on the anti-TNF for at least 4 months
• The dose of 5-aminosalicylic acid (5-ASA) must have been stable for at least 4 weeks prior to enrollment.
• The dose of steroids must have been stable for at least 2 weeks prior to enrollment.
• The dose of antibiotics must have been stable for at least 2 weeks prior to enrollment.
• 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
• Patient is, in the investigator's opinion, willing and able to comply with the protocol requirements

You may not qualify if:

• In order to participate in this study, a patient Must Not:
• Have a known, serious radiographic contrast allergy (gadolinium in particular)
• Have a hematologic abnormality as evidenced by hematocrit \< 25%, white blood cell \< 2,500/ul or platelet values \< 100,000/ul without another explanation.
• Have liver dysfunction, as evidenced by enzymes (AST and ALT) greater than three times the Upper limit normal.
• 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.
• Bone marrow dysfunction, as evidenced by a 20% or more deviation from normal hematocrit, white blood cell count or platelet values without another explanation.
• Be an organ transplant recipient.
• 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.
• Non-cardiac condition that limits lifespan to \< 1 year.
• 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.
• Have anal dysplasia
• Patients that have received radiation to the pelvic/perianal area.
• Presence of abscess or other collections not drained (revealed by baseline radiologic study).
• Presence of setons unless they are removed before treatment beginning.
• Rectal and/ or anal stenosis that cannot be adequately evaluated for dysplasia by Examination under anesthesia or endoscopy.

Sponsors & Collaborators

• Joshua M Hare: lead

MeSH Terms

Conditions

Crohn Disease

Condition Hierarchy (Ancestors)

Inflammatory Bowel Diseases; Gastroenteritis; Gastrointestinal Diseases; Digestive System Diseases; Intestinal Diseases

Study Officials

• David Kerman, MD

  ISCI / University of Miami

  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: Sponsor

Study Record Dates

• First Submitted: February 1, 2016
• First Posted: February 9, 2016
• Study Start: December 1, 2019
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 1, 2026
• Last Updated: January 27, 2026

Record last verified: 2026-01