NCT02677350

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

A Phase I, Pilot Trial to Evaluate the Safety and Efficacy of Injection of Allogeneic Mesenchymal Bone-Marrow Derived Human Stem Cells in Patients With Fistulizing Crohn's Disease.

• Status: Withdrawn
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Joshua M Hare · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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. As stated in the above response, these first four (4) patients will have an initial safety follow up period of one month prior to proceeding with the treatment of further patients. Follow up: Clinical (CDAI, PDAI, and perianal examination +/- under anesthesia), and endoscopic ultrasound will be performed at the time of each treatment. MRI evaluation will be performed at screening, 4 weeks after the fourth treatment (week 16) and at 16 months, if fistula closure has not been achieved. Clinical evaluation will be performed at months 1, 3, 6, 12. Endoscopic ultrasound and MRI will also be performed as needed for symptomatic patients if an abscess is suspected. Duration of Study participation: 17 Months (Follow-up visits will be at 4, 7, 10 and 16 Months post treatment. The Allo-hMSCs will be supplied from an allogeneic human mesenchymal stem cell source manufactured by the University of Miami.

Conditions

• Crohn's Disease
• Fistulizing Crohn's Disease
• Stem Cells

Interventions

Type	Name	Description
DRUG	Allogeneic Bone Marrow derived Human Mesenchymal Stem Cells (hMSCs)	Peri-fistula injections

Timeline

• Start date: 2019-12-01
• Primary completion: 2025-12-01
• Completion: 2026-12-01
• First posted: 2016-02-09
• Last updated: 2026-01-27

Regulatory

• FDA-regulated drug study