A Phase I Study Evaluating Autologous Bone Marrow Derived Mesenchymal Stromal for Crohn's Disease. (EPIC/MSC/IBD)
In this Phase I trial the investigators intend to show safety and tolerability of autologous MSC, expanded using a non-xenogeneic, human component platelet lysate expansion media. Fresh, non cryopreserved, autologous MSCs will delivered intravenously as a single bolus dose in a dose escalation phase I study. The investigators intend to test whether the product is clinically safe in adults (18-65 years old) with CD and to determine maximal deliverable dose. Secondary endpoint will monitor effectiveness using CDAI as an endpoint.
Biological: autologous mesenchymal stromal cell
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I Study Evaluating Safety and Tolerability of Autologous Bone Marrow Derived Mesenchymal Stromal (MSC) Cells in Adults With Moderate to Severe Crohn's Disease.|
- Number of adverse events [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]Monitoring for adverse events at time of MSC infusion and in 12 months following
- Crohn's disease activity Index (CDAI) [ Time Frame: 12 months ] [ Designated as safety issue: No ]CDAI score before and after intervention will be monitored over 12 months
|Study Start Date:||July 2012|
|Estimated Primary Completion Date:||December 2015 (Final data collection date for primary outcome measure)|
|Experimental: autologous mesenchymal stromal cells||Biological: autologous mesenchymal stromal cell|
EPIC MSC/IBD is made up of autologous marrow-derived mesenchymal stromal cells ex vivo expanded numerically for approximately 14 days using pooled human Platelet Lysate (phPL), harvested on the day of infusion, washed and suspended at a concentration of 4 million cells/ml in Plasmalyte A with 0.5% human serum albumin. This is a phase I dose-escalation, open label, non-randomized, non-placebo controlled, single group assignment study to evaluate the safety and tolerability of a single intravenous infusion of EPIC MSC2011-001. EPIC EPIC MSC/IBD will be infused intravenously and will be administered at one of three dose levels: (Tier 1) 2 million cells/kg patient body weight; (Tier 2) 5 million cells/kg, and (Tier 3) 10 million cells/kg. This Phase I clinical trial will enroll 16-20 subjects with moderate to severe Crohn's. The duration of this study for each patient is 12 weeks. The investigators anticipate that this study will be completed within 2 years of commencement.
Primary objective: To describe and compare the safety and tolerability of a single infusion of fresh autologous bone marrow derived Mesenchymal stromal cells infused to patients with moderate to severe Crohn's disease.
Secondary objective: Efficacy of autologous bone marrow derived Mesenchymal stromal cells infusion to patients with moderate to severe Crohn's disease as assessed through disease activity index, and quality of life index.
Safety variables:Adverse events (AEs),Laboratory parameters (hematology, biochemistry, urinalysis), Vital signs.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01659762
|Contact: Subra Kugathasan, MDfirstname.lastname@example.org|
|Contact: Tanvi Dhere, MDemail@example.com|
|United States, Georgia|
|Atlanta, Georgia, United States, 30322|
|Principal Investigator: Subra Kugathasan, MD|
|Sub-Investigator: Tanvi Dhere, MD|
|Principal Investigator:||Subra Kugathasan, MD||Emory University|
|Principal Investigator:||Tanvi Dhere, MD||Emory University|