A Phase I Study Evaluating Autologous Bone Marrow Derived Mesenchymal Stromal for Crohn's Disease. (EPIC/MSC/IBD)

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 by Emory University
Sponsor:
Collaborator:
Atlanta Clinical and Translational Science Institute
Information provided by (Responsible Party):
Jacques Galipeau, MD, Emory University
ClinicalTrials.gov Identifier:
NCT01659762
First received: August 6, 2012
Last updated: June 25, 2014
Last verified: June 2014

August 6, 2012
June 25, 2014
July 2012
June 2015   (final data collection date for primary outcome measure)
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
Same as current
Complete list of historical versions of study NCT01659762 on ClinicalTrials.gov Archive Site
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
Same as current
Not Provided
Not Provided
 
A Phase I Study Evaluating Autologous Bone Marrow Derived Mesenchymal Stromal for Crohn's Disease.
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.

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.

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.

Interventional
Phase 1
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Crohn's Disease
Biological: autologous mesenchymal stromal cell
Experimental: autologous mesenchymal stromal cells
Intervention: Biological: autologous mesenchymal stromal cell
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
16
Not Provided
June 2015   (final data collection date for primary outcome measure)

Inclusion Criteria

  • Men and women 18-65 years of age.
  • Patient must have had CD for at least 3 months from the time of initial diagnosis. The diagnosis of CD must have been confirmed by endoscopic and histological evidence.
  • Patients must have active Crohn's disease as defined by a Crohns Disease Activity Index (CDAI) score between >220 at screening and baseline.
  • Patients should have no need for immediate surgery (i.e. due to obstruction, strictures, active abscess or perforations ).
  • Subjects must be refractory (defined as lack of response for at least 3 months) to immunomodulators (including 6-mercaptopurine and azathioprine and methotrexate) or anti-TNF therapy at present or some point in the course of their disease. Lack of response is defined by failure to reduce the CDAI score by at least 70 points.
  • The following medications will be allowed: mesalamine and prednisone (stable dose for at least 2 weeks prior to enrollment).
  • Subjects on anti TNF therapy will require a minimum of 4 weeks washout period prior to screening.
  • Subjects on non-steroidal analgesics require a minimum of 2 weeks washout period prior to screening
  • If female and of child-bearing age, patient must be non-pregnant, non-breastfeeding, and use adequate contraception;
  • Patient is willing to participate in the study and has signed the informed consent.
Both
18 Years to 65 Years
No
Contact: Subra Kugathasan, MD 404-727-7049 skugath@emory.edu
Contact: Tanvi Dhere, MD 404-727-7049 tdhere@emory.edu
United States
 
NCT01659762
IRB00051454, EPIC001
Yes
Jacques Galipeau, MD, Emory University
Jacques Galipeau, MD
Atlanta Clinical and Translational Science Institute
Principal Investigator: Subra Kugathasan, MD Emory University
Principal Investigator: Tanvi Dhere, MD Emory University
Emory University
June 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP