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
  Purpose

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.


Condition Intervention Phase
Crohn's Disease
Biological: autologous mesenchymal stromal cell
Phase 1

Study Type: Interventional
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.

Resource links provided by NLM:


Further study details as provided by Emory University:

Primary Outcome Measures:
  • 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


Secondary Outcome Measures:
  • 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


Estimated Enrollment: 16
Study Start Date: July 2012
Estimated Primary Completion Date: June 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: autologous mesenchymal stromal cells Biological: autologous mesenchymal stromal cell

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01659762

Contacts
Contact: Subra Kugathasan, MD 404-727-7049 skugath@emory.edu
Contact: Tanvi Dhere, MD 404-727-7049 tdhere@emory.edu

Locations
United States, Georgia
Emory University Recruiting
Atlanta, Georgia, United States, 30322
Principal Investigator: Subra Kugathasan, MD         
Sub-Investigator: Tanvi Dhere, MD         
Sponsors and Collaborators
Jacques Galipeau, MD
Atlanta Clinical and Translational Science Institute
Investigators
Principal Investigator: Subra Kugathasan, MD Emory University
Principal Investigator: Tanvi Dhere, MD Emory University
  More Information

Additional Information:
No publications provided

Responsible Party: Jacques Galipeau, MD, Professor, Emory University
ClinicalTrials.gov Identifier: NCT01659762     History of Changes
Other Study ID Numbers: IRB00051454, EPIC001
Study First Received: August 6, 2012
Last Updated: June 25, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by Emory University:
inflammatory bowel disease
Crohn's disease

Additional relevant MeSH terms:
Crohn Disease
Inflammatory Bowel Diseases
Gastroenteritis
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases

ClinicalTrials.gov processed this record on July 24, 2014