We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT01659762
Previous Study | Return to List | Next Study

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01659762
Recruitment Status : Completed
First Posted : August 8, 2012
Last Update Posted : October 17, 2016
Atlanta Clinical and Translational Science Institute
Information provided by (Responsible Party):
Jacques Galipeau, MD, Emory University

Brief Summary:
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 or disease Intervention/treatment Phase
Crohn's Disease Biological: autologous mesenchymal stromal cell Phase 1

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (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.
Study Start Date : July 2012
Actual Primary Completion Date : July 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Crohn's Disease

Arm Intervention/treatment
Experimental: autologous mesenchymal stromal cells Biological: autologous mesenchymal stromal cell

Primary Outcome Measures :
  1. Number of adverse events [ Time Frame: 12 months ]
    Monitoring for adverse events at time of MSC infusion and in 12 months following

Secondary Outcome Measures :
  1. Crohn's disease activity Index (CDAI) [ Time Frame: 12 months ]
    CDAI score before and after intervention will be monitored over 12 months

Information from the National Library of Medicine

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, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01659762

Layout table for location information
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
Sponsors and Collaborators
Emory University
Atlanta Clinical and Translational Science Institute
Layout table for investigator information
Principal Investigator: Subra Kugathasan, MD Emory University
Principal Investigator: Tanvi Dhere, MD Emory University
Layout table for additonal information
Responsible Party: Jacques Galipeau, MD, Professor, Emory University
ClinicalTrials.gov Identifier: NCT01659762    
Other Study ID Numbers: IRB00051454
EPIC001 ( Other Identifier: Other )
First Posted: August 8, 2012    Key Record Dates
Last Update Posted: October 17, 2016
Last Verified: October 2016
Keywords provided by Jacques Galipeau, MD, Emory University:
inflammatory bowel disease
Crohn's disease
Additional relevant MeSH terms:
Layout table for MeSH terms
Crohn Disease
Inflammatory Bowel Diseases
Gastrointestinal Diseases
Digestive System Diseases
Intestinal Diseases