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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
Sponsor:
Collaborator:
Atlanta Clinical and Translational Science Institute
Information provided by (Responsible Party):
Jacques Galipeau, MD, Emory University

Tracking Information
First Submitted Date  ICMJE August 6, 2012
First Posted Date  ICMJE August 8, 2012
Last Update Posted Date October 17, 2016
Study Start Date  ICMJE July 2012
Actual Primary Completion Date July 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 7, 2012)
Number of adverse events [ Time Frame: 12 months ]
Monitoring for adverse events at time of MSC infusion and in 12 months following
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 7, 2012)
Crohn's disease activity Index (CDAI) [ Time Frame: 12 months ]
CDAI score before and after intervention will be monitored over 12 months
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase I Study Evaluating Autologous Bone Marrow Derived Mesenchymal Stromal for Crohn's Disease.
Official Title  ICMJE 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.
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.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Crohn's Disease
Intervention  ICMJE Biological: autologous mesenchymal stromal cell
Study Arms  ICMJE Experimental: autologous mesenchymal stromal cells
Intervention: Biological: autologous mesenchymal stromal cell
Publications * Dhere T, Copland I, Garcia M, Chiang KY, Chinnadurai R, Prasad M, Galipeau J, Kugathasan S. The safety of autologous and metabolically fit bone marrow mesenchymal stromal cells in medically refractory Crohn's disease - a phase 1 trial with three doses. Aliment Pharmacol Ther. 2016 Sep;44(5):471-81. doi: 10.1111/apt.13717. Epub 2016 Jul 7.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 7, 2012)
16
Original Estimated Enrollment  ICMJE Same as current
Study Completion Date  ICMJE Not Provided
Actual Primary Completion Date July 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01659762
Other Study ID Numbers  ICMJE IRB00051454
EPIC001 ( Other Identifier: Other )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Jacques Galipeau, MD, Emory University
Study Sponsor  ICMJE Emory University
Collaborators  ICMJE Atlanta Clinical and Translational Science Institute
Investigators  ICMJE
Principal Investigator: Subra Kugathasan, MD Emory University
Principal Investigator: Tanvi Dhere, MD Emory University
PRS Account Emory University
Verification Date October 2016

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