A Phase I Study Evaluating Autologous Bone Marrow Derived Mesenchymal Stromal for Crohn's Disease.

Crohn's Disease Clinical Trial

— EPIC/MSC/IBD  

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01659762
• Other study ID #: IRB00051454
• Secondary ID: EPIC001
• Status: Completed
• Phase: Phase 1
• First received: August 6, 2012
• Last updated: October 13, 2016
• Start date: July 2012

Study information

• Verified date: October 2016
• Source: Emory University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. primary completion date: July 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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.

Study Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Crohn Disease
• Crohn's Disease

Intervention

Biological:
• autologous mesenchymal stromal cell

Locations

Country	Name	City	State
United States	Emory University	Atlanta	Georgia

Sponsors (2)

Lead Sponsor	Collaborator
Emory University	Atlanta Clinical and Translational Science Institute

Country where clinical trial is conducted

United States, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of adverse events	Monitoring for adverse events at time of MSC infusion and in 12 months following	12 months	Yes
Secondary	Crohn's disease activity Index (CDAI)	CDAI score before and after intervention will be monitored over 12 months	12 months	No

External Links

•   Study posting on Crohn's and Colitis Foundation of America (CCFA) website