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

Phase 1

Completed

• Conditions: Crohn's Disease
• Interventions: Biological: autologous mesenchymal stromal cell

• Registration Number: NCT01659762
• Lead Sponsor: 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.

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 Timeline

• Study Start: 2012-07
• Study First Submitted: 2012-08-06
• Study First Submitted QC: 2012-08-07
• Study First Posted: 2012-08-08
• Primary Completion: 2015-07
• Status Verified: 2016-10
• Last Update Submitted: 2016-10-13
• Last Update Posted: 2016-10-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
autologous mesenchymal stromal cells	autologous mesenchymal stromal cell	-

Primary Outcome Measures

Name	Time	Method
Number of adverse events	12 months	Monitoring for adverse events at time of MSC infusion and in 12 months following

Secondary Outcome Measures

Name	Time	Method
Crohn's disease activity Index (CDAI)	12 months	CDAI score before and after intervention will be monitored over 12 months

Trial Locations

Locations (1)

Emory University; 🇺🇸 Atlanta, Georgia, United States