NCT01764100

Mesenchymal Stromal Cells (MSCs) for the Treatment of Graft Versus Host Disease (GVHD)

Mesenchymal Stromal Cells (MSC) for the Treatment of Severe (Grade II-IV) Steroid-resistant Graft Versus Host Disease (GVHD): a Phase I Trial

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Ettore Biagi, MD · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This is a bicentric, prospective, non randomized study. Pediatric and adult patients will be treated. Rationale: MSC have shown promising effects by reversal of severe therapy-resistant acute GvHD. As a common therapeutic line of action is not shared for steroid resistant GVHD, it is important to establish the toxicity and the feasibility of preparation and infusion of third party MSCs for acute steroid resistant GVHD and acute phases of chronic steroid resistant GVHD. A total of 10 patients (pediatric and adults) need to be enrolled in the study. Patients who present clinical signs of either acute or chronic steroid resistant GVHD will receive by intravenous infusion at least two fixed doses of mesenchymal stem cells with 5 to 7 days of interval one from the other, derived from HLA unrelated donor different from the HSC donor (third party donor) regardless of the rate of HLA mismatch. Primary objectives are to establish the feasibility and the toxicity of preparation and infusions of third party MSCs for the treatment of steroid resistant acute and acute phases of chronic grade II-IV GVHD. Secondary objectives are: 1. To document the efficacy of MSC infusion in steroid resistant acute and acute phases of chronic GVHD grade II-IV. 2. To document the rate of GVHD recurrence in MSCs infused patients. 3. To document relapse of hematological malignancies post MSC infusions in patients undergoing MSCs treatment for steroid refractory GvHD. 4. To document the overall survival of MSC infused patients for steroid refractory GvHD.

Conditions

• Graft vs Host Disease

Interventions

Type	Name	Description
GENETIC	Mesenchymal stromal cells	Mesenchymal stromal cells (MSC) intravenous infusion at least two fixed doses of mesenchymal stem cells (1 ± 0.5 x 106/kg recipient body weight for each injection) with 5 to 7 days of interval one from the other, derived from HLA unrelated donor different from the HSC donor (third party donor).

Timeline

• Start date: 2009-09-01
• Primary completion: 2013-05-01
• Completion: 2013-09-01
• First posted: 2013-01-09
• Last updated: 2013-01-09

Locations

3 sites across 1 country: Italy