Mesenchymal Stromal Cells (MSCs) for the Treatment of Graft Versus Host Disease (GVHD) (MSC-GvHD)
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:
- To document the efficacy of MSC infusion in steroid resistant acute and acute phases of chronic GVHD grade II-IV.
- To document the rate of GVHD recurrence in MSCs infused patients.
- To document relapse of hematological malignancies post MSC infusions in patients undergoing MSCs treatment for steroid refractory GvHD.
- To document the overall survival of MSC infused patients for steroid refractory GvHD.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Mesenchymal Stromal Cells (MSC) for the Treatment of Severe (Grade II-IV) Steroid-resistant Graft Versus Host Disease (GVHD): a Phase I Trial|
- Any toxic effect reported during MSCs infusion or in the subsequent 10 days by clinical monitoring [ Time Frame: During MSCs infusion or in the subsequent 10 days ] [ Designated as safety issue: Yes ]
- Feasibility as the possibility of producing adequate lots of patient dedicated MSCs for any patients presenting with steroid resistant GVHD [ Time Frame: three years ] [ Designated as safety issue: No ]
- Number of patients with GvHD resolution [ Time Frame: One month ] [ Designated as safety issue: No ]
- Complete resolution of GvHD: Control of all signs and symptoms attributed to acute GvHD
- Partial resolution of GvHD: Control of some signs and symptoms attributed to acute GvHD with an improvement of overall grade
- Refractory GvHD: No change in signs or symptoms of GvHD within 10 days of MSC infusion.
- Worsening GvHD: Any progress of GvHD signs and symptoms that increase overall grade. a and b will be defined as response. c and d will be defined as no response.
- Determination of recurrence of GvHD [ Time Frame: After 1 month from MSCs infusion ] [ Designated as safety issue: No ]
- Relapse of haematological disease [ Time Frame: Every three months ] [ Designated as safety issue: No ]
- Survival [ Time Frame: Every three months ] [ Designated as safety issue: No ]
|Study Start Date:||September 2009|
|Estimated Study Completion Date:||September 2013|
|Estimated Primary Completion Date:||May 2013 (Final data collection date for primary outcome measure)|
Experimental: Mesenchymal Stromal Cells (MSC)
Intravenous injections for a dose of 1 ± 0.5 x 106 MSC/kg recipient body weight
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).
|Contact: Ettore Biagi, MD||+39 039 233 ext email@example.com|
|U.O. Ematologia - Ospedali Riuniti di Bergamo||Recruiting|
|Bergamo, BG, Italy, 24128|
|Contact: Alessandro Rambaldi, MD firstname.lastname@example.org|
|Principal Investigator: Alessandro Rambaldi, MD|
|Clinica Pediatrica CTMO - Azienda Ospedaliera San Gerardo||Recruiting|
|Monza, MB, Italy, 20052|
|Contact: Ettore Biagi, MD email@example.com|
|Principal Investigator: Ettore Biagi, MD|
|U.O. Ematologia CTMO - Azienda Ospedaliera San Gerardo||Recruiting|
|Monza, MB, Italy, 20052|
|Contact: Matteo Parma, MD firstname.lastname@example.org|
|Principal Investigator: Matteo Parma, MD|
|Principal Investigator:||Ettore Biagi, MD||San Gerardo Hospital|