Horse ATG in Patients With AA or Low/Int-1 Risk MDS
The goal of this clinical research study is to learn if horse anti-thymocyte globulin (hATG), given in combination with methylprednisolone, cyclosporine, and G-CSF (filgrastim or pegfilgrastim), can help to control AA and/or low-int-1 risk MDS. The safety of this drug combination will also be studied.
hATG is made from horse blood and targets immune cells known as T-lymphocytes. Since T-lymphocytes are believed to be involved in causing low blood counts in AA and in some cases of MDS, killing these cells may help treat the disease.
Methylprednisolone and cyclosporine work to suppress immune cells called lymphocytes. This may help to improve low blood counts in AA and in some cases of MDS.
Filgrastim and pegfilgrastim are designed to cause white blood cells to grow. This may help to fight infections and help improve the white blood cell count.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Phase II Study of Horse Anti-Thymocyte Globulin (hATG), Cyclosporine, Methylprednisone, and GCSF (Filgrastim or Pegfilgrastim) in Patients With Aplastic Anemia (AA), or Low/Int-1 Risk Myelodysplastic Syndrome (MDS)|
- Achievement of Response [ Time Frame: 3 months ] [ Designated as safety issue: No ]Achievement of response defined: In aplastic anemia (AA) patients, response rate measured by complete response (CR) or partial response (PR); in myelodysplastic syndrome (MDS) patients, the response rate is measured by CR, PR, or hematologic improvement (HI). Criteria for HI per the Modified International Working Group Response Criteria in MDS. Patients eligible for trial and receive any dose of hATG and cyclosporine, included in estimating response rates and counted as treatment failures if response cannot be assessed for any reason.
- Time to Response [ Time Frame: 3 months ] [ Designated as safety issue: Yes ]Time to response, duration of CR, and overall survival estimated according to Kaplan-Meier. Time to response defined as interval between treatment start and date of response. Duration of CR defined as time interval between date of CR and date of first evidence of disease recurrence or death, within patients who achieved CR. Patients alive and recurrence-free at last follow-up censored at that time. Overall survival defined as time from treatment start until death or last follow-up time. Toxicity summarized according to standard CTC criteria.
|Study Start Date:||June 2012|
|Estimated Primary Completion Date:||June 2018 (Final data collection date for primary outcome measure)|
Experimental: hATG + Cyclosporine + Methylprednisone + GCSF
hATG (ATGAM) 40 mg/kg/d by vein over 8 hours daily on days 1 - 4. Methylprednisone 1 mg/kg/day by vein daily for 4 days, on days 1 - 4, to be given prior to the hATG infusion each day.
Cyclosporine 5 mg/kg by mouth daily given in 2 divided doses starting on day 1 and given for 6 months (180 days).
G-CSF starting on day 5, administered as:
Pegfilgrastim 6 mg subcutaneously (SQ) one time on day 5 and/or Filgrastim 300-480 mcg SQ starting on day 5 as needed to keep ANC >/= 1.5.
40 mg/kg by vein on Days 1 - 4.
35 mg/kg by vein on Days 1 - 4 (in patients with MDS age >/= 55).
Other Names:Drug: Cyclosporine
5 mg/kg by mouth daily given in 2 divided doses starting on Day 1 and given for 6 months (180 days).
Other Names:Drug: Methylprednisone
1 mg/kg by vein daily for 4 days on Days 1 - 4, to be given prior to the hATG infusion.
Other Names:Drug: Pegfilgrastim
6 mg subcutaneously one time on Day 5 as needed to keep ANC >/= 1.5.
Other Names:Drug: Filgrastim
300-480 mcg subcutaneously on Day 5 as needed to keep ANC >/= 1.5.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01624805
|Contact: Tapan Kadia, MD||713-563-3534|
|United States, Texas|
|University of Texas MD Anderson Cancer Center||Recruiting|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Tapan Kadia, MD||M.D. Anderson Cancer Center|