Horse ATG in Patients With Aplastic Anemia (AA) or Low/Int-1 Risk Myelodysplastic Syndrome (MDS)
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|ClinicalTrials.gov Identifier: NCT01624805|
Recruitment Status : Recruiting
First Posted : June 21, 2012
Last Update Posted : October 24, 2018
If you are reading and signing this form on behalf of a potential participant, please note: Any time the words "you," "your," "I," or "me" appear, it is meant to apply to the potential participant.
You are being asked to take part in this study because you have aplastic anemia (AA) or low/int-1 risk myelodysplastic syndrome (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-sndz 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-sndz 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.
This is an investigational study. hATG, cyclosporine, methylprednisolone, and filgrastim-sndz/pegfilgrastim are all FDA approved and commercially available for use in patients with AA and MDS. The use of filgrastim-sndz/pegfilgrastim with this drug combination is investigational.
Up to 100 patients will take part in this study. All will be enrolled at MD Anderson.
|Condition or disease||Intervention/treatment||Phase|
|Leukemia||Drug: hATG Drug: Cyclosporine Drug: Methylprednisone Drug: Pegfilgrastim Drug: Filgrastim||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||100 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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)|
|Study Start Date :||June 2012|
|Estimated Primary Completion Date :||June 2019|
|Estimated Study Completion Date :||June 2019|
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).
5 mg/kg by mouth daily given in 2 divided doses starting on Day 1 and given for 6 months (180 days).
1 mg/kg by vein daily for 4 days on Days 1 - 4, to be given prior to the hATG infusion.
6 mg subcutaneously one time on Day 5 as needed to keep ANC >/= 1.5.
300-480 mcg subcutaneously on Day 5 as needed to keep ANC >/= 1.5.
- Achievement of Response [ Time Frame: 3 months ]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 ]Time to response defined as interval between treatment start and date of response. Time to response estimated according to Kaplan-Meier.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|