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, 2017
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 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.
This is an investigational study. hATG, cyclosporine, methylprednisolone, and filgrastim/pegfilgrastim are all FDA approved and commercially available for use in patients with AA and MDS. The use of filgrastim/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 Drug Administration:
If you are found to be eligible to take part in this study, you will be admitted to the hospital. On the first day, before starting the hATG, a small amount of diluted hATG will be injected under your skin to make sure that you are not allergic to it. If you have a reaction, more testing may be done. If the reaction is severe, you will be taken off study.
On Days 1-4 you will receive methylprednisolone by vein over about 10 minutes. You will receive hATG by vein over 8 hours.
If you are able to tolerate ATG without any significant side effects, you should be out of the hospital in about 4-5 days.
On Days 1-180 you will take cyclosporine by mouth 2 times a day at about the same time every day.
On Day 5 (or starting on Day 5) you will receive filgrastim or pegfilgrastim by an injection under your skin. Your doctor will decide which drug you will receive. If you receive pegfilgrastim, you will receive it one time on Day 5. If you receive filgrastim, you will receive it starting on Day 5. You will continue to receive it until your blood counts recover.
You will be given standard drugs to help decrease the risk of side effects. You may be given a drug called eltrombopag to help increase your platelet counts if you have low platelets or complications related to low platelets. You may ask the study staff for information about how the drugs are given and their risks.
One time weekly for the first 4-6 weeks and then 1 time a month during Months 2-6
- Your complete medical history will be recorded.
- You will have a physical exam, including measurement of your height, weight, and vital signs (blood pressure, heart rate, breathing rate, and temperature).
- Blood (about 2 tablespoons) will be drawn for routine tests.
At the end of Month 3 and then when the doctor thinks it is needed, you will have a bone marrow aspirate/biopsy to check the status of the disease.
Length of Treatment:
You may continue taking the study drug for up to 6 months. You will no longer be able to take the study drug if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your participation on the study will be over once you have completed the follow-up visits.
When you are off treatment, every 6-12 months you will be called by a member of the study staff. You will be asked about any side effects you may be having. The phone calls will take about 5-10 minutes.
|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|
U.S. FDA Resources
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.
- 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.
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|