Horse ATG in Patients With AA or Low/Int-1 Risk MDS | Aplastic Anemia and MDS International Foundation

Clinical Trial: NCT01624805

Horse ATG in Patients With AA or Low/Int-1 Risk MDS
For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov:
Purpose: 

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.

Study Visits:

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.

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.

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.

Status: 
Recruiting
Study Date: 
Tue, 06/19/2012 to Fri, 06/01/2018
Bone Marrow Disease(s): 
aplastic anemia
myelodysplastic syndromes (MDS)
Associated Drug(s): 
Intervention: 
Drug: hATG 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: Horse Anti-Thymocyte Globulin ATG Antithymocyte Globulin Thymoglobulin 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: Sandimmune CYA Cyclosporin A 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: Methylprednisolone Depo-Medrol Medrol Solu-Medrol Drug: Pegfilgrastim 6 mg subcutaneously one time on Day 5 as needed to keep ANC >/= 1.5. Other Names: Neulasta PEG=G-CSF Drug: Filgrastim 300-480 mcg subcutaneously on Day 5 as needed to keep ANC >/= 1.5. Other Names: G-CSF Neupogen