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)
N/A
N/A
Both
Leukemia
Trial Information
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 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.
Inclusion Criteria:
1. Patients with the diagnosis of MDS (Low, Int-1 by IPSS, or hypocellular) who are
either previously treated or untreated are eligible for this trial.
2. Patients with the diagnosis of aplastic anemia who are either previously treated or
untreated are eligible if they are not currently candidates for an allogeneic stem
cell transplant.
3. All ages are eligible.
4. Patients must have been off of cytotoxic, immunosuppressive (except steroids), or
targeted therapy for at least 2 weeks prior to entering this study, and have
recovered from the toxic effects of that therapy to grade 1 or less.
5. Adequate organ function as defined as: liver function (bilirubin < 2mg/dL, AST and/or
ALT <3 x ULN), kidney function (creatinine < 2.5 x ULN ).
6. ECOG performance status of
7. Women of child-bearing potential and men must agree to use adequate contraception
(hormonal or barrier method of birth control; abstinence) prior to study entry and
for the duration of study participation. Should a woman become pregnant or suspect
she is pregnant while participating in this study, she should inform her treating
physician immediately.
8. A negative urine pregnancy test is required within 1 week for all women of
childbearing potential prior to enrolling on this trial.
9. Patient must have the ability to understand the requirements of the study and signed
informed consent. A signed informed consent by the patient or his legally authorized
representative is required prior to their enrollment on the protocol.
10. Patients should have an indication for therapy for their disease such as transfusion
dependence or morbidity associated with their cytopenia(s) such as bleeding, severe
fatigue, or frequent/multiple infections (eg. neutropenia).
Exclusion Criteria:
1. Pregnant women are excluded from this study. Because there is an unknown but
potential risk for adverse events in nursing infants secondary to treatment of the
mother with the study agents, breastfeeding should be discontinued if the mother is
treated on this study.
2. Known HIV infection
3. Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
arrhythmia, or psychiatric illness/social situations that would limit compliance with
study requirements.
4. Patient with documented hypersensitivity to any of the component medications.
Type of Study:
Interventional
Study Design:
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
Achievement of Response
Outcome Description:
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.
Outcome Time Frame:
3 months
Safety Issue:
No
Principal Investigator
Tapan Kadia, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
UT MD Anderson Cancer Center
Authority:
United States: Institutional Review Board
Study ID:
2012-0334
NCT ID:
NCT01624805
Start Date:
June 2012
Completion Date:
Related Keywords:
- Leukemia
- Leukemia
- Aplastic Anemia
- AA
- Low/Int-1 Risk Myelodysplastic Syndrome
- MDS
- hATG
- Horse Antithymocyte Globulin
- ATG
- Antithymocyte Globulin
- Thymoglobulin
- Cyclosporine
- Sandimmune
- CYA
- Cyclosporin A
- Methylprednisone
- Methylprednisolone
- Depo-Medrol
- Medrol
- Solu-Medrol
- pegfilgrastim
- Neulasta
- PEG-G-CSF
- Filgrastim
- C-CSF
- Neupogen
- Anemia, Aplastic
- Leukemia
- Myelodysplastic Syndromes
- Preleukemia
Name | Location |
|---|---|
| UT MD Anderson Cancer Center | Houston, Texas 77030 |
