Working... Menu

Horse ATG in Patients With Aplastic Anemia (AA) or Low/Int-1 Risk Myelodysplastic Syndrome (MDS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01624805
Recruitment Status : Recruiting
First Posted : June 21, 2012
Last Update Posted : October 24, 2018
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

Brief Summary:

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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Intervention Model: Single Group Assignment
Masking: None (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)
Study Start Date : June 2012
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : June 2019

Arm Intervention/treatment
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.

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

Drug: Filgrastim
300-480 mcg subcutaneously on Day 5 as needed to keep ANC >/= 1.5.
Other Names:
  • G-CSF
  • Neupogen

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 <3 x ULN), kidney function (creatinine < 2.5 x ULN ).
  6. ECOG performance status of </= 2.
  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.

Information from the National Library of Medicine

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 identifier (NCT number): NCT01624805

Layout table for location contacts
Contact: Tapan Kadia, MD 713-563-3534

Layout table for location information
United States, Texas
University of Texas MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Layout table for investigator information
Principal Investigator: Tapan Kadia, MD M.D. Anderson Cancer Center

Additional Information:
Layout table for additonal information
Responsible Party: M.D. Anderson Cancer Center Identifier: NCT01624805     History of Changes
Other Study ID Numbers: 2012-0334
NCI-2012-01096 ( Registry Identifier: NCI CTRP )
First Posted: June 21, 2012    Key Record Dates
Last Update Posted: October 24, 2018
Last Verified: October 2018

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by M.D. Anderson Cancer Center:
Horse Antithymocyte Globulin
Antithymocyte Globulin
Aplastic Anemia
Low/Int-1 Risk Myelodysplastic Syndrome
Cyclosporin A

Additional relevant MeSH terms:
Layout table for MeSH terms
Myelodysplastic Syndromes
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Antilymphocyte Serum
Methylprednisolone Acetate
Methylprednisolone Hemisuccinate
Prednisolone acetate
Prednisolone hemisuccinate
Prednisolone phosphate
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents
Antirheumatic Agents
Calcineurin Inhibitors