Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome

June 18, 2026 updated by: M.D. Anderson Cancer Center

Phase II Study of Horse Anti-Thymocyte Globulin (hATG), Cyclosporine, Methylprednisolone, and GCSF (Filgrastim or Pegfilgrastim) in Patients With Aplastic Anemia (AA), or Low/Int-1 Risk Myelodysplastic Syndrome (MDS)

This phase II trial studies methylprednisolone, horse anti-thymocyte globulin, cyclosporine, filgrastim, and/or pegfilgrastim or pegfilgrastim biosimilar in treating patients with aplastic anemia or low or intermediate-risk myelodysplastic syndrome. Horse anti-thymocyte globulin 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 aplastic anemia and in some cases of myelodysplastic syndromes, 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 aplastic anemia and myelodysplastic syndromes. 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. Giving methylprednisolone and horse anti-thymocyte globulin together with cyclosporine, filgrastim, and/or pegfilgrastim may be an effective treatment for patients with aplastic anemia or myelodysplastic syndrome.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

PRIMARY OBJECTIVES:

I. To evaluate the efficacy of the combination of hATG (horse anti-thymocyte globulin), methylprednisolone, cyclosporine, and GCSF (filgrastim) in achieving response (complete response [CR], partial response [PR], or hematologic improvement [HI]) in patients with aplastic anemia, or myelodysplastic syndromes (MDS).

SECONDARY OBJECTIVES:

I. To assess the safety, tolerability, and toxicities of the combination of hATG, methylprednisolone, cyclosporine, and GCSF in patients with aplastic anemia, or MDS. II. To assess time to response, response duration, and overall survival of patients with aplastic anemia, or MDS being treated with the combination of hATG, methylprednisolone, cyclosporine, and GCSF.

OUTLINE:

Patients receive methylprednisolone intravenously (IV) over 10 minutes on days 1-4 and IV or orally (PO) with taper over days 5-30. Patients also receive horse anti-thymocyte globulin IV over 8 hours daily on days 1-4, cyclosporine PO twice daily (BID) on days 1-180, and pegfilgrastim or pegfilgrastim biosimilar subcutaneously (SC) on day 5 and/or filgrastim SC beginning on day 5 and continuing until absolute neutrophil count recovers. Treatment continues for up to 6 months in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 6-12 months.

Study Type

Interventional

Enrollment (Estimated)

140

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • M D Anderson Cancer Center
        • Contact:
        • Principal Investigator:
          • Tapan M. Kadia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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. Patients ages 18 years and older 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 below:

    • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)
Patients receive methylprednisolone IV over 10 minutes on days 1-4 and IV or PO with taper over days 5-30. Patients also receive horse anti-thymocyte globulin IV over 8 hours daily on days 1-4, cyclosporine PO BID on days 1-180, and pegfilgrastim or pegfilgrastim biosimilar SC on day 5 and/or filgrastim SC beginning on day 5 and continuing until absolute neutrophil count recovers. Treatment continues for up to 6 months in the absence of disease progression or unacceptable toxicity.
Biological: Pegfilgrastim
Given SC
Other Names:
  • Filgrastim SD-01
  • filgrastim-SD/01
  • Fulphila
  • HSP-130
  • Jinyouli
  • Neulasta
  • Neulastim
  • Pegfilgrastim Biosimilar HSP-130
  • SD-01
  • SD-01 sustained duration G-CSF
Biological: Filgrastim
Given SC
Other Names:
  • G-CSF
  • r-metHuG-CSF
  • Neupogen
  • Recombinant Methionyl Human Granulocyte Colony Stimulating Factor
  • rG-CSF
  • Tevagrastim
  • FILGRASTIM, LICENSE HOLDER UNSPECIFIED
Biological: Anti-Thymocyte Globulin
Given IV
Other Names:
  • ATGAM
  • ATG
  • Antithymocyte Globulin
  • Antithymocyte Serum
  • ATS
  • Thymoglobulin
Drug: Cyclosporine
Given PO
Other Names:
  • 27-400
  • Sandimmune
  • Ciclosporin
  • CsA
  • Neoral
  • Gengraf
  • Sandimmun
  • Cyclosporin
  • Cyclosporin A
  • OL 27-400
  • SangCya
Drug: Methylprednisolone
Given IV or PO
Other Names:
  • Medrol
  • Wyacort
  • Adlone
  • Caberdelta M
  • DepMedalone
  • Depo Moderin
  • Depo-Nisolone
  • Duralone
  • Emmetipi
  • Esametone
  • Firmacort
  • Medlone 21
  • Medrate
  • Medrol Veriderm
  • Medrone
  • Mega-Star
  • Meprolone
  • Methylprednisolonum
  • Metilbetasone Solubile
  • Metrocort
  • Metypresol
  • Metysolon
  • Predni-M-Tablinen
  • Prednilen
  • Radilem
  • Sieropresol
  • Solpredone
  • Summicort
  • Urbason
  • Veriderm Medrol

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Achievement of response
Time Frame: Up to 6 years
Measured by complete response (CR), partial response, or hematologic improvement.
Up to 6 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to response
Time Frame: The interval between treatment start and the date of response, assessed up to 6 years
Estimated according to the Kaplan-Meier method.
The interval between treatment start and the date of response, assessed up to 6 years
Duration of CR
Time Frame: Time interval between the date of CR to the date of first evidence of disease recurrence or death, assessed up to 6 years
Estimated according to the Kaplan-Meier method.
Time interval between the date of CR to the date of first evidence of disease recurrence or death, assessed up to 6 years
Overall survival
Time Frame: Time from treatment start until the death or last follow-up time, assessed up to 6 years
Estimated according to the Kaplan-Meier method.
Time from treatment start until the death or last follow-up time, assessed up to 6 years
Incidence of adverse events
Time Frame: Up to 6 years
Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0. Tabulated by grade and course of therapy. Numbers of required dose reductions will be included in the toxicity report.
Up to 6 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

M.D. Anderson Cancer Center

Collaborators

National Cancer Institute (NCI)

Investigators

  • Principal Investigator: Tapan M Kadia, M.D. Anderson Cancer Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 25, 2012

Primary Completion (Estimated)

June 30, 2029

Study Completion (Estimated)

June 30, 2029

Study Registration Dates

First Submitted

June 19, 2012

First Submitted That Met QC Criteria

June 20, 2012

First Posted (Estimated)

June 21, 2012

Study Record Updates

Last Update Posted (Actual)

June 22, 2026

Last Update Submitted That Met QC Criteria

June 18, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2012-0334 (Other Identifier: M D Anderson Cancer Center)
  • NCI-2012-01096 (Registry Identifier: CTRP (Clinical Trial Reporting Program))

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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