Actively Recruiting

Phase 2
Age: 18Years +
All Genders
NCT01624805

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

Led by M.D. Anderson Cancer Center · Updated on 2026-04-15

140

Participants Needed

1

Research Sites

731 weeks

Total Duration

On this page

Sponsors

M

M.D. Anderson Cancer Center

Lead Sponsor

N

National Cancer Institute (NCI)

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients diagnosed with myelodysplastic syndrome (low, intermediate-1 by IPSS, or hypocellular), previously treated or untreated
  • Patients diagnosed with aplastic anemia, previously treated or untreated, who are not candidates for allogeneic stem cell transplant
  • Age 18 years or older
  • Off cytotoxic, immunosuppressive (except steroids), or targeted therapy for at least 2 weeks and recovered to grade 1 or less from toxic effects
  • Adequate liver function (bilirubin less than 2 mg/dL, AST less than 3 times upper limit of normal)
  • Adequate kidney function (creatinine less than 2.5 times upper limit of normal)
  • ECOG performance status of 2 or less
  • Women of child-bearing potential and men must agree to use adequate contraception during the study
  • Negative urine pregnancy test within 1 week prior to enrollment for women of childbearing potential
  • Ability to understand study requirements and provide signed informed consent
  • Indication for therapy such as transfusion dependence or morbidity related to cytopenias like bleeding, severe fatigue, or frequent infections
Not Eligible

You will not qualify if you...

  • Pregnant women
  • Nursing mothers (breastfeeding should be discontinued if treated on study)
  • Known HIV infection
  • Uncontrolled illness including active infection, symptomatic heart failure, unstable angina, cardiac arrhythmia, or psychiatric/social conditions limiting study compliance
  • Documented hypersensitivity to any study medication components

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

M D Anderson Cancer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

T

Tapan Kadia, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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