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Immunosuppressant

Combination Drug Therapy for Aplastic Anemia and Myelodysplastic Syndrome

Phase 2

Recruiting

Led By Tapan M Kadia

Research Sponsored by M.D. Anderson Cancer Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Eastern Cooperative Oncology Group (ECOG) performance status of =< 2

Must not have

Known human immunodeficiency virus (HIV) infection

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 6 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is studying a combination of drugs to treat patients with aplastic anemia or myelodysplastic syndrome.

Who is the study for?

This trial is for patients with aplastic anemia or low/intermediate-risk myelodysplastic syndrome. Eligible participants include those not currently fit for a stem cell transplant, off recent cytotoxic therapy, and with specific levels of bilirubin, AST, creatinine within set limits. They must have an ECOG status of ≤2, agree to contraception if applicable, and show a need for treatment due to symptoms like fatigue or infections.

What is being tested?

The study tests the combination of methylprednisolone (a steroid), horse anti-thymocyte globulin (an immune system suppressor), cyclosporine (another immunosuppressant), filgrastim and pegfilgrastim (both promote white blood cell growth) in treating aplastic anemia or myelodysplastic syndrome. The goal is to see if this regimen can improve blood counts and treat the disease effectively.

What are the potential side effects?

Potential side effects may include immune system suppression leading to increased infection risk; allergic reactions especially from horse-derived ATG; liver enzyme elevations; kidney function changes; bone marrow suppression causing low blood counts; nausea; headaches; high blood pressure from steroids.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I stopped my cancer treatment 2 weeks ago and have recovered from major side effects.

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I can take care of myself but might not be able to do heavy physical work.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am HIV positive.

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I do not have any serious illnesses or social situations that would stop me from following the study's requirements.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 6 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 6 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 6 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Achievement of response

Secondary study objectives

Duration of CR

Incidence of adverse events

Overall survival

+1 more

Side effects data

From undefined Phase 3 trial • 659 Patients • NCT00006011

73%

Leukopenia

71%

Alopecia

61%

Neutropenia

52%

Anemia

40%

Constitutional

34%

Nausea

29%

Vomiting

28%

Other Gastrointestinal

24%

Other Pain

21%

Thrombocytopenia

18%

Metabolic

17%

Other Hematologic

12%

Other Neurologic

12%

Pulmonary

11%

Cardiac left ventricular function

11%

Other Cardiovascular

10%

Infection Without Neutropenia

Genitourinary/Renal

Endocrine

Other Dermatologic

Auditory

Peripheral Sensory Neurologic

2nd Primary

Stomatitis

Other Infection/Fever

Musculoskeletal

Ocular/Visual

Myalgia

Lymphatics

Hepatic

Allergy

Hemorrhage

Sexual

100%

80%

60%

40%

20%

Study treatment Arm

Arm 1

Arm 2

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)Experimental Treatment5 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pegfilgrastim

2013

Completed Phase 3

~4440

Filgrastim

2000

Completed Phase 3

~3690