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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 6 years
Awards & highlights

Study 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.Check my eligibility
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.See study design
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.

Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Achievement of response
Secondary outcome measures
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
7%
Genitourinary/Renal
7%
Endocrine
6%
Other Dermatologic
6%
Auditory
5%
Peripheral Sensory Neurologic
5%
2nd Primary
4%
Stomatitis
4%
Other Infection/Fever
4%
Musculoskeletal
4%
Ocular/Visual
3%
Myalgia
2%
Lymphatics
2%
Hepatic
2%
Allergy
1%
Hemorrhage
1%
Sexual
100%
80%
60%
40%
20%
0%
Study treatment Arm
Arm 1
Arm 2

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
~4410
Filgrastim
2000
Completed Phase 3
~3670
Anti-Thymocyte Globulin
2009
Completed Phase 4
~980
Cyclosporine
1997
Completed Phase 3
~1830
Methylprednisolone
2015
Completed Phase 4
~2280

Find a Location

Who is running the clinical trial?

M.D. Anderson Cancer CenterLead Sponsor
2,973 Previous Clinical Trials
1,789,206 Total Patients Enrolled
83 Trials studying Myelodysplastic Syndromes (MDS)
5,910 Patients Enrolled for Myelodysplastic Syndromes (MDS)
National Cancer Institute (NCI)NIH
13,665 Previous Clinical Trials
40,925,714 Total Patients Enrolled
115 Trials studying Myelodysplastic Syndromes (MDS)
30,353 Patients Enrolled for Myelodysplastic Syndromes (MDS)
Tapan M KadiaPrincipal InvestigatorM.D. Anderson Cancer Center
13 Previous Clinical Trials
1,073 Total Patients Enrolled
1 Trials studying Myelodysplastic Syndromes (MDS)
458 Patients Enrolled for Myelodysplastic Syndromes (MDS)

Media Library

Anti-Thymocyte Globulin (Immunosuppressant) Clinical Trial Eligibility Overview. Trial Name: NCT01624805 — Phase 2
Myelodysplastic Syndromes (MDS) Research Study Groups: Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)
Myelodysplastic Syndromes (MDS) Clinical Trial 2023: Anti-Thymocyte Globulin Highlights & Side Effects. Trial Name: NCT01624805 — Phase 2
Anti-Thymocyte Globulin (Immunosuppressant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01624805 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
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
2012. "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". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01624805.
All > Aplastic Anemia > Restasis
~13 spots leftby Jun 2025
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