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
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|ClinicalTrials.gov Identifier: NCT01624805|
Recruitment Status : Recruiting
First Posted : June 21, 2012
Last Update Posted : December 4, 2020
|Condition or disease||Intervention/treatment||Phase|
|Aplastic Anemia de Novo Myelodysplastic Syndrome Myelodysplastic Syndrome Previously Treated Myelodysplastic Syndrome||Biological: Anti-Thymocyte Globulin Drug: Cyclosporine Biological: Filgrastim Drug: Methylprednisolone Biological: Pegfilgrastim||Phase 2|
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).
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.
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 (Clinical Trial)|
|Estimated Enrollment :||100 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||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)|
|Actual Study Start Date :||June 25, 2012|
|Estimated Primary Completion Date :||June 30, 2021|
|Estimated Study Completion Date :||June 30, 2022|
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: Anti-Thymocyte Globulin
Given IV or PO
- Achievement of response [ Time Frame: Up to 6 years ]Measured by complete response (CR), partial response, or hematologic improvement.
- 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.
- 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.
- 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.
- 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.
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 ClinicalTrials.gov identifier (NCT number): NCT01624805
|Contact: Tapan Kadia, MDfirstname.lastname@example.org|
|United States, Texas|
|M D Anderson Cancer Center||Recruiting|
|Houston, Texas, United States, 77030|
|Contact: Tapan M. Kadia 713-563-3534 email@example.com|
|Principal Investigator: Tapan M. Kadia|
|Principal Investigator:||Tapan M Kadia||M.D. Anderson Cancer Center|