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

Related Clinical Trial
Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia Long Term Follow-up Observational Study After Clinical Trials of AMG531 (Romiplostim) in Patients With Untreated Aplastic Anemia Efficacy and Safety of Lower-dose Decitabine in Refractory Aplastic Anemia Quantitative MRI of Bone Marrow Fat Fraction in Patients With Trepanobiopsy Avatrombopag Usage in NSAA Ibrutinib for the Treatment of COVID-19 in Patients Requiring Hospitalization Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7Abnormalities After Immunosuppressive Therapy REGN7257 in Adult Patients With Severe Aplastic Anemia That Is Refractory to or Relapsed on Immunosuppressive Therapy Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing MUD SCT A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) Investigation of the Cylex® ImmuKnow® Assay Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant Umbilical Cord Blood Transplantation From Unrelated Donors Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study Unrelated Umbilical Cord Blood (UBC)Transplantation New York Blood Center National Cord Blood Program Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-Match Bone Marrow Transplantation of Patients in Remission Using Partially Matched Relative Donor Nonmyeloablative Allo SCT for the Treatment of Hematologic Disorders Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis Effects of Aerobic Training and Inspiratory Muscle Training in Patients During Hematopoietic Stem Cell Transplantation A Phase II Study of Umbilical Cord Blood Transplantation Transplants With Unlicensed Preserved Cord Blood Safety Study of Cord Blood Units for Stem Cell Transplants Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation Screening Gene Mutations in Myeloid Cancers by Next Generation Sequencing to Improve Treatment Results Safety Study of CD3/CD19 Depleted Haploidentical Stem Cells Trial of Two Central Venous Catheter (CVC) Flushing Schemes in Pediatric Hematology and Oncology Patients Blood Transplantation for Patients With Hematologic Malignancies or Bone Marrow Failure States Unrelated Cord Blood Transplant Plus a Haplo-Identical (Half-Matched), T-Cell Depleted Stem Transplant From a Related Donor for Subjects With High Risk Malignancies Extended Platelet Parameters as a Means to Differentiate Immune Thrombocytopenia From Hypo-proliferative Thrombocytopenias. Protection Against Benzene Toxicity Treatment of Menorrhagia in Women With Thrombocytopenia Using Platelets or Platelets and Hormones Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA Etiology of Blood Dyscrasias: Analysis of the International Agranulocytosis and Aplastic Anemia Study Data Multi-Center Trial of Anti-Thymocyte Globulin in Treatment of Aplastic Anemia and Other Hematologic Disorders King’s Invasive Aspergillosis Study II Identification of Mechanism in the Erythroid Response in Patients With Myelodysplasia Undergoing Chelation Therapy Aplastic Anemia Epidemiology: Incidence and Case-control Drug Etiology of Aplastic Anemia and Related Dyscrasias hATG+CsA vs hATG+CsA+Eltrombopag for SAA Non-Myeloablative Allogeneic Stem Cell Transplantation With Matched Unrelated Donors for Treatment of Hematologic Malignancies, Renal Cell Carcinoma, and Aplastic Anemia Conditioning Regimens for Patients With Severe Aplastic Anemia Transplanted With Marrow From an Unrelated Donor Study of Allogeneic Bone Marrow and T-Cell Depleted, CD34+ Peripheral Blood Stem Cell Transplantation in Patients With Aplastic Anemia Posaconazole Prophylaxis During ATG Treatment for hMDS/AA Patients Efficacy and Safety of Thrombopoietin In Patients With Severe and Very Severe Aplastic Anemia Study of MRI Monitoring in Patients With Aplastic Anemia and Low or Int-1 Risk of MDS Complicated With Iron Overload A Pilot Study of the Thrombopoietin-Receptor Agonist Eltrombopag in Refractory Aplastic Anemia Patients Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia Flu+CPM+rATG Conditioning Regimes for Unrelated Bone Marrow Transplantation (UBMT)(or Mobilized Peripheral Blood)in Severe Aplastic Anemia (SAA) Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia Eltrombopag in Combination With Rabbit Anti-thymocyte Globulin/Cyclosporine A in Naive Aplastic Anemia (AA) Subjects Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia 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 Efficacy and Safety of Eltrombopag + CSA in Patients With Moderate Aplastic Anemia (EMAA) Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia Multi Center Case Control Study on Multiple Risk Factors of Aplastic Anemia NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia Horse ATG/CsA in Aplastic Anemia Patients Unresponsive to or With a Suboptimal Response to Rabbit ATG/CsA Treatment Safety and Efficacy Study of Ex Vivo Immunotherapy for Treatment of Aplastic Anemia Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA) Haploidentical Transplantation in Severe Aplastic Anemia A Description of Bacteria in the Mouths of Patients With Severe Aplastic Anemia Efficacy and Safety of Eltrombopag + Tacrolimus in Chinese Refractory or Relapsed Aplastic Anemia Patients A Study to Evaluate the Safety and Efficacy of Hetrombopag Olamine in Severe Aplastic Anemia (SAA) Patient Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Cyclophosphamide and Anti-thymocyte Globulin Followed By Methotrexate and Cyclosporine in Preventing Chronic Graft-Versus-Host Disease in Patients With Severe Aplastic Anemia Undergoing Donor Bone Marrow Transplant Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin Regimens in Severe Aplastic Anemia Patients A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia Cyclophosphamide, Antithymocyte Globulin, and Total-Body Irradiation in Treating Patients With Severe Aplastic Anemia Undergoing Umbilical Cord Blood Transplant Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia Stem Cell Factor Medication for Aplastic Anemia Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome Extended Dosing With Eltrombopag for Severe Aplastic Anemia Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF) Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Aplastic Anemia Stem Cell Mobilization Potential in Patients With Aplastic Anemia in Remission A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia Collection of Blood and Bone Marrow From Patients With Aplastic Anemia for Analysis of Adhesion Molecules, Chemokines and Their Receptors Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia Alefacept in Patients With Relapsed/Refractory Aplastic Anemia Oral Manifestations of Aplastic Anemia The Efficacy of Immunosuppressive Therapy Combined With Cord Blood Transfusion in Treatment of Severe Aplastic Anemia Clinical Study of Non Severe Aplastic Anemia Treated With Cyclosporine, Androgen and Levamisole Mesenchymal Stem Cells Transplantation to Patients With Relapsed/Refractory Aplastic Anemia. Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic Anemia Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA) ATG Combined With Cyclophosphamide And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia A Novel TBI Free Conditioning Protocol for Haploidentical Transplant in Acquired Aplastic Anemia: Combination Therapy of Severe Aplastic Anemia Comparing Therapies for the Treatment of Severe Aplastic Anemia Mesenchymal Stem Cells Co-transplantation in Alternative Donor Transplantation of Severe Aplastic Anemia. Study of AMG531(Romiplostim) in Patients With Aplastic Anemia Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia Improving Immunosuppressive Treatment for Patients With Severe Aplastic Anemia Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia A Single-Arm Phase 2 Study With Optimized Standard Protocol for Severe Aplastic Anemia Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia A Study to Assess Efficacy and Safety of PF-06462700 in Japanese Participants With Aplastic Anemia Alemtuzumab and Rituximab in Aplastic Anemia Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia Comparison of Two Different Doses of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA) Safety and Efficacy of Patient’s Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia Ambispective Observational Study to Evaluate the Incidence and Management of Aplastic Anemia in Spain Moderate-dose Cyclophosphamide for Childhood Acquired Aplastic Anemia

Brief 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

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)

Brief Summary

      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.
    

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 Phase

Phase 2

Study Type

Interventional


Primary Outcome

Achievement of response

Secondary Outcome

 Time to response

Condition

Aplastic Anemia

Intervention

Anti-Thymocyte Globulin

Study Arms / Comparison Groups

 Treatment (methylprednisolone, hATG, cyclosporine, G-CSF)
Description:  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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

100

Start Date

June 25, 2012

Completion Date

June 30, 2022

Primary Completion Date

June 30, 2021

Eligibility Criteria

        Inclusion Criteria:

          -  Patients with the diagnosis of MDS (low, int-1 by International Prognostic Scoring
             System [IPSS], or hypocellular) who are either previously treated or untreated are
             eligible for this trial

          -  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

          -  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

          -  Bilirubin < 2 mg/dL

          -  Aspartate aminotransferase (AST) < 3 x upper limit of normal (ULN)

          -  Creatinine < 2.5 x ULN

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

          -  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

          -  A negative urine pregnancy test is required within 1 week for all women of
             childbearing potential prior to enrolling on this trial

          -  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

          -  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 (e.g. neutropenia)

        Exclusion Criteria:

          -  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

          -  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

          -  Patient with documented hypersensitivity to any of the component medications
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Tapan M Kadia, 713-563-3534, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01624805

Organization ID

2012-0334

Secondary IDs

NCI-2012-01096

Responsible Party

Sponsor

Study Sponsor

M.D. Anderson Cancer Center

Collaborators

 National Cancer Institute (NCI)

Study Sponsor

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


Verification Date

December 2020