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Trial record 6 of 22 for:    Aplastic Anemia | Recruiting, Not yet recruiting, Available Studies | NIH, U.S. Fed

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 : June 4, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
M.D. Anderson Cancer Center

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.

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

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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, 2019
Estimated Study Completion Date : June 30, 2019


Arm Intervention/treatment
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
Other Names:
  • Antithymocyte Globulin
  • Antithymocyte Serum
  • ATG
  • ATGAM
  • ATS
  • Thymoglobulin

Drug: Cyclosporine
Given PO
Other Names:
  • 27-400
  • Ciclosporin
  • CsA
  • Cyclosporin
  • Cyclosporin A
  • Gengraf
  • Neoral
  • OL 27-400
  • Sandimmun
  • Sandimmune
  • SangCya

Biological: Filgrastim
Given SC
Other Names:
  • FILGRASTIM, LICENSE HOLDER UNSPECIFIED
  • G-CSF
  • Neupogen
  • r-metHuG-CSF
  • Recombinant Methionyl Human Granulocyte Colony Stimulating Factor
  • rG-CSF
  • Tevagrastim

Drug: Methylprednisolone
Given IV or PO
Other Names:
  • Adlone
  • Caberdelta M
  • DepMedalone
  • Depo Moderin
  • Depo-Nisolone
  • Duralone
  • Emmetipi
  • Esametone
  • Firmacort
  • Medlone 21
  • Medrate
  • Medrol
  • Medrol Veriderm
  • Medrone
  • Mega-Star
  • Meprolone
  • Methylprednisolonum
  • Metilbetasone Solubile
  • Metrocort
  • Metypresol
  • Metysolon
  • Predni-M-Tablinen
  • Prednilen
  • Radilem
  • Sieropresol
  • Solpredone
  • Summicort
  • Urbason
  • Veriderm Medrol
  • Wyacort

Biological: Pegfilgrastim
Given SC
Other Names:
  • Filgrastim SD-01
  • filgrastim-SD/01
  • Fulphila
  • HSP-130
  • Jinyouli
  • Neulasta
  • Neulastim
  • Pegfilgrastim Biosimilar HSP-130
  • SD-01
  • SD-01 sustained duration G-CSF




Primary Outcome Measures :
  1. Achievement of response [ Time Frame: Up to 6 years ]
    Measured by complete response (CR), partial response, or hematologic improvement.


Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. 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.

  4. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

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


Contacts
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Contact: Tapan Kadia, MD 713-563-3534 tkadia@mdanderson.org

Locations
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United States, Texas
M D Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Tapan M. Kadia    713-563-3534    tkadia@mdanderson.org   
Principal Investigator: Tapan M. Kadia         
Sponsors and Collaborators
M.D. Anderson Cancer Center
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Tapan M Kadia M.D. Anderson Cancer Center

Additional Information:
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Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT01624805     History of Changes
Other Study ID Numbers: 2012-0334
NCI-2012-01096 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
2012-0334 ( Other Identifier: M D Anderson Cancer Center )
P30CA016672 ( U.S. NIH Grant/Contract )
First Posted: June 21, 2012    Key Record Dates
Last Update Posted: June 4, 2019
Last Verified: May 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Anemia
Anemia, Aplastic
Preleukemia
Myelodysplastic Syndromes
Syndrome
Disease
Pathologic Processes
Hematologic Diseases
Bone Marrow Diseases
Precancerous Conditions
Neoplasms
Cyclosporine
Methylprednisolone
Methylprednisolone Acetate
Methylprednisolone Hemisuccinate
Prednisolone
Prednisolone acetate
Cyclosporins
Antilymphocyte Serum
Lenograstim
Prednisolone hemisuccinate
Prednisolone phosphate
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents