- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01624805
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
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)
Study Overview
Status
Conditions
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 Type
Enrollment (Estimated)
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Tapan Kadia, MD
- Phone Number: 713-563-3534
- Email: tkadia@mdanderson.org
Study Locations
-
-
Texas
-
Houston, Texas, United States, 77030
- Recruiting
- M D Anderson Cancer Center
-
Contact:
- Tapan M. Kadia
- Phone Number: 713-563-3534
- Email: tkadia@mdanderson.org
-
Principal Investigator:
- Tapan M. Kadia
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / 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.
|
Given SC
Other Names:
Given SC
Other Names:
Given IV
Other Names:
Given PO
Other Names:
Given IV or PO
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Achievement of response
Time Frame: Up to 6 years
|
Measured by complete response (CR), partial response, or hematologic improvement.
|
Up to 6 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
The interval between treatment start and the date of response, assessed up to 6 years
|
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.
|
Time interval between the date of CR to the date of first evidence of disease recurrence or death, assessed up to 6 years
|
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.
|
Time from treatment start until the death or last follow-up time, assessed up to 6 years
|
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.
|
Up to 6 years
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Tapan M Kadia, M.D. Anderson Cancer Center
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Neoplasms
- Disease
- Bone Marrow Diseases
- Hematologic Diseases
- Precancerous Conditions
- Bone Marrow Failure Disorders
- Syndrome
- Myelodysplastic Syndromes
- Preleukemia
- Anemia
- Anemia, Aplastic
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Anti-Infective Agents
- Autonomic Agents
- Peripheral Nervous System Agents
- Enzyme Inhibitors
- Anti-Inflammatory Agents
- Antirheumatic Agents
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Antiemetics
- Gastrointestinal Agents
- Glucocorticoids
- Hormones
- Hormones, Hormone Substitutes, and Hormone Antagonists
- Antineoplastic Agents, Hormonal
- Neuroprotective Agents
- Protective Agents
- Dermatologic Agents
- Adjuvants, Immunologic
- Antifungal Agents
- Calcineurin Inhibitors
- Prednisolone
- Methylprednisolone Acetate
- Methylprednisolone
- Methylprednisolone Hemisuccinate
- Prednisolone acetate
- Prednisolone hemisuccinate
- Prednisolone phosphate
- Lenograstim
- Thymoglobulin
- Antilymphocyte Serum
- Cyclosporine
- Cyclosporins
Other Study ID Numbers
- 2012-0334 (Other Identifier: M D Anderson Cancer Center)
- NCI-2012-01096 (Registry Identifier: CTRP (Clinical Trial Reporting Program))
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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