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Thymoglobulin and Cyclosporine in Patients With Aplastic Anemia or Myelodysplastic Syndrome
This study is currently recruiting participants.
Verified by M.D. Anderson Cancer Center, August 2009
First Received: December 9, 2008   Last Updated: August 19, 2009   History of Changes
Sponsor: M.D. Anderson Cancer Center
Collaborator: Genzyme
Information provided by: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT00806598
  Purpose

The goal of this clinical research study is to learn if combining the drugs thymoglobulin, methylprednisolone, cyclosporine, and G-CSF (NeupogenTM) can help to control severe AA or hypoplastic MDS. The safety of this combination therapy will also be studied.


Condition Intervention Phase
Myelodysplastic Syndrome
Aplastic Anemia
Drug: Thymoglobulin
Drug: Cyclosporine
Drug: Methylprednisolone
Drug: G-CSF
Phase II

Study Type: Interventional
Study Design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title: Phase II Study of Combination of Thymoglobulin, Cyclosporine, Methylprednisone, and GCSF in Patients With Newly Diagnosed Aplastic Anemia or With Hypoplastic or Low/Intermediate-1 Risk Myelodysplastic Syndrome

Resource links provided by NLM:


Further study details as provided by M.D. Anderson Cancer Center:

Primary Outcome Measures:
  • Overall Response [ Time Frame: 3 Month, 6 Month (start of treatment until disease progression) ] [ Designated as safety issue: No ]

Estimated Enrollment: 60
Study Start Date: May 2005
Estimated Study Completion Date: December 2010
Estimated Primary Completion Date: December 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Thymoglobulin + Cyclosporin: Experimental
Combination of Thymoglobulin + Methylprednisone + Cyclosporin + G-CSF
Drug: Thymoglobulin

3.5 or 2.5 mg/kg/day IV for 5 days

  • Aplastic anemia patients receive 3.5 mg/kg/day for 5 days
  • MDS patients <55 years receive 3.5 mg/kg/day for 5 days
  • MDS patients >55 years receive 2.5 mg/kg/day for 5 days
Drug: Cyclosporine
5 mg/kg orally for 6 months; start after completing thymoglobulin.
Drug: Methylprednisolone
1 mg/kg/day IV for 4 days, given before each dose of thymoglobulin.
Drug: G-CSF
G-CSF 5 microgram/kg subcutaneously daily up to 3 months, start after thymoglobulin.

  Show Detailed Description

  Eligibility

Ages Eligible for Study:   15 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of severe aplastic anemia (bone marrow cellularity < 30%, with two of three peripheral counts at the time of initial presentation or currently low with ANC < 500/mL, pre-transfusion PLT< 20,000/mL, or pre-transfusion hemoglobin < 8 g/dL and presence of no other underlying disorder.
  • Diagnosis of MDS (WHO) with bone marrow cellularity < 30%, with two of three peripheral counts at the time of initial presentation or currently low with ANC < 500/mL, pre-transfusion PLT < 20,000/mL, or pre-transfusion hemoglobin < 8 g/dL.
  • Patients with MDS who have received prior biological therapy (not chemotherapy) are eligible. Hypomethylating agents and histone deacetylase inhibitors are considered as biological therapy.
  • Age 15 or greater
  • Adequate renal function (creatinine less than or equal to 2.0 mg/dL) unless related to the disease
  • Adequate hepatic function (bilirubin less than or equal to 3.5 mg/dL) unless related to the disease
  • No other investigational therapy in the past 14 days
  • Able to sign consent form
  • Able to comply with the need for contraception (abstinence, condom, birth control pill, or other acceptable form of contraception) during the entire study period
  • Diagnosis of MDS (WHO) with bone marrow cellularity greater than 30%, with low or intermediate-1 risk by the IPSS score, and requiring treatment (i.e. transfusion-dependent)

Exclusion Criteria:

  • Active and uncontrolled infection
  • HIV positive test
  • Pregnant or breast feeding
  • Active and uncontrolled medical illness (pulmonary, cardiac, neurological, or other) that in the opinion of treating physician would likely interfere with study treatment
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00806598

Contacts
Contact: Tapan M Kadia, M.D. 713/563-3534 tkadia@mdanderson.org

Locations
United States, Texas
U.T.M.D. Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Tapan M. Kadia, M.D.     713-563-3534     tkadia@mdanderson.org    
Principal Investigator: Tapan M. Kadia, M.D.            
Sponsors and Collaborators
M.D. Anderson Cancer Center
Genzyme
Investigators
Principal Investigator: Tapan M. Kadia, M.D. U.T.M.D. Anderson Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: The University of Texas M.D. Anderson Cancer Center ( Tapan Mahendra Kadia, M.D./Assistant Professor )
Study ID Numbers: 2005-0115
Study First Received: December 9, 2008
Last Updated: August 19, 2009
ClinicalTrials.gov Identifier: NCT00806598     History of Changes
Health Authority: United States: Institutional Review Board

Keywords provided by M.D. Anderson Cancer Center:
Myelodysplastic Syndrome
Aplastic Anemia
Thymoglobulin
Cyclosporine
Methylprednisolone
G-CSF

Additional relevant MeSH terms:
Anti-Inflammatory Agents
Anti-Infective Agents
Cyclosporine
Molecular Mechanisms of Pharmacological Action
Precancerous Conditions
Immunologic Factors
Antineoplastic Agents
Methylprednisolone
Physiological Effects of Drugs
Hormones, Hormone Substitutes, and Hormone Antagonists
Antiemetics
Prednisolone acetate
Hormones
Neuroprotective Agents
Cyclosporins
Preleukemia
Pathologic Processes
Therapeutic Uses
Antifungal Agents
Syndrome
Anemia, Aplastic
Dermatologic Agents
Methylprednisolone Hemisuccinate
Disease
Antineoplastic Agents, Hormonal
Hematologic Diseases
Lenograstim
Myelodysplastic Syndromes
Adjuvants, Immunologic
Gastrointestinal Agents

ClinicalTrials.gov processed this record on February 08, 2010