Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier:
NCT00520468
First received: August 23, 2007
Last updated: August 1, 2012
Last verified: August 2012
  Purpose

Objectives:

Primary: To evaluate the response rate of total cytokine-immunotherapy for low-risk myelodysplastic syndromes (MDS).

Secondary: To evaluate response duration, survival and side effects of the treatment.


Condition Intervention Phase
Myelodysplastic Syndrome
Drug: Erythropoietin
Drug: Cyclosporin A
Drug: G-CSF
Drug: Prednisone
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Treatment of Myelodysplastic Syndrome (MDS) With Cytokine-Immunotherapy for Low-Risk MDS

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Number of Participants With Response [ Time Frame: Response evaluation within first 3 months from start of therapy, then every 3 to 6 months ] [ Designated as safety issue: No ]
    Periodic bone marrow samples (every 3-6 months) to check cells related to disease before/during/after study. Response classifications categorized by the International Working Group Response Criteria for Myelodysplastic Syndrome (MDS) as: Complete Remission, Partial Response, Hematologic Improvement or No Response.


Enrollment: 15
Study Start Date: June 2004
Study Completion Date: June 2009
Primary Completion Date: June 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cytokine-Immunotherapy
Erythropoietin 40,000 units subcutaneously (SQ) weekly; G-CSF 300 mcg SQ twice a week; Prednisone 60 mg/Day for 7 days, taper over 1 month; Cyclosporin A 300 mg orally daily
Drug: Erythropoietin
40,000 units injected under the skin (SQ) weekly
Other Names:
  • Darbepoetin alfa
  • Aranesp
  • Erythropoiesis stimulating protein
Drug: Cyclosporin A
300 mg (tablets) by mouth daily for 6 months
Other Names:
  • CYA
  • Cyclosporine
  • Sandimmune
Drug: G-CSF
300 mcg injected under the skin (SQ) two times per week
Other Names:
  • Filgrastim
  • Neupogen
Drug: Prednisone
60 mg per day for 7 days, taper over 1 month

Detailed Description:

MDS is a disease that produces low blood counts and may cause anemia, infections, and/or bleeding. Erythropoietin and Granulocyte colony-stimulating factor (G-CSF or GCSF) are drugs that stimulate the production of red cells and white cells. Prednisone and cyclosporin are drugs that work against MDS by affecting your immune system.

Before you can start treatment on this study, you will have what are called "screening tests". These tests will help the doctor decide if you are eligible to take part in the study. You will have a complete medical history and physical exam. Routine blood tests (between 4-6 tablespoons) will be performed. You will have a bone marrow aspiration. To collect a bone marrow aspirate, an area of the hip or chest bone is numbed with anesthetic and a small amount of bone marrow is withdrawn through a large needle. Women who are able to have children must have a negative blood or urine pregnancy test.

If you are found to be eligible to take part in this study, you will receive erythropoietin as an injection under the skin once a week and G-CSF as an injection under the skin 1-2 times a week for as long as you respond well to treatment. You will take prednisone by mouth every day for a month and cyclosporin tablets by mouth every day for 6 months.

During this study, you will need to visit your doctor for a physical exam and measurement of your vital signs. The frequency of doctor visits will vary depending on your physical condition, but will be required at least once every 3 months.

Blood tests (about 2 teaspoons) will be done about every 1-2 weeks during the first 12 weeks of treatment, then every 2 to 4 weeks for the remainder of the study. The blood samples will be collected for routine lab tests. Periodic (every 3 to 6 months) bone marrow samples will also be taken to check cells related to the disease before, during, and after completion of this study.

You will be taken off study if the disease gets worse or intolerable side effects occur.

This is an investigational study. All drugs are FDA approved and commercially available. Up to 60 patients will take part in this study. All will be enrolled at M. D. Anderson.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients with MDS and </= 10% blasts or International Prognostic Scoring System (IPSS) low or intermediate 1. No prior intensive chemotherapy or high-dose ara-C (>/= 1g/m2). Prior cytokines, biologic therapies, targeted therapies, or single agent chemotherapy allowed. Procrit, G-CSF are allowed before therapy. Patients with blasts < 5% must have an indication for therapy, such as transfusion needs, symptomatic anemia or Hb < 11g/dl, platelets < 100 x 10 9/L, or granulocytes < 10 9/L.
  2. Performance 0-2 (Eastern Cooperative Oncology Group (ECOG)). Adequate liver function (bilirubin of < 2mg/dl) and renal function (creatinine < 2mg/dl). Adequate cardiac functions (New York Heart Association (NYHA) cardiac III-IV excluded)
  3. Signed informed consent

Exclusion Criteria:

  1. Nursing and pregnant females are excluded. Women of childbearing potential should practice effective methods of contraception. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
  2. Patients with active and uncontrolled infections.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00520468

Locations
United States, Texas
U.T.M.D. Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
M.D. Anderson Cancer Center
Investigators
Principal Investigator: Gautam Borthakur, MBBS M.D. Anderson Cancer Center
  More Information

Additional Information:
No publications provided

Responsible Party: M.D. Anderson Cancer Center
ClinicalTrials.gov Identifier: NCT00520468     History of Changes
Other Study ID Numbers: 2004-0253
Study First Received: August 23, 2007
Results First Received: September 24, 2009
Last Updated: August 1, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by M.D. Anderson Cancer Center:
Myelodysplastic Syndrome
MDS
Erythropoietin
Darbepoetin alfa
Aranesp
Erythropoiesis stimulating protein
G-CSF
Filgrastim
Neupogen
Prednisone
Cyclosporin A
Sandimmune
CYA
Cyclosporine

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Syndrome
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Disease
Pathologic Processes
Cyclosporins
Cyclosporine
Darbepoetin alfa
Epoetin alfa
Prednisone
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Therapeutic Uses
Dermatologic Agents
Antirheumatic Agents
Hematinics
Hematologic Agents
Anti-Inflammatory Agents
Glucocorticoids
Hormones

ClinicalTrials.gov processed this record on October 19, 2014