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Sponsors and Collaborators: |
Sidney Kimmel Comprehensive Cancer Center National Cancer Institute (NCI) |
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Information provided by: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00004871 |
RATIONALE: Azacitidine plus phenylbutyrate may help leukemia cells develop into normal white blood cells.
PURPOSE: Phase I trial to study the effectiveness of combining azacitidine and phenylbutyrate in treating patients who have acute myeloid leukemia or myelodysplastic syndrome.
Condition | Intervention | Phase |
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Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases |
Drug: azacitidine Drug: sodium phenylbutyrate |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment |
Official Title: | Phase I, Dose De-Escalation to Minimal Effective Pharmacologic Dose Trial of Sodium Phenylbutyrate (PB, NSC 657802) in Combination With 5-Azacytidine (5-AZA, NSC 102816) in Patients With Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML) |
Study Start Date: | May 2000 |
OBJECTIVES:
OUTLINE: This is a dose deescalation study of azacitidine.
Patients receive azacitidine subcutaneously daily on days 1-5 and 29-33 followed by phenylbutyrate IV continuously on days 5-12 and 33-40. Treatment continues for at least 2 courses in the absence of disease progression. Patients with responsive disease may receive an additional 2 months of therapy.
Cohorts of 3-6 patients receive deescalating doses of azacitidine until the minimal effective pharmacologic dose (MEPD) is determined. The MEPD is defined as the dose above the dose at which more than 1 of 6 patients do not meet the target enzyme inhibition of greater than 90%.
Once the MEPD and toxicity have been established for a 5 day schedule, daily dose schedule of azacitidine is increased to 10, 14, and 21 days, followed by phenylbutyrate for 7 days. Courses are repeated every 28 days.
PROJECTED ACCRUAL: Approximately 32 patients will be accrued for this study within 2 years.
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Histologically or cytologically confirmed myelodysplastic syndrome (MDS) indicating one of the following:
RA or RARS must have at least one of the following:
Any stage of MDS allowed including:
Relapsed, refractory, or untreated acute myeloid leukemia (AML) with the following:
Untreated AML with poor risk factors for response to standard therapy including:
PATIENT CHARACTERISTICS:
Age:
Performance status:
Life expectancy:
Hematopoietic:
Hepatic:
Renal:
Cardiovascular:
Pulmonary:
Other:
PRIOR CONCURRENT THERAPY:
Biologic therapy:
Chemotherapy:
Endocrine therapy:
Radiotherapy:
Surgery:
United States, Maryland | |
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | |
Baltimore, Maryland, United States, 21231-2410 |
Study Chair: | Steven D. Gore, MD | Sidney Kimmel Comprehensive Cancer Center |
Study ID Numbers: | CDR0000067531, JHOC-99072307, NCI-T99-0092, JHOC-J9950 |
Study First Received: | March 7, 2000 |
Last Updated: | July 23, 2008 |
ClinicalTrials.gov Identifier: | NCT00004871 History of Changes |
Health Authority: | United States: Federal Government |
recurrent adult acute myeloid leukemia untreated adult acute myeloid leukemia refractory anemia refractory anemia with ringed sideroblasts refractory anemia with excess blasts refractory anemia with excess blasts in transformation |
chronic myelomonocytic leukemia de novo myelodysplastic syndromes previously treated myelodysplastic syndromes secondary myelodysplastic syndromes atypical chronic myeloid leukemia myelodysplastic/myeloproliferative disease, unclassifiable |
Antimetabolites Chronic Myelomonocytic Leukemia Precancerous Conditions Leukemia, Myeloid, Acute Refractory Anemia Leukemia Preleukemia Acute Myelocytic Leukemia Anemia, Refractory Acute Myeloid Leukemia, Adult Neoplasm Metastasis Azacitidine Myelodysplastic Myeloproliferative Disease 4-phenylbutyric acid |
Hematologic Diseases Leukemia, Myeloid, Chronic, Atypical, BCR-ABL Negative Leukemia, Myelomonocytic, Chronic Myelodysplastic Syndromes Myeloproliferative Disorders Anemia Leukemia, Myeloid Recurrence Leukemia, Myelogenous, Chronic, BCR-ABL Positive Anemia, Refractory, with Excess of Blasts Chronic Myelogenous Leukemia Myelodysplastic-Myeloproliferative Diseases Bone Marrow Diseases |
Antimetabolites Antimetabolites, Antineoplastic Disease Neoplasms by Histologic Type Molecular Mechanisms of Pharmacological Action Precancerous Conditions Antineoplastic Agents 4-phenylbutyric acid Hematologic Diseases Myelodysplastic Syndromes Myeloproliferative Disorders Enzyme Inhibitors |
Leukemia, Myeloid Leukemia, Myeloid, Acute Pharmacologic Actions Leukemia Preleukemia Neoplasms Pathologic Processes Therapeutic Uses Syndrome Azacitidine Myelodysplastic-Myeloproliferative Diseases Bone Marrow Diseases |