NCT00004871

Brief Summary

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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 7, 2000

Completed
2 months until next milestone

Study Start

First participant enrolled

May 1, 2000

Completed
2.7 years until next milestone

First Posted

Study publicly available on registry

January 27, 2003

Completed
Last Updated

March 10, 2010

Status Verified

March 1, 2010

First QC Date

March 7, 2000

Last Update Submit

March 9, 2010

Conditions

LeukemiaMyelodysplastic SyndromesMyelodysplastic/Myeloproliferative Diseases

Keywords

recurrent adult acute myeloid leukemiauntreated adult acute myeloid leukemiarefractory anemiarefractory anemia with ringed sideroblastsrefractory anemia with excess blastsrefractory anemia with excess blasts in transformationchronic myelomonocytic leukemiade novo myelodysplastic syndromespreviously treated myelodysplastic syndromessecondary myelodysplastic syndromesatypical chronic myeloid leukemiamyelodysplastic/myeloproliferative disease, unclassifiable

Interventions

azacitidineDRUG
sodium phenylbutyrateDRUG

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
DISEASE CHARACTERISTICS: * Histologically or cytologically confirmed myelodysplastic syndrome (MDS) indicating one of the following: * Refractory anemia (RA) * Primary refractory leukopenia or thrombocytopenia with MDS morphology * RA with excess blasts (RAEB) * RA with ringed sideroblasts (RARS) * Chronic myelomonocytic leukemia * RAEB in transformation * RA or RARS must have at least one of the following: * Absolute neutrophil count less than 1,000/mm\^3 * Untransfused hemoglobin less than 8 g/dL * Platelet count less than 20,000/mm\^3 * Anemia * Thrombocytopenia requiring transfusion * High risk chromosomal abnormalities * Any stage of MDS allowed including: * Previously untreated MDS * Refractory MDS allowed if failure to achieve remission following prior intensive chemotherapy of at least 1 month ago * Relapsed, refractory, or untreated acute myeloid leukemia (AML) with the following: * WBC less than 30,000/mm\^3 * Stable for at least 2 weeks * Unlikely to require cytotoxic therapy during study * Untreated AML with poor risk factors for response to standard therapy including: * Greater than 60 years old * AML occurs in setting of antecedent hematologic disorder * High risk chromosomes (e.g., abnormalities of chromosome 5 or 7 or complex cytogenetic abnormalities) * Medical conditions that preclude cytotoxic chemotherapy as primary therapy * Refusal of cytotoxic chemotherapy allowed * No clinical evidence of CNS leukostasis or CNS leukemia PATIENT CHARACTERISTICS: Age: * 18 and over Performance status: * Zubrod 0-2 Life expectancy: * Not specified Hematopoietic: * See Disease Characteristics * Hemoglobin at least 8 g/dL (transfusion allowed) Hepatic: * Bilirubin less than 2.0 mg/dL (unless due to hemolysis or Gilbert's disease) Renal: * Creatinine less than 2.0 mg/dL Cardiovascular: * No disseminated intravascular coagulation Pulmonary: * No pulmonary leukostasis Other: * No active infection * Not pregnant or nursing * Negative pregnancy test * Fertile patients must use effective contraception 2 weeks prior, during and 3 months after study PRIOR CONCURRENT THERAPY: Biologic therapy: * At least 3 weeks since prior biologic therapy including colony stimulating factors and recovered Chemotherapy: * See Disease Characteristics * At least 3 weeks since prior chemotherapy and recovered Endocrine therapy: * At least 3 weeks since prior hormonal therapy and recovered Radiotherapy: * At least 3 weeks since prior radiotherapy and recovered Surgery: * Not specified

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Baltimore, Maryland, 21231-2410, United States

Location

MeSH Terms

Conditions

LeukemiaMyelodysplastic SyndromesMyelodysplastic-Myeloproliferative DiseasesLeukemia, Myeloid, AcuteAnemia, RefractoryAnemia, Refractory, with Excess of BlastsLeukemia, Myelomonocytic, ChronicLeukemia, Myeloid, Chronic, Atypical, BCR-ABL NegativeMyeloproliferative Disorders

Interventions

Azacitidine4-phenylbutyric acid

Condition Hierarchy (Ancestors)

Neoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow DiseasesLeukemia, MyeloidAnemiaChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Aza CompoundsOrganic ChemicalsCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Study Officials

  • Steven D. Gore, MD

    Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Purpose
TREATMENT
Sponsor Type
OTHER

Study Record Dates

First Submitted

March 7, 2000

First Posted

January 27, 2003

Study Start

May 1, 2000

Last Updated

March 10, 2010

Record last verified: 2010-03

Locations

US(1)