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A Pilot Study of Phenylbutyrate, Dexamethasone and GM-CSF in Refractory or Relapsed t(8;21) Acute Myeloid Leukemia


Phase 2
18 Years
N/A
Not Enrolling
Both
Leukemia

Thank you

Trial Information

A Pilot Study of Phenylbutyrate, Dexamethasone and GM-CSF in Refractory or Relapsed t(8;21) Acute Myeloid Leukemia


OBJECTIVES:

- Determine the objective response (complete hematologic remission induction) of
phenylbutyrate, dexamethasone, and sargramostim (GM-CSF) in patients with refractory or
relapsed t(8;21) acute myeloid leukemia.

- Determine the correlation between histone acetylation, differentiation, and apoptosis
in bone marrow mononuclear cells with response rate in patients treated with this
regimen.

- Determine the overall survival of patients on this regimen.

- Determine the correlation between histone acetylation, differentiation, and apoptosis
in bone marrow mononuclear cells with pharmacokinetics of this regimen in these
patients.

- Determine the safety and toxicity of this regimen in these patients.

OUTLINE: This is a multicenter study.

Patients receive phenylbutyrate IV continuously and sargramostim (GM-CSF) subcutaneously on
days 1-7 and 15-21. Patients also receive oral dexamethasone on days 1-4 and 15-18.
Treatment continues every 28 days in the absence of disease progression or unacceptable
toxicity until complete hematologic remission is induced. Patients with stable disease at
the end of 1 course receive at least 2 additional courses.

Patients are followed twice a week for 3 months, monthly for 1 year, every three months for
the next 4 years, and then annually thereafter.

PROJECTED ACCRUAL: A total of 9-24 patients will be accrued for this study in at least 2
years.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- Diagnosis of t(8;21) acute myeloid leukemia (AML)

- Failed standard induction chemotherapy or stem cell transplantation (SCT) OR

- Relapsed after standard induction chemotherapy or SCT OR

- Refused or not a candidate for SCT or matched allogeneic sibling bone marrow
transplantation or donor lymphocyte infusion OR

- Refused of not a candidate for autologous SCT or bone marrow transplantation

- No CNS leukemia

PATIENT CHARACTERISTICS:

Age:

- 18 and over

Performance status:

- ECOG 0-2

Life expectancy:

- At least 7 days

Hematopoietic:

- Not specified

Hepatic:

- AST or ALT no greater than 3 times upper limit of normal (ULN)

- Bilirubin no greater than 3 times ULN

- No hepatic disease that would preclude study

Renal:

- Creatinine no greater than 2 mg/dL

- Creatinine clearance at least 60 mL/min

- No renal disease that would preclude study

Cardiovascular:

- No cardiac disease that would preclude study

- No New York Heart Association class III or IV heart disease

- No myocardial infarction within past 8 weeks

Other:

- No active infection except cystitis

- Not pregnant or nursing

- No altered mental status or seizure disorder

- No other serious disease that would preclude study

PRIOR CONCURRENT THERAPY:

Biologic therapy:

- See Disease Characteristics

Chemotherapy:

- See Disease Characteristics

Endocrine therapy:

- Not specified

Radiotherapy:

- Not specified

Surgery:

- Not specified

Other:

- At least 3 weeks since prior investigational antineoplastic drugs

Type of Study:

Interventional

Study Design:

Primary Purpose: Treatment

Principal Investigator

Johnson Liu, MD

Investigator Role:

Study Chair

Investigator Affiliation:

National Heart, Lung, and Blood Institute (NHLBI)

Authority:

United States: Federal Government

Study ID:

CDR0000068165

NCT ID:

NCT00006240

Start Date:

October 2000

Completion Date:

August 2001

Related Keywords:

  • Leukemia
  • recurrent adult acute myeloid leukemia
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid

Name

Location

Mount Sinai Medical Center, NYNew York, New York  10029
University of Pittsburgh Cancer InstitutePittsburgh, Pennsylvania  15213
Warren Grant Magnuson Clinical Center - NCI Clinical Studies SupportBethesda, Maryland  20892-1182
National Heart, Lung, and Blood InstituteBethesda, Maryland  20892