LeukemiaPhase 2NCT00006240
Phenylbutyrate, Dexamethasone, and Sargramostim in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia
A Phase 2 study testing sargramostim, dexamethasone and oral sodium phenylbutyrate in people with Leukemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 4 sites in United States.
Trial summary
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leukemia
- What is being tested?
- sargramostim, dexamethasone and oral sodium phenylbutyrate
- Type of treatment
- Pill or oral medicine
- How many people
- Not given
- Where
- 4 sitesUnited States
- Study dates
- Oct 2000to Aug 2001
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support
Bethesda, Maryland, 20892-1182
National Heart, Lung, and Blood Institute
Bethesda, Maryland, 20892
Mount Sinai Medical Center, NY
New York, New York, 10029
University of Pittsburgh Cancer Institute
Pittsburgh, Pennsylvania, 15213-3489
About this study
Official title: A Pilot Study of Phenylbutyrate, Dexamethasone and GM-CSF in Refractory or Relapsed t(8;21) Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of chemotherapy.
PURPOSE: Phase II trial to study the effectiveness of combining phenylbutyrate, dexamethasone, and sargramostim in treating patients who have refractory or relapsed acute myeloid leukemia.
Read the detailed description
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.
- Conditions
- Leukemia
- Being tested
- sargramostim, dexamethasone, oral sodium phenylbutyrate
- Keywords
- recurrent adult acute myeloid leukemia
- Medical subject headings
- Leukemia, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sargramostimBiological
dexamethasoneDrug
oral sodium phenylbutyrateDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jul 2003 (estimated)
- Study startOct 2000
- Study completionAug 2001
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Investigators
- Johnson Liu, MD, Study chair, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00006240
- Sponsor's protocol ID
- CDR0000068165
- Other IDs
- NHLBI-00-H-0156, NCI-171
- Status last verified
- Nov 2002
- First submitted
- 11 Sep 2000
- Last updated
- 28 Apr 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Phenylbutyrate, Dexamethasone and GM-CSF in Refractory or Relapsed t(8;21) Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT00006240
- Acronym / sponsor ID
- CDR0000068165
- Conditions
- Leukemia
- Keywords
- recurrent adult acute myeloid leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- Oct 2000
- Study completion
- Aug 2001 (actual)
- First posted
- 28 Jul 2003 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of chemotherapy.
PURPOSE: Phase II trial to study the effectiveness of combining phenylbutyrate, dexamethasone, and sargramostim in treating patients who have refractory or relapsed acute myeloid leukemia.
Detailed description
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.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| sargramostim | Biological | – |
| dexamethasone | Drug | – |
| oral sodium phenylbutyrate | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Johnson Liu, MD study chair
National Heart, Lung, and Blood Institute (NHLBI)
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing sargramostim, dexamethasone and oral sodium phenylbutyrate in people with Leukemia, run by National Heart, Lung, and Blood Institute (NHLBI) at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Apr 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2015.