Ignite Creation Date:
2024-05-05 @ 11:21 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004871
Status:
COMPLETED
Last Update Posted:
2010-03-10
First Post:
2000-03-07
Brief Title:
Azacitidine Plus Phenylbutyrate in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome
Sponsor:
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Organization:
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Study Overview
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
Status:
COMPLETED
Status Verified Date:
2010-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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
PURPOSE Phase I trial to study the effectiveness of combining azacitidine and phenylbutyrate in treating patients who have acute myeloid leukemia or myelodysplastic syndrome
Detailed Description:
OBJECTIVES
Determine the safety and toxicity of azacitidine in combination with phenylbutyrate in patients with recurrent refractory or untreated acute myeloid leukemia or myelodysplastic syndrome
Determine the minimal effective pharmacologic dose of azacitidine required to consistently inhibit DNA methyltransferase in this patient population
Obtain preliminary clinical andor laboratory data suggesting potential therapeutic activity of this combination regimen in these patients
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
Determine the safety and toxicity of azacitidine in combination with phenylbutyrate in patients with recurrent refractory or untreated acute myeloid leukemia or myelodysplastic syndrome
Determine the minimal effective pharmacologic dose of azacitidine required to consistently inhibit DNA methyltransferase in this patient population
Obtain preliminary clinical andor laboratory data suggesting potential therapeutic activity of this combination regimen in these patients
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
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| JHOC-J9950 | US NIH GrantContract | None | httpsreporternihgovquickSearchP30CA006973 |
| U01CA070095 | NIH | None | None |
| R01CA067803 | NIH | None | None |
| P30CA006973 | NIH | None | None |
| JHOC-99072307 | None | None | None |
| NCI-T99-0092 | None | None | None |