Ignite Creation Date:
2025-12-24 @ 10:19 PM
Ignite Modification Date:
2025-12-26 @ 5:38 PM
Study NCT ID:
NCT04746235
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-10-03
First Post:
2021-02-01
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Venetoclax and ASTX727 for the Treatment of Relapsed, Refractory, or Newly Diagnosed Acute Myeloid Leukemia
Sponsor:
M.D. Anderson Cancer Center
Organization:
Study Overview
Official Title:
A Phase 2 Study of Venetoclax in Combination With ASTX727 in Patients With Relapsed/Refractory Acute Myeloid Leukemia, and Newly Diagnosed Elderly Patients With AML Who Are Not Candidates for Intensive Chemotherapy
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-09
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:
This phase II trial studies the possible benefits of venetoclax and ASTX727 in treating patients with acute myeloid leukemia that has come back (relapsed) or does not respond to treatment (refractory), or elderly patients with newly diagnosed acute myeloid leukemia who are not candidates for intensive chemotherapy. Venetoclax may help block the formation of growths that may become cancer. ASTX727 is the combination of a fixed dose of 2 drugs, cedazuridine and decitabine. Cedazuridine may slow down how fast decitabine is broken down by the body, and decitabine may block abnormal cells or cancer cells from growing. Giving venetoclax and ASTX727 may help to control the disease.
Detailed Description:
PRIMARY OBJECTIVES:
I. To determine the overall response rate (ORR) of decitabine and cedazuridine (ASTX727) in combination with venetoclax in patients with refractory/relapsed acute myeloid leukemia (AML).
II. To determine the overall response rate (ORR) of ASTX727 in combination with venetoclax in elderly ((≥ 75 years or ≥ 18 years with comorbidities) patients with newly diagnosed acute myeloid leukemia (AML) not eligible for intensive chemotherapy.
SECONDARY OBJECTIVES:
I. To determine the duration of response, disease-free survival (DFS), and overall survival (OS) of patients with refractory/relapsed AML treated with this combination.
II. To determine similar outcomes for newly diagnosed patients with AML who are not candidates for intensive chemotherapy.
III. To determine the safety of venetoclax in combination with ASTX727 in patients with refractory/ relapsed AML, and newly diagnosed patients with AML not candidates for intensive chemotherapy.
IV. To determine the number of relapsed patients able to proceed to stem cell transplantation upon achieving response with the combination venetoclax/ASTX727 regimen.
EXPLORATORY OBJECTIVE:
I. To characterize the pharmacokinetic (PK) profiles of ASTX727 when combined with venetoclax.
OUTLINE:
Patients receive decitabine and cedazuridine orally (PO) daily on days 1-5 and venetoclax PO daily on days 1-28 of the first cycle and on days 1-21 of subsequent cycles. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients with an objective response are followed every 3-6 months for up to 5 years.
I. To determine the overall response rate (ORR) of decitabine and cedazuridine (ASTX727) in combination with venetoclax in patients with refractory/relapsed acute myeloid leukemia (AML).
II. To determine the overall response rate (ORR) of ASTX727 in combination with venetoclax in elderly ((≥ 75 years or ≥ 18 years with comorbidities) patients with newly diagnosed acute myeloid leukemia (AML) not eligible for intensive chemotherapy.
SECONDARY OBJECTIVES:
I. To determine the duration of response, disease-free survival (DFS), and overall survival (OS) of patients with refractory/relapsed AML treated with this combination.
II. To determine similar outcomes for newly diagnosed patients with AML who are not candidates for intensive chemotherapy.
III. To determine the safety of venetoclax in combination with ASTX727 in patients with refractory/ relapsed AML, and newly diagnosed patients with AML not candidates for intensive chemotherapy.
IV. To determine the number of relapsed patients able to proceed to stem cell transplantation upon achieving response with the combination venetoclax/ASTX727 regimen.
EXPLORATORY OBJECTIVE:
I. To characterize the pharmacokinetic (PK) profiles of ASTX727 when combined with venetoclax.
OUTLINE:
Patients receive decitabine and cedazuridine orally (PO) daily on days 1-5 and venetoclax PO daily on days 1-28 of the first cycle and on days 1-21 of subsequent cycles. Treatment repeats every 28 days for up to 24 cycles in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients with an objective response are followed every 3-6 months for up to 5 years.
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: