Ignite Creation Date:
2025-12-26 @ 10:18 PM
Ignite Modification Date:
2025-12-26 @ 10:18 PM
Study NCT ID:
NCT04915612
Status:
COMPLETED
Last Update Posted:
2025-04-27
First Post:
2021-02-25
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Liposomal Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin for the Treatment of Relapsed Refractory Pediatric Patients With Acute Myeloid Leukemia
Sponsor:
M.D. Anderson Cancer Center
Organization:
Study Overview
Official Title:
A Phase I Study of Liposomal Cytarabine and Daunorubicin (CPX-351) in Combination With Gemtuzumab Ozogamicin (GO) in Relapsed Refractory Pediatric Patients With Acute Myeloid Leukemia (AML)
Status:
COMPLETED
Status Verified Date:
2025-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This phase I trial studies the best dose and side effects of liposomal cytarabine, daunorubicin, and gemtuzumab ozogamicin in treating pediatric patients with acute myeloid leukemia that has returned after treatment (relapsed) or does not respond to treatment (refractory). Chemotherapy drugs, such as liposomal cytarabine and daunorubicin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Gemtuzumab ozogamicin is a monoclonal antibody, called gemtuzumab, linked to a toxic agent called ozogamicin. Gemtuzumab attaches to CD33 positive cancer cells in a targeted way and delivers ozogamicin to kill them. Giving liposomal cytarabine and daunorubicin and gemtuzumab ozogamicin may help to control the disease.
Detailed Description:
PRIMARY OBJECTIVE:
I. To determine the maximum tolerated dose (MTD) and safety of liposomal cytarabine and daunorubicin (CPX-351) in combination with gemtuzumab ozogamicin (GO) in relapsed refractory pediatric patients with acute myeloid leukemia (AML).
SECONDARY OBJECTIVE:
I. To determine the preliminary assessment of efficacy by overall response (OR), including complete remission (CR), CR with incomplete blood count recovery and partial remission), overall survival (OS), event-free survival (EFS) and duration of response (DOR) of pediatric patients treated with this combination.
EXPLORATORY OBJECTIVES:
I. To determine the minimal residual disease (MRD) after treatment with this combination and its impact in long-term outcome (OS and EFS).
II. To determine the effect of the level of pre-treatment expression of CD33 with response to this combination.
III. To determine the effect of this treatment combination on responding pediatric patients transitioning to hematopoietic stem cell transplant (HSCT) i.e., number and percentage of patients that are able to transition to HSCT.
OUTLINE:
INDUCTION 1 (28 days): Patients receive CPX-351 intravenously (IV) over 90 minutes on days 1, 3, and 5 and GO IV over 2 hours on day 1 in the absence of disease progression or unacceptable toxicity.
INDUCTION 2: Patients who do not attain a defined clinical response after cycle Induction 1 receive CPX-351 IV on days 1 and 3 and GO IV over 2 hours on day 1 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Beginning 4 weeks after last induction, patients receive CPX-351 IV over 90 minutes on days 1 and 3 and GO IV over 2 hours on day 1 in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 28 days.
I. To determine the maximum tolerated dose (MTD) and safety of liposomal cytarabine and daunorubicin (CPX-351) in combination with gemtuzumab ozogamicin (GO) in relapsed refractory pediatric patients with acute myeloid leukemia (AML).
SECONDARY OBJECTIVE:
I. To determine the preliminary assessment of efficacy by overall response (OR), including complete remission (CR), CR with incomplete blood count recovery and partial remission), overall survival (OS), event-free survival (EFS) and duration of response (DOR) of pediatric patients treated with this combination.
EXPLORATORY OBJECTIVES:
I. To determine the minimal residual disease (MRD) after treatment with this combination and its impact in long-term outcome (OS and EFS).
II. To determine the effect of the level of pre-treatment expression of CD33 with response to this combination.
III. To determine the effect of this treatment combination on responding pediatric patients transitioning to hematopoietic stem cell transplant (HSCT) i.e., number and percentage of patients that are able to transition to HSCT.
OUTLINE:
INDUCTION 1 (28 days): Patients receive CPX-351 intravenously (IV) over 90 minutes on days 1, 3, and 5 and GO IV over 2 hours on day 1 in the absence of disease progression or unacceptable toxicity.
INDUCTION 2: Patients who do not attain a defined clinical response after cycle Induction 1 receive CPX-351 IV on days 1 and 3 and GO IV over 2 hours on day 1 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION: Beginning 4 weeks after last induction, patients receive CPX-351 IV over 90 minutes on days 1 and 3 and GO IV over 2 hours on day 1 in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up at 28 days.
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?: