Categories:
Diseases Treated:
Acute myeloid leukemia (AML), acute undifferentiated leukemia, mixed phenotype acute leukemia
Eligibility Overview:
- Diagnosis of relapsed or refractory acute myeloid leukemia (AML), acute undifferentiated leukemia or mixed phenotype acute leukemia
- At least 2 years old and younger than 22 years old
- Recovered from acute effects of prior therapy and no evidence of graft-versus-host disease (GVHD)
- Adequate liver, kidney and heart functions
-
Brief Summary
Current survival rates for children with refractory or relapsed AML are poor, with fewer than 40% of these patients becoming long-term survivors. Most treatments for patients with relapsed AML are based on conventional chemotherapeutic agents, such as cytarabine, followed by hematopoietic stem cell transplantation. This clinical trial will study the safety and activity of venetoclax, a BCL-2 inhibitor, in combination with chemotherapy for pediatric patients with relapsed or refractory AML. Preclinical and emerging clinical data have shown venetoclax to be active and tolerable in adult patients with AML. The early responses seen in adult patients treated with venetoclax and very low-dose cytarabine suggest treatment with venetoclax may sensitize AML cells to cytarabine.
The FDA has approved venetoclax to treat chronic lymphocytic leukemia (CLL) in patients who have failed prior therapy.
Primary Objective
- To determine a tolerable combination of venetoclax plus chemotherapy in pediatric patients with relapsed or refractory AML
Secondary Objective
- To estimate the overall response rate to the combination of venetoclax and chemotherapy in pediatric patients with relapsed or refractory AML.
Eligibility Criteria
Inclusion criteria, among others:
- Diagnosis of relapsed or refractory acute myeloid leukemia (AML), acute undifferentiated leukemia or mixed phenotype acute leukemia
- At least 2 years old and younger than 22 years old
- Recovered from acute effects of prior therapy and no evidence of graft-versus-host disease (GVHD)
- Adequate liver, kidney and heart functions
Exclusion Criteria include:
- Pregnancy or breastfeeding
- Down syndrome, acute promyelocytic leukemia, juvenile myelomonocytic leukemia, or bone marrow failure syndrome
- Gastrointestinal impairment
Study Sites
St. Jude Children’s Research Hospital
Memphis, TennesseeCollaborating sites in the U.S.
-
About this study
This study will test a new drug, called venetoclax, in children with acute myeloid leukemia (AML) that did not respond to prior therapy (refractory) or that came back after previous treatment (relapsed). Venetoclax is also known as also called Venclexta®. It is a type of drug called a BCL-2 inhibitor. The drug binds to a protein called BCL-2, which may be found on some types of leukemia cells. Blocking this protein may help kill cancer cells and make them more sensitive to other anti-cancer chemotherapy drugs.
The FDA has approved venetoclax to treat another kind of leukemia, called chronic lymphocytic leukemia (CLL), in adults who have received other treatments. The FDA has not approved venetoclax to treat childhood AML.
Purpose of this clinical trial
The main purpose of this study is to find out the best dose of venetoclax and chemotherapy that can be given safely to children with AML. Researchers also want to find out how AML responds to this combination treatment.
Eligibility overview
- Diagnosis of relapsed or refractory acute myeloid leukemia (AML), acute undifferentiated leukemia or mixed phenotype acute leukemia
- At least 2 years old and younger than 22 years old
- Recovered from acute effects of prior therapy and no evidence of graft-versus-host disease (GVHD)
- Adequate liver, kidney and heart functions
-
VENAML Quick View Sponsors St. Jude Children's Research Hospital ClinicalTrials.gov identifier NCT03194932 Trial Start Date July 2017 Estimated Enrollment 54 Study Type Interventional Study Phase Phase I Conditions Acute myeloid leukemia (AML), acute undifferentiated leukemia or mixed phenotype acute leukemia Ages At least 2 years old and younger than 22 Principal investigator Jeffrey Rubnitz, MD, PhD Study Sites St. Jude Children's Research Hospital and collaborating sites in the U.S. For a consultation or to discuss VENAML St. Jude Physician/Patient Referral Office
1-888-226-4343
referralinfo@stjude.org
Contact
Jeffrey Rubnitz, MD, PhD
Email: jeffrey.rubnitz@stjude.org
St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105 USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334
Email: referralinfo@stjude.org
The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.