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Enzyme
Chemotherapy for Acute Myeloid Leukemia in Young Patients with Down Syndrome
Phase 3
Waitlist Available
Led By Jason N Berman
Research Sponsored by Children's Oncology Group
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Patient has previously untreated de novo AML and meets the criteria for AML with >= 20% bone marrow blasts as set out in the World Health Organization (WHO) Myeloid Neoplasm classification
Patients must have constitutional trisomy 21 (Down syndrome) or trisomy 21 mosaicism (by karyotype or fluorescence in situ hybridization [FISH])
Must not have
Patients with promyelocytic leukemia (French-American-British [FAB] M3)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 2 years from study entry
Awards & highlights
Summary
This trial studies a chemotherapy treatment that adjusts based on how well patients respond initially. It targets younger patients with Down syndrome who have certain types of blood cancer. The treatment aims to effectively kill cancer cells while reducing side effects.
Who is the study for?
This trial is for young patients with Down syndrome who have newly diagnosed acute myeloid leukemia or myelodysplastic syndrome. They must not have received any anti-leukemic therapy except cytarabine for transient myeloproliferative disease, and they should not have promyelocytic leukemia. Patients need to show a certain percentage of blasts in their bone marrow or blood and meet specific diagnostic criteria.
What is being tested?
The study tests response-based chemotherapy using drugs like Asparaginase Erwinia chrysanthemi, Cytarabine, Daunorubicin Hydrochloride, among others. It aims to categorize patients by risk based on their initial treatment response and adjust subsequent treatments accordingly to reduce side effects while treating the cancer effectively.
What are the potential side effects?
Chemotherapy drugs used may cause side effects such as nausea, vomiting, hair loss, increased risk of infection due to low blood cell counts, bleeding or bruising more easily than normal due to low platelet counts, fatigue from anemia (low red blood cell count), and potential liver problems.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have a new diagnosis of AML with more than 20% bone marrow blasts.
Select...
I have Down syndrome confirmed by genetic testing.
Select...
I have a blood disorder with low blood counts but not enough to be considered AML.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I have been diagnosed with a specific type of leukemia (FAB M3).
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 2 years from study entry
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 2 years from study entry
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Event-free survival (EFS)
Other study objectives
Mean duration of hospitalization
Mean length on protocol therapy
Mean time to absolute neutrophil count (ANC) recovery
+6 moreTrial Design
2Treatment groups
Experimental Treatment
Group I: Arm B (high risk)Experimental Treatment5 Interventions
INDUCTION II: Patients receive high dose cytarabine IV over 1-3 hours Q12 hours on days 1-4 and mitoxantrone hydrochloride IV over 15-30 minutes on days 3-6. Induction II continues for a minimum of 28 days.
INTENSIFICATION I: Patients receive high dose cytarabine IV over 1-3 hours Q12 hours and etoposide IV over 90-120 minutes on days 1-5. Intensification I continues for a minimum of 28 days.
INTENSIFICATION II: Patients receive high dose cytarabine IV over 3 hours Q12 hours on days 1, 2, 8, and 9. Patients also receive asparaginase or asparaginase Erwinia chrysanthemi IM or IV over 30 minutes on days 2 and 9. Intensification II continues for a minimum of 28 days.
Group II: Arm A (standard risk)Experimental Treatment5 Interventions
INDUCTION II: Patients receive cytarabine IV continuously over 96 hours, daunorubicin hydrochloride IV over 1-15 minutes, and thioguanine PO BID on days 1-4. Induction II continues for a minimum of 28 days.
INDUCTION III: Patients receive cytarabine, daunorubicin hydrochloride, and thioguanine as in Induction II. Induction III continues for a minimum of 28 days.
INTENSIFICATION I: Patients receive cytarabine IV continuously over 168 hours on days 1-7 and etoposide IV over 60-120 minutes on days 1-3. Intensification I continues for a minimum of 28 days.
INTENSIFICATION II: Patients receive cytarabine and etoposide as in Intensification I. Intensification II continues for a minimum of 28 days.
(This arm is closed to accrual and treatment with amendment #4A 01/07/2019)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Asparaginase
2005
Completed Phase 4
~5220
Cytarabine
2016
Completed Phase 3
~3330
Daunorubicin Hydrochloride
2011
Completed Phase 3
~5330
Thioguanine
2012
Completed Phase 4
~10830
Mitoxantrone Hydrochloride
2016
Completed Phase 3
~650
Etoposide
2010
Completed Phase 3
~2960
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for Myeloid Leukemia include chemotherapy, targeted therapy, and sometimes immunotherapy. Chemotherapy works by killing rapidly dividing cancer cells, which helps to reduce the number of leukemic cells in the body.
Targeted therapies, such as tyrosine kinase inhibitors, specifically inhibit molecules involved in the growth and survival of cancer cells, thereby stopping them from dividing and spreading. Immunotherapy boosts the body's immune system to recognize and destroy cancer cells.
These treatments are essential for Myeloid Leukemia patients as they address the aggressive nature of the disease by directly targeting the mechanisms that allow leukemic cells to proliferate and spread.
Molecular targeting in acute myeloid leukemia.Targeting phosphatidylinositol 3-kinase signaling in acute myelogenous leukemia.Remission induction, consolidation and novel agents in development for adults with acute myeloid leukaemia.
Molecular targeting in acute myeloid leukemia.Targeting phosphatidylinositol 3-kinase signaling in acute myelogenous leukemia.Remission induction, consolidation and novel agents in development for adults with acute myeloid leukaemia.
Find a Location
Who is running the clinical trial?
National Cancer Institute (NCI)NIH
13,868 Previous Clinical Trials
41,010,557 Total Patients Enrolled
Children's Oncology GroupLead Sponsor
457 Previous Clinical Trials
239,550 Total Patients Enrolled
Jason N BermanPrincipal InvestigatorChildren's Oncology Group
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- It has been over 8 weeks since my temporary blood disorder with high blast cells resolved.I received my last cytarabine dose for TMD treatment within the last 30 days.I have a new diagnosis of AML with more than 20% bone marrow blasts.I have Down syndrome confirmed by genetic testing.My organ functions are not a concern for this study's eligibility.I've tried or cannot undergo a bone marrow test due to health reasons, but my blood test shows more than 20% blasts.I have a blood disorder with low blood counts but not enough to be considered AML.My child has not had cancer treatment except for cytarabine for TMD.Your bone marrow tests show a high number of blast cells that are increasing over time.I had a temporary blood disorder but don't have AML or MDS.I have had heart surgery before, but my heart works well now.I have been diagnosed with a specific type of leukemia (FAB M3).
Research Study Groups:
This trial has the following groups:- Group 1: Arm A (standard risk)
- Group 2: Arm B (high risk)
Awards:
This trial has 2 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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