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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])

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 2 years

Awards & highlights

Study Summary

This trial is studying how effective response-based chemotherapy is in treating younger patients with Down syndrome who have acute myeloid leukemia or myelodysplastic syndrome.

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.Check my eligibility

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.See study design

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 below

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I have a new diagnosis of AML with more than 20% bone marrow blasts.

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I have Down syndrome confirmed by genetic testing.

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I have a blood disorder with low blood counts but not enough to be considered AML.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Event-free survival probability at 2 years

Other outcome measures

Mean duration of hospitalization

Mean length on protocol therapy

Mean time to absolute neutrophil count (ANC) recovery

+6 more

Trial 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

Cytarabine

2016

Completed Phase 3

~3310

Asparaginase

2005

Completed Phase 4

~5000

Daunorubicin Hydrochloride

2011

Completed Phase 3

~5070

Thioguanine

2012

Completed Phase 4

~10830

Mitoxantrone Hydrochloride

2016

Completed Phase 3

~650

Etoposide

2010

Completed Phase 3

~2440

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

National Cancer Institute (NCI)NIH

13,654 Previous Clinical Trials

40,932,841 Total Patients Enrolled

Children's Oncology GroupLead Sponsor

453 Previous Clinical Trials

237,388 Total Patients Enrolled

Jason N BermanPrincipal InvestigatorChildren's Oncology Group

Media Library

Asparaginase (Enzyme) Clinical Trial Eligibility Overview. Trial Name: NCT02521493 — Phase 3

Myeloid Leukemia Research Study Groups: Arm A (standard risk), Arm B (high risk)

Myeloid Leukemia Clinical Trial 2023: Asparaginase Highlights & Side Effects. Trial Name: NCT02521493 — Phase 3

Asparaginase (Enzyme) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02521493 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are the most common conditions that Cytarabine is used to improve?

"Cytarabine can be used to help patients with small cell lung cancer (sclc), leukemia, myelocytic, acute, and lymphoma."

Answered by AI

Is Cytarabine a common drug in other research?

"City of Hope Comprehensive Cancer Center was the first to study cytarabine in 1997. So far, there have been a total of 1,678 completed clinical trials worldwide. As of right now, 633 studies are ongoing; many of these trials taking place in Fort Myers, Florida."

Answered by AI

What is the estimated total number of participants for this experiment?

"Currently, this trial is not enrolling patients. It was initially posted on November 23rd, 2015 and updated for the last time on October 19th, 2020. However, if you are looking for other studies, there are 2949 trials actively admitting participants with down syndrome and 633 trials for Cytarabine that are still recruiting patients."

Answered by AI

Might this clinical trial have benefits for elderly patients?

"According to the recruitment guidelines for this clinical trial, applicants must be between 91 days and 3 years old."

Answered by AI

Does this research trial have many sites in America?

"This clinical trial is seeking patients from 100 different hospitals, including Golisano Children's Hospital of Southwest Florida in Fort Myers, Florida, Blank Children's Hospital in Des Moines, Iowa, and Beaumont Children's Hospital-Royal Oak in Royal Oak, Michigan."

Answered by AI

Could I be a participant of this research project?

"This trial is looking for 312 patients with Down syndrome that are between 91 days and 3 years old. The applicants must meet the following requirements: They cannot have received any prior chemotherapy, radiation therapy or other anti-leukemic treatments (with the exception of cytarabine for TMD), they must have trisomy 21 or trisomy 21 mosaicism, attempts to obtain a bone marrow sample must be made unless it is clinically prohibitive, and they must have cytopenias and/or bone marrow blasts but not meet the criteria for AML diagnosis. There are no minimal organ function requirements for enrollment."

Answered by AI

What is the maximum dosage of Cytarabine that patients can safely take?

"Cytarabine has been deemed safe by our analysts at Power. This is due to Phase 3 trials providing some evidence of efficacy as well as numerous rounds of data supporting safety."

Answered by AI

Recent research and studies

BloodJournal

High-dose Arac Is Essential for the Treatment of ML-DS Independent of Postinduction MRD: Results of the COG AAML1531 Trial

Hitzler, Johann, Todd Alonzo, Robert Gerbing, Amy Beckman, Betsy Hirsch, Susana Raimondi, Karen Chisholm, et al.. 2021. “High-dose Arac Is Essential for the Treatment of ML-DS Independent of Postinduction MRD: Results of the COG AAML1531 Trial”. Blood. American Society of Hematology. doi:10.1182/blood.2021012206.

clinicaltrials.govStudy

Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome

2015. "Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome in Younger Patients With Down Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02521493.

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