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DNA Methyltransferase Inhibitor

Epigenetic Priming for Acute Myeloid Leukemia

Q: Is it possible to become a participant in this research endeavor?

To gain admittance to this study, individuals must have myelodysplastic syndromes and be between the ages of 29 days and 21 years. In total, 206 patients will be accepted.

Q: Has Stem Cell Transplant been subject to prior investigation through clinical trials?

In 1995, the National Institutes of Health Clinical Center first conducted a trial on Stem Cell <a href="https://www.withpower.com/clinical-trials/transplant">Transplant</a>. In total, there have been 2,648 completed trials since then and 1,331 are currently ongoing--most notably in Memphis, <a href="https://www.withpower.com/clinical-trials/tennessee">Tennessee</a>.

Q: What is the geographic distribution of this research program?

St. Jude Children's Research Hospital in Memphis, <a href="https://www.withpower.com/clinical-trials/tennessee">Tennessee</a>, Children's Hospital of Orange County in <a href="https://www.withpower.com/clinical-trials/california">California</a> and the Children's Hospital of Central California are a few from 11 different clinical trial sites that have been selected to accept patients for this study.

Q: What medical afflictions are alleviated through Stem Cell Transplant?

Stem Cell <a href="https://www.withpower.com/clinical-trials/transplant">Transplant</a> is a viable intervention for individuals suffering from merkel cell <a href="https://www.withpower.com/clinical-trials/cancer">cancer</a>, synovitis, and sympathetic ophthalmia.

Q: How many individuals have opted to participate in this research project?

As of October 31st, 2022, this study is no longer accepting new participants. Initially posted on June 15th 2017, it has since been discontinued. However, there are presently 1649 <a href="https://www.withpower.com/clinical-trials/mds">MDS</a>-related clinical trial and 1331 Stem Cell <a href="https://www.withpower.com/clinical-trials/transplant">Transplant</a> studies actively searching for applicants.

Q: Is there an age limit for eligibility in this trial?

The requirements for inclusion in this study stipulate that the minimum age of participants is 29 days, while the maximum eligible age is 21 years.

Q: Are enrollment slots for this research study still available?

The details on clinicaltrials.gov indicates that no additional patients are being recruited for this particular study, which was initially posted in June 2017 and last updated in October 2022. Fortunately, there are 2980 other trials actively enrolling participants at the current time.

Q: What risks can be associated with Stem Cell Transplantation?

Our company has given stem cell <a href="https://www.withpower.com/clinical-trials/transplant">transplant</a> a safety rating of 2 out of 3, as it is still in the Phase 2 trial stage and there is evidence to suggest that it is safe but no data yet indicating its efficacy.

Phase 2

Waitlist Available

Led By Jeffrey E. Rubnitz, MD, PhD

Research Sponsored by St. Jude Children's Research Hospital

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age > 28 days and < 22 years at time of study entry inclusive

Patients must have one of the following diagnoses: Acute myeloid leukemia fulfilling the criteria of the WHO Classification, or >5% but < 20% marrow myeloblasts and evidence of a clonal de novo AML genetic abnormality [e.g., t(8;21), inv(16), t(9;11)], or Myeloid sarcoma with confirmation of myeloid differentiation, or High grade myelodysplastic syndrome with greater than 5% blasts, or Patients with treatment related myeloid neoplasms with cumulative anthracycline dose not exceeding 230 mg/m2 doxorubicin equivalents

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from diagnosis to completion of five days of single agent dmti (up to 2 weeks after start of therapy)

Awards & highlights

Study Summary

This trial is studying how well giving azacitidine or decitabine before standard chemotherapy works in treating patients with acute myeloid leukemia.

Who is the study for?

This trial is for patients under 22 years old with newly diagnosed acute myeloid leukemia or high-grade myelodysplastic syndrome, without prior treatment except possibly one dose of intrathecal therapy and certain emergency treatments. They must not have Down syndrome, certain other leukemias or bone marrow syndromes, uncontrolled infections, or be pregnant.Check my eligibility

What is being tested?

The study tests if using drugs called azacitidine and decitabine (DMTis) before standard chemotherapy can help treat acute myeloid leukemia in young patients. It checks the safety of this approach and whether it affects disease markers and survival rates.See study design

What are the potential side effects?

Possible side effects include reactions to the drugs such as nausea, fatigue, blood count changes leading to increased infection risk, liver issues, and potential allergic reactions. The exact side effects will vary depending on individual patient responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I am between 1 month and 22 years old.

Select...

I have a specific type of blood cancer or condition as per WHO guidelines.

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I am a woman who can have children and I have a negative pregnancy test from the last 2 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from diagnosis to completion of five days of single agent dmti (up to 2 weeks after start of therapy)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from diagnosis to completion of five days of single agent dmti (up to 2 weeks after start of therapy)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from diagnosis to completion of five days of single agent dmti (up to 2 weeks after start of therapy) for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Change in genome-wide methylation burden of leukemia cells from diagnosis to after five days of single agent DMTi

Cox model hazard ratio for association of event-free survival with genome-wide methylation burden

Proportion of evaluable patients who tolerate five days of single agent DMTi before a standard chemotherapy combination

Secondary outcome measures

Kaplan-Meier estimate of event-free survival

Kaplan-Meier estimate of overall survival

Proportion of MRD-evaluable subjects with detectable minimal residual disease after receiving five days of a single agent DMTi followed by araC+daunorubicin+etoposide.

Side effects data

From 2010 Phase 2 & 3 trial • 135 Patients • NCT00176904

40%

Death

Auto recovery

Primary graft failure

100%

80%

60%

40%

20%

Study treatment Arm

Patients Treated With Stem Cell Transplant

Trial Design

16Treatment groups

Experimental Treatment

Part 2 Dose Expansion with DAC - Intermediate-Risk Patients are randomized to receive 5 days of single agent decitabine as part of Inductions I & II and intermediate-risk Intensifications I, II and III. Sorafenib will be given to patients with FLT3-ITD. For these patients, DAC will be limited to the first two courses of Induction chemotherapy. They will not receive DAC with Intensification therapy. Interventions: decitabine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, sorafenib, mitoxantrone, erwinia asparaginase, asparaginase erwinia chrysanthemi (recombinant)-rywn, ITMHA.

Group II: DAC+ADE | DAC+FLAG+Ida+Sor | MA+Sor | Asp+AraC+SorExperimental Treatment13 Interventions

Part 1 Tolerability with DAC - High Risk (with donor) Patients are randomized to receive 5 days of single agent decitabine as part of Inductions I & II and then receive high-risk Intensifications I or high risk intensification III without decitabine. Patients will proceed to stem cell transplant. Sorafenib is limited to patients with FLT3-ITD+/NUP98-NSD1+ or FLT3-ITD+/WT1mut somatic mutations. Interventions: decitabine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, mitoxantrone, sorafenib, ITMHA, erwinia asparaginase, stem cell transplant

Group III: DAC+ADE | DAC+FLAG+Ida+Sor | DAC+AE+Sor|DAC+MA+SorExperimental Treatment11 Interventions

Part 2 Dose Expansion with DAC - Low Risk Patients are randomized to receive 5 days of single agent decitabine as part of Inductions I & II and low-risk Intensifications I & II. Sorafenib will be given to patients with FLT3-ITD. For these patients, DAC will be limited to the first two courses of Induction chemotherapy. They will not receive DAC with Intensification therapy. Interventions: decitabine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, sorafenib, mitoxantrone, ITMHA.

Group IV: DAC+ADE | DAC+FLAG+Ida+Sor | AE | MA+Sor | Asp+AraC+SorExperimental Treatment12 Interventions

Part 1 Tolerability with DAC - High Risk (no donor) Patients are randomized to receive 5 days of single agent decitabine as part of Inductions I & II and then receive high-risk Intensifications I, II & III without decitabine. Sorafenib is limited to patients with FLT3-ITD+/NUP98-NSD1+ or FLT3-ITD+/WT1mut somatic mutations. Interventions: decitabine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, sorafenib, mitoxantrone, erwinia asparaginase, ITMHA.

Group V: DAC+ADE | DAC+FLAG+Ida | AE | MA | Asp+AraCExperimental Treatment11 Interventions

Part 1 Tolerability with DAC - Intermediate Risk Patients are randomized to receive 5 days of single agent decitabine as part of Inductions I & II and then receive intermediate-risk Intensifications I, II & III without decitabine. Interventions: decitabine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, mitoxantrone, erwinia asparaginase, ITMHA.

Group VI: DAC+ADE | DAC+FLAG+Ida | AE | MAExperimental Treatment10 Interventions

Part 1 Tolerability with DAC - Low Risk Patients are randomized to receive 5 days of single agent decitabine as part of Inductions I & II and then receive low-risk Intensifications I & II without decitabine. Interventions: decitabine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, mitoxantrone, ITMHA.

Group VII: DAC |+ADE|+FLAG+Ida+Sor|+MA+Sor|+Asp+AraC+SorExperimental Treatment13 Interventions

Part 2 Dose Expansion with DAC - High Risk (with donor) Patients are randomized to receive 5 days of single agent decitabine as part of Inductions I & II and high-risk Intensifications I or high risk intensification III. Patients will proceed to stem cell transplant. Sorafenib will be given to patients with FLT3-ITD. For these patients, DAC will be limited to the first two courses of Induction chemotherapy. They will not receive DAC with Intensification therapy. Interventions: decitabine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, mitoxantrone, sorafenib, ITMHA, erwinia asparaginase, stem cell transplant

Part 2 Dose Expansion with DAC - High Risk (no donor) Patients are randomized to receive 5 days of single agent decitabine as part of Inductions I & II and high-risk Intensifications I, II and III. Sorafenib will be given to patients with FLT3-ITD. For these patients, DAC will be limited to the first two courses of Induction chemotherapy. They will not receive DAC with Intensification therapy. Interventions: decitabine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, sorafenib, mitoxantrone, erwinia asparaginase, asparaginase erwinia chrysanthemi (recombinant)-rywn, ITMHA.

Part 2 Dose Expansion with AZA - Intermediate-Risk Patients are randomized to receive 5 days of single agent azacitidine as part of Inductions I & II and intermediate-risk Intensification I, II, and III. Sorafenib will be given to patients with FLT3-ITD. For these patients, AZA will be limited to the first two courses of Induction chemotherapy. They will not receive AZA with Intensification therapy. Interventions: azacitidine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, sorafenib, mitoxantrone, erwinia asparaginase, asparaginase erwinia chrysanthemi (recombinant)-rywn, ITMHA.

Group X: AZA+ADE | AZA+FLAG-Ida+Sor | AE | MA+Sor | Asp+AraC+SorExperimental Treatment12 Interventions

Part 1 Tolerability with AZA - High Risk (no donor) Patients are randomized to receive 5 days of single agent azacitidine as part of Induction I & II and high-risk intensifications I, II & III without azacitidine. Sorafenib is limited to patients with FLT3-ITD+/NUP98-NSD1+ or FLT3-ITD+/WT1mut somatic mutations. Interventions: azacitidine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, sorafenib, mitoxantrone, erwinia asparaginase, ITMHA.

Group XI: AZA+ADE | AZA+FLAG+Ida+Sor | MA+Sor | Asp+AraC+SorExperimental Treatment13 Interventions

Part 1 Tolerability with AZA- High Risk (with donor) Patients are randomized to receive 5 days of single agent azacitidine as part of Induction I Induction II and high-risk Intensifications I or high risk intensification III without azacitidine. Patients will proceed to stem cell transplant. Sorafenib is limited to patients with FLT3-ITD+/NUP98-NSD1+ or FLT3-ITD+/WT1mut somatic mutations. Interventions: azacitidine cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, mitoxantrone, sorafenib, ITMHA, erwinia asparaginase, stem cell transplant.

Group XII: AZA+ADE | AZA+FLAG+Ida+Sor | AZA+AE+Sor | AZA+MA+SorExperimental Treatment11 Interventions

Part 2 Dose Expansion with AZA - Low Risk Patients are randomized to receive 5 days of single agent azacitidine as part of Inductions I & II and low- risk Intensifications I & II. Sorafenib will be given to patients with FLT3-ITD. For these patients, AZA will be limited to the first two courses of Induction chemotherapy. They will not receive AZA with Intensification therapy. Interventions: azacitidine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, sorafenib, mitoxantrone, ITMHA.

Group XIII: AZA+ADE | AZA+FLAG+Ida | AE | MA | Asp+AraCExperimental Treatment11 Interventions

Part 1 Tolerability with AZA - Intermediate Risk Patients are randomized to receive 5 days of single agent azacitidine as part of Inductions I & II and then receive intermediate risk Intensifications I, II & III without azacitidine. Interventions: azacitidine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, mitoxantrone, erwinia asparaginase, ITMHA,

Group XIV: AZA+ADE | AZA+FLAG+Ida | AE | MAExperimental Treatment10 Interventions

Part 1 Tolerability with AZA - Low Risk Patients are randomized to receive 5 days of single agent azacitidine as part of Inductions I & II and then receive low-risk intensifications I & II without azacitidine. Interventions: azacitidine, cytarabine, daunorubicin, etoposide,dexrazoxane, fludarabine, idarubicin, G-CSF, mitoxantrone., ITMHA.

Group XV: AZA |+ADE|+FLAG+Ida+Sor|+MA+Sor|+Asp+AraC+SorExperimental Treatment13 Interventions

Part 2 Dose Expansion with AZA - High Risk (with donor) Patients are randomized to receive 5 days of single agent azacitidine as part of Inductions I & II and high-risk Intensification I or high risk intensification III. Patients will proceed to stem cell transplant. Sorafenib will be given to patients with FLT3-ITD. For these patients, AZA will be limited to the first two courses of Induction chemotherapy. They will not receive AZA with Intensification therapy. Interventions: azacitidine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G-CSF, mitoxantrone, sorafenib, ITMHA, erwinia asparaginase, stem cell transplant.

Part 2 Dose Expansion with AZA - High Risk (no donor) Patients are randomized to receive 5 days of single agent azacitidine as part of Inductions I & II and high-risk Intensifications I, II and III. Sorafenib will be given to patients with FLT3-ITD. For these patients, AZA will be limited to the first two courses of Induction chemotherapy. They will not receive AZA with Intensification therapy. Interventions: azacitidine, cytarabine, daunorubicin, etoposide, dexrazoxane, fludarabine, idarubicin, G- CSF, sorafenib, mitoxantrone, erwinia asparaginase, asparaginase erwinia chrysanthemi (recombinant)-rywn, ITMHA.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Decitabine

2004

Completed Phase 3

~1680

G-CSF

2014

Completed Phase 4

~1610

Cytarabine

2016

Completed Phase 3

~3310

Daunorubicin

2013

Completed Phase 4

~4940

Sorafenib

2014

Completed Phase 3

~1670

Etoposide

2010

Completed Phase 3

~2440

Erwinia asparaginase

2012

Completed Phase 2

~30

Idarubicin

2014

Completed Phase 4

~4330

Fludarabine

2012

Completed Phase 3

~1100

Mitoxantrone

2008

Completed Phase 3

~1510

Dexrazoxane

2016

Completed Phase 2

~80

Stem Cell Transplant

2007

Completed Phase 3

~1350

Azacitidine

2012

Completed Phase 3

~1440

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

St. Jude Children's Research HospitalLead Sponsor

427 Previous Clinical Trials

5,306,371 Total Patients Enrolled

Jeffrey E. Rubnitz, MD, PhDPrincipal InvestigatorSt. Jude Children's Research Hospital

5 Previous Clinical Trials

202 Total Patients Enrolled

Media Library

Azacitidine (DNA Methyltransferase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT03164057 — Phase 2

Acute Myeloid Leukemia Research Study Groups: DAC|+ADE | +FLAG+Ida+Sor | +AE+Sor | +MA+Sor | +Asp+AraC+Sor, AZA+ADE | AZA+FLAG+Ida+Sor | MA+Sor | Asp+AraC+Sor, AZA+ADE | AZA+FLAG+Ida | AE | MA, DAC+ADE | DAC+FLAG+Ida | AE | MA, AZA+ADE | AZA+FLAG+Ida+Sor | AZA+AE+Sor | AZA+MA+Sor, DAC+ADE | DAC+FLAG+Ida+Sor | DAC+AE+Sor|DAC+MA+Sor, AZA+ADE | AZA+FLAG+Ida | AE | MA | Asp+AraC, DAC+ADE | DAC+FLAG+Ida | AE | MA | Asp+AraC, AZA| +ADE | +FLAG+Ida+Sor| +AE+Sor| +MA+Sor| +Asp+AraC+Sor, AZA+ADE | AZA+FLAG-Ida+Sor | AE | MA+Sor | Asp+AraC+Sor, DAC+ADE | DAC+FLAG+Ida+Sor | AE | MA+Sor | Asp+AraC+Sor, AZA | + ADE | +FLAG+Ida+Sor| +AE+Sor | +MA+Sor | +Asp+AraC+Sor, DAC |+ADE |+FLAG+Ida+Sor |+AE+Sor|+MA+Sor|+Asp+AraC+Sor, DAC+ADE | DAC+FLAG+Ida+Sor | MA+Sor | Asp+AraC+Sor, DAC |+ADE|+FLAG+Ida+Sor|+MA+Sor|+Asp+AraC+Sor, AZA |+ADE|+FLAG+Ida+Sor|+MA+Sor|+Asp+AraC+Sor

Acute Myeloid Leukemia Clinical Trial 2023: Azacitidine Highlights & Side Effects. Trial Name: NCT03164057 — Phase 2

Azacitidine (DNA Methyltransferase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03164057 — Phase 2

Frequently Asked Questions

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

Is it possible to become a participant in this research endeavor?

"To gain admittance to this study, individuals must have myelodysplastic syndromes and be between the ages of 29 days and 21 years. In total, 206 patients will be accepted."

Answered by AI

Has Stem Cell Transplant been subject to prior investigation through clinical trials?

"In 1995, the National Institutes of Health Clinical Center first conducted a trial on Stem Cell Transplant. In total, there have been 2,648 completed trials since then and 1,331 are currently ongoing--most notably in Memphis, Tennessee."

Answered by AI

What is the geographic distribution of this research program?

"St. Jude Children's Research Hospital in Memphis, Tennessee, Children's Hospital of Orange County in California and the Children's Hospital of Central California are a few from 11 different clinical trial sites that have been selected to accept patients for this study."

Answered by AI

What medical afflictions are alleviated through Stem Cell Transplant?

"Stem Cell Transplant is a viable intervention for individuals suffering from merkel cell cancer, synovitis, and sympathetic ophthalmia."

Answered by AI

How many individuals have opted to participate in this research project?

"As of October 31st, 2022, this study is no longer accepting new participants. Initially posted on June 15th 2017, it has since been discontinued. However, there are presently 1649 MDS-related clinical trial and 1331 Stem Cell Transplant studies actively searching for applicants."

Answered by AI

Is there an age limit for eligibility in this trial?

"The requirements for inclusion in this study stipulate that the minimum age of participants is 29 days, while the maximum eligible age is 21 years."

Answered by AI

Are enrollment slots for this research study still available?

"The details on clinicaltrials.gov indicates that no additional patients are being recruited for this particular study, which was initially posted in June 2017 and last updated in October 2022. Fortunately, there are 2980 other trials actively enrolling participants at the current time."

Answered by AI

What risks can be associated with Stem Cell Transplantation?

"Our company has given stem cell transplant a safety rating of 2 out of 3, as it is still in the Phase 2 trial stage and there is evidence to suggest that it is safe but no data yet indicating its efficacy."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia

2017. "A Trial of Epigenetic Priming in Patients With Newly Diagnosed Acute Myeloid Leukemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03164057.

All > Acute Myeloid Leukemia > Nexavar