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Leukemia

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Leukemia, Lymphoid

Leukemia, Myeloid

Lymphoma

Leukemia, Myeloid, Acute

Acute Disease

Neoplasms by Histologic Type

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Immunoproliferative Disorders

Disease Attributes

Myeloid Leukemia

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Micronutrients

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Vitamins

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Etoposide

Cytarabine

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Etoposide phosphate

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Immunosuppressive Agents

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Tyrosine Kinase Inhibitors

Protein Kinase Inhibitors

Folinic Acid

Folate

Vitamin B9

Tyrosine

The starting dose is Dose Level 1 at 25 mg/m2/day. Dose escalation will proceed from level 1 to 2 to 3, and so on, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Methotrexate will be given to patients with ALL on Day 0, dose assigned by age.

The starting dose is Dose Level 1 at 25 mg/m2/day. Dose escalation will proceed from level 1 to 2 to 3, and so on, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT cytarabine will be given to patients with AML on Day 0, dose assigned by age.

Dose escalation will proceed from level 1 to level 2 for AC220 at 40mg/m2/day, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Methotrexate will be given to patients with ALL on Day 0, dose assigned by age.

Dose escalation will proceed from level 2 to 3 for AC220 at 60mg/m2/day, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Methotrexate will be given to patients with ALL on Day 0, dose assigned by age. If toxicity at Dose Level 3 would allow further escalation, but demonstrated sufficient AC220 activity, no further dose escalation will be required.

If the study dose of 60 mg/m2/day at Dose Level 3 is well tolerated but does not show sufficient AC220 activity, the study may proceed to Dose Level 4 at 90 mg/m2/day. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Methotrexate will be given to patients with ALL on Day 0, dose assigned by age. If toxicity at Dose Level 4 would allow further escalation, but demonstrated sufficient AC220 activity, no further dose escalation will be required.

If the study dose of 90 mg/m2/day at Dose Level 4 is well tolerated but does not show sufficient AC220 activity, the study may proceed to Dose Level 5 at 130 mg/m2/day. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Methotrexate will be given to patients with ALL on Day 0, dose assigned by age. Dose Level 5 is the highest dose for this study.

Dose escalation will proceed from level 1 to level 2 for AC220 at 40mg/m2/day, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT cytarabine will be given to patients with AML on Day 0, dose assigned by age.

Dose escalation will proceed from level 2 to 3 for AC220 at 60mg/m2/day, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT cytarabine will be given to patients with AML on Day 0, dose assigned by age. If toxicity at Dose Level 3 would allow further escalation, but demonstrated sufficient AC220 activity, no further dose escalation will be required.

If the study dose of 60 mg/m2/day at Dose Level 3 is well tolerated but does not show sufficient AC220 activity, the study may proceed to Dose Level 4 at 90 mg/m2/day. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT cytarabine will be given to patients with AML on Day 0, dose assigned by age. If toxicity at Dose Level 4 would allow further escalation, but demonstrated sufficient AC220 activity, no further dose escalation will be required.

If the study dose of 90 mg/m2/day at Dose Level 4 is well tolerated but does not show sufficient AC220 activity, the study may proceed to Dose Level 5 at 130 mg/m2/day. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT cytarabine will be given to patients with AML on Day 0, dose assigned by age. Dose Level 5 is the highest dose for this study.

Dose assigned at study entry. AC220 will be given orally once daily on days 7-28.

AC220

All patients receive 1000 mg/m2/day IV given every 12 hours on days 1 through 5. Additionally, AML patients and patients with ambiguous leukemia receive cytarabine intrathecally on day "1" of course 1 and 2. Dose defined by age:

* 20 mg for patients age \<1 yr
* 30 mg for patients age 1-1.99 years of age
* 50 mg for patients age 2-2.99 years of age
* 70 mg for patients \>3 years of age

IT methotrexate given intrathecally to patients with ALL on day "0" of course 1 and 2. Dose defined by age

* 6 mg for patients age \< 1yr
* 8 mg for patients age 1-1.99
* 10 mg for patients age 2-2.99
* 12 mg for patients 3-8.99 years of age
* 15 mg for patients \>9 years of age

Todd Cooper, MD

This is a phase I study of the investigational drug AC220 combined with cytarabine and etoposide in pediatric patients with relapsed acute lymphoblastic leukemia (ALL) and acute myelogenous leukemia (AML).

This is a study for pediatric patients with relapsed acute lymphoblastic leukemia (ALL) or acute myelogenous leukemia (AML). Some people diagnosed with leukemia have changes in a receptor located on the surface of white blood cells called FLT3. This is known as a FLT3 mutation. FLT3 plays an important role in the way cells grow and divide. In normal cells, the FLT3 receptor is switched off most of the time and only switches on when it gets a chemical signal from outside. But cells with the FLT3 mutation have the grow signal permanently switched on. This means leukemia cells with the FLT3 mutation are growing and dividing all the time. Doctors have found that people with leukemia cells that carry FLT3 mutations are less likely to go into remission with chemotherapy and have a higher risk of the leukemia coming back after treatment.

This is a study of an investigational drug called AC220. AC220 is considered investigational because it has not been approved in the United States by the Food and Drug Administration (FDA). AC220 is a drug which is able to "turn off" the FLT3 grow signal. AC220 will be given with cytarabine and etoposide to treat the relapsed leukemia. This is a phase I study, which means that the study is being done to find the highest dose of AC220 that can be given safely with the drugs cytarabine and etoposide to children and young adults.

AC220 for Children With Relapsed/Refractory ALL or AML

A Phase I Study of AC220 for Children With Relapsed or Refractory ALL or AML

The incidence of dose limiting toxicity (DLT) will be measured. The maximum tolerated dose will be the highest study dose at which 1 or fewer of six patients experience DLT during cycle 1 of therapy. Plasma inhibitor activity (PIA) will be measured Pre-treatment and on Days 7, 14, 21 and 28 of Course 1. For the MTD to be considered biologically active, we will require that 7 of 9 patients achieve PIA of \> 90% at 3 of 4 trough time points.

Possible outcomes are: Complete Remission (CR), Complete Remission without Platelet Recovery (CRp), complete response with incomplete hematologic recovery (CRi), Stable Disease, Progressive Disease, Induction Death, or Relapse

PIA samples will be collected pre-treatment and on Days 7, 14, 21 and 28 of Course 1.

Ambit Biosciences Corporation

Therapeutic Advances in Childhood Leukemia Consortium

The definitions of AE and SAE do not differ from the clinicaltrials.gov definitions.

The starting dose is Dose Level 1 at 25 mg/m\^2/day. Dose escalation will proceed from level 1 to 2 to 3, and so on, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Methotrexate will be given to patients with ALL on Day 0, dose assigned by age.

ALL Dose Level 1

The starting dose is Dose Level 1 at 25 mg/m\^2/day. Dose escalation will proceed from level 1 to 2 to 3, and so on, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT cytarabine will be given to patients with AML on Day 0, dose assigned by age.

AML Dose Level 1

Dose escalation will proceed from level 1 to level 2 for AC220 at 40mg/m\^2/day, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Methotrexate will be given to patients with ALL on Day 0, dose assigned by age.

ALL Dose Level 2

Dose escalation will proceed from level 1 to level 2 for AC220 at 40mg/m\^2/day, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Cytarabine will be given to patients with AML on Day 0, dose assigned by age.

AML Dose Level 2

Dose escalation will proceed from level 2 to 3 for AC220 at 60mg/m\^2/day, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Methotrexate will be given to patients with ALL on Day 0, dose assigned by age. If toxicity at Dose Level 3 would allow further escalation, but demonstrated sufficient AC220 activity, no further dose escalation will be required.

ALL Dose Level 3

Dose escalation will proceed from level 2 to 3 for AC220 at 60mg/m\^2/day, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Cytarabine will be given to patients with AML on Day 0, dose assigned by age. If toxicity at Dose Level 3 would allow further escalation, but demonstrated sufficient AC220 activity, no further dose escalation will be required.

AML Dose Level 3

ALL AC220 @ 25mg/m2/Day (Dose Level 1)

AML AC220 @ 25mg/m2/Day (Dose Level 1)

ALL AC220 @ 40mg/m2/Day (Dose Level 2)

AML AC220 @ 40mg/m2/Day (Dose Level 2)

ALL AC220 @ 60mg/m2/Day (Dose Level 3)

Dose escalation will proceed from level 2 to 3 for AC220 at 60mg/m\^2/day, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT cytarabine will be given to patients with AML on Day 0, dose assigned by age. If toxicity at Dose Level 3 would allow further escalation, but demonstrated sufficient AC220 activity, no further dose escalation will be required.

AML AC220 @ 60mg/m2/Day (Dose Level 3)

Total

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

# Patients with Prior HSCT

FLT3/ITD+ (FLT3-internal tandem duplication mutation)

No patients with ALL were enrolled into Dose Level 1

Peggy Romano, BA, CCRP

ALL AC220 @ 25mg/m^2/Day (Dose Level 1)

AML AC220 @ 25mg/m^2/Day (Dose Level 1)

ALL AC220 @ 40mg/m^2/Day (Dose Level 2)

AML AC220 @ 40mg/m^2/Day (Dose Level 2)

ALL AC220 @ 60mg/m^2/Day (Dose Level 3)

AML AC220 @ 60mg/m^2/Day (Dose Level 3)

No patients with ALL were enrolled during Dose Level 1.

The Dose of AC220 That is Safe and Biologically Active When Given in Sequential Combination With Ara-C/Etoposide

Patients with a diagnosis of acute lymphocytic leukemia at study entry

Patients With ALL

Patients with a diagnosis of acute myeloid leukemia with internal tandem duplication (ITD) on exon 14 of the FLT3 gene

Patients With AML Having FLT3-WT

Patients with a diagnosis of acute myeloid leukemia having the FLT3 wild-type receptor (FLT3-WT)

Patients With AML Having FLT3-ITD

Five of 22 patients were not evaluable for response per protocol because they were removed from protocol therapy prior to disease assessment without meeting PD criteria

Disease Response

Patients Receiving Protocol Therapy

Nineteen of 22 patients had PIA assessment.

Count of Participants According to Inhibition of FLT3 Phosphorylation

Dose escalation will proceed from level 1 to level 2 for AC220 at 40mg/m\^2/day, assuming the maximum tolerated dose is not exceeded. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT cytarabine will be given to patients with AML on Day 0, dose assigned by age.

If the study dose of 60 mg/m\^2/day at Dose Level 3 is well tolerated but does not show sufficient AC220 activity, the study may proceed to Dose Level 4 at 90 mg/m2/day. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Methotrexate will be given to patients with ALL on Day 0, dose assigned by age. If toxicity at Dose Level 4 would allow further escalation, but demonstrated sufficient AC220 activity, no further dose escalation will be required.

ALL AC220 @ 90mg/m^2/Day (Dose Level 4)

If the study dose of 60 mg/m\^2/day at Dose Level 3 is well tolerated but does not show sufficient AC220 activity, the study may proceed to Dose Level 4 at 90 mg/m\^2/day. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT cytarabine will be given to patients with AML on Day 0, dose assigned by age. If toxicity at Dose Level 4 would allow further escalation, but demonstrated sufficient AC220 activity, no further dose escalation will be required.

AML AC220 @ 90mg/m^2/Day (Dose Level 4)

If the study dose of 90 mg/m\^2/day at Dose Level 4 is well tolerated but does not show sufficient AC220 activity, the study may proceed to Dose Level 5 at 130 mg/m\^2/day. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT Methotrexate will be given to patients with ALL on Day 0, dose assigned by age. Dose Level 5 is the highest dose for this study.

ALL AC220 @ 130mg/m^2/Day (Dose Level 5)

If the study dose of 90 mg/m\^2/day at Dose Level 4 is well tolerated but does not show sufficient AC220 activity, the study may proceed to Dose Level 5 at 130 mg/m\^2/day. Patients will received etoposide and cytarabine on Days 1-5, and AC220 on Day 7 through 28. IT cytarabine will be given to patients with AML on Day 0, dose assigned by age. Dose Level 5 is the highest dose for this study.

AML AC220 @ 130mg/m^2/Day (Dose Level 5)

Overall Study

Hematologic Neoplasms

Hodgkin Disease

Lymphoma, Non-Hodgkin

Hodgkin Lymphoma

Panobinostat

Histone Deacetylase Inhibitors

Patients with ALL and AML will be treated in one arm of the study.

Patients with NHL or HD will be treated in one arm of the study.

Dose will be assigned at study entry. Patients will take panobinostat orally 3 times a week given on a Monday, Wednesday, Friday schedule, every week. One course is 28 days (4 weeks). Patients will get 2 courses and may receive up to 8 courses total.

All patients will receive 70 mg of intrathecal cytarabine on day "0" of course 1. The day "0" dose must be given at least 24 hours prior to initiation of panobinostat. Omit the day "0" dose of intrathecal cytarabine if the patient received intrathecal therapy within 72 hours of treatment.

All patients will receive 70 mg of intrathecal cytarabine on day "29" of course 1-8 in conjunction with their disease evaluation.

Dose will be assigned at study entry. Patients will take panobinostat orally 3 times a week on a Monday, Wednesday, Friday schedule, every other week. Once course is 28 days (4 weeks). Patients will get 2 course and may receive up to 8 courses of therapy.

Julio Barredo, MD

This study is for patients with relapsed or refractory Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Hodgkin's Disease (HD) or Non-Hodgkin's Lymphoma (NHL). Panobinostat is a new drug that is considered investigational because it has not been approved in the United States by the Food and Drug Administration (FDA), or in any other country. Panobinostat is a histone deacetylase inhibitor (HDACi) and interferes with gene expression found in cells causing them to stop growing or die. Panobinostat has been used in several hundred adults who had leukemia, HD, NHL and other solid tumors. Panobinostat has not been given to children.

This is a phase I study. In a phase I study, drugs are tested to the highest dose that can be safely given. Drugs are given at gradually increasing dosages until there are unacceptable side effects. The goal of the Phase I study is to find out the dose of panobinostat that can be safely given to children with relapsed ALL, AML, HD and NHL.

A Study Of Panobinostat In Children With Refractory Hematologic Malignancies

A Phase I Dose Finding Study Of Panobinostat In Children With Refractory Hematologic Malignancies

Novartis

Anti-Inflammatory Agents

Hydrocortisone

Hydrocortisone 17-butyrate 21-propionate

Hydrocortisone acetate

Hydrocortisone hemisuccinate

Fludarabine

Fludarabine phosphate

Azacitidine

Participants with Acute Myeloid Leukemia (AML)

Intervention:

* Azacytidine (Dose Level 1 @ 75 mg/m2/day)
* Fludarabine 30 mg/m2/dose
* Cytarabine 2000 mg/m2/dose
* Intrathecal (IT) Cytarabine

Patients with Acute Lymphocytic Leukemia

Intervention:

* Azacytidine (Dose Level 1 @ 75 mg/m2/day)
* Fludarabine 30 mg/m2/dose
* Cytarabine 2000 mg/m2/dose
* Intrathecal Methotrexate (IT MTX)

Dose assigned at study entry (75 mg/m2/day). Given subcutaneously, once daily on days 1 to 5, for a total of 5 doses.

Azacytidine

30 mg/m2/dose, intravenous infusion over 30 minutes, once daily, on days 6 to 10, total 5 doses

2000 mg/m2/dose intravenous infusion over 3 hours, starting 4 hours after the beginning of fludarabine, once daily, on days 6 to 10, total 5 doses.

Intrathecally to AML patients on day 1 of course 1 and 2.

* Omit on day 1 of course 1 if patient received IT therapy within 7 days prior to study enrollment
* IT therapy may be given during the end of course 1 disease evaluation and repeated every 7 days
* For patients with CNS disease, IT cytarabine can be given weekly until the CSF is clear. Two additional doses of IT cytarabine should be given weekly after the initial CSF clearing. It is permitted to change to intrathecal triple therapy (ITT) if persistent blasts are present in the CSF based on the treating physician's clinical judgment. Cytarabine dose defined by age:
* 30 mg for patients age 1-1.99
* 50 mg for patients age 2-2.99
* 70 mg for patients \>3 years of age

ITT Dosing:

Age (yrs) - Dose Methotrexate (MTX), Hydrocortisone (HC), Cytarabine (ARAC):

1. - 1.99 MTX: 8 mg, HC: 15 mg, ARAC: 30 mg
2. - 2.99 MTX: 10 mg, HC: 25 mg, ARAC: 50 mg

   * 3 - MTX: 12 mg, HC: 35 mg, ARAC: 70 mg

Intrathecal (IT) Cytarabine

* Intrathecally to patients with ALL on day 1 of course 1 and 2.
* Omit IT MTX on Day 1 of course 1 if patient received IT therapy within 7 days prior to study enrollment
* IT therapy may be given during the end of course 1 disease evaluation and repeated every 7 days
* For patients with CNS 2 or 3 disease, IT MTX can be given weekly until the CSF is clear. Two additional doses of IT MTX should be given weekly after the initial clearing of the CSF. It is permitted to change to ITT if persistent blasts are present in the CSF. Methotrexate dose defined by age
* 8 mg for patients age 1-1.99
* 10 mg for patients age 2-2.99
* 12 mg for patients 3-8.99 years of age
* 15 mg for patients \>9 years of age

Triple IT Therapy Dosing:

Age (yrs): Dose Methotrexate (MTX), Hydrocortisone (HC), Cytarabine (ARAC):

1. - 1.99 MTX: 8 mg, HC: 8 mg, ARAC: 16 mg
2. - 2.99 MTX: 10 mg, HC: 10 mg, ARAC: 20 mg
3. - 8.99 MTX: 12 mg, HC: 12 mg, ARAC: 24 mg

   * 9 MTX: 15 mg, HC: 15 mg, ARAC: 30 mg

Intrathecal Methotrexate (IT MTX)

Weili Sun, MD, PhD

This is a Phase I study with a conditional cohort expansion phase to evaluate the feasibility of, and to obtain preliminary efficacy data about, pretreatment with Azacytidine (AZA) for 5 days followed by fludarabine/cytarabine chemotherapy regimen in pediatric acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) patients who are refractory to primary treatment or who relapsed.

Growing evidence indicates that aberrant DNA hypermethylation is associated with leukemia development, drug resistance, and relapse. It has been shown that pretreating leukemia cells with AZA or decitabine could partially reverse the aberrant DNA methylation, restore the expression of tumor suppressor gene important for apoptosis, and sensitize cells to subsequent killing by cytotoxic agent. Since cytarabine and decitabine share the same mechanisms of resistance, we use AZA to test the novel epigenetic "priming" approach. This is a phase I clinical study with expansion phase, using hypomethylating agent, 5-azacytidine (AZA), in sequential with chemotherapy to evaluate whether epigenetic "priming" can reverse aberrant DNA methylation, overcome drug resistance, and increase response in relapsed/refractory AML.

The chemotherapy regimen to be used in this study is fludarabine and cytarabine. This regimen has substantial activity in leukemia and has been widely used in treating pediatric patients with relapsed/refractory AML and ALL in the past several decades. In BFM relapsed AML 2001/01 study, FLAG (fludarabine, cytarabine and G-CSF) chemotherapy regimen showed significant activity in AML with 4 year OS around 36%. Since the use of G-CSF in conjunction with fludarabine/cytarabine didn't improve the overall survival of patient in a randomized trial, only fludarabine and cytarabine will be used in this study to decrease the incidence of leukocytosis related complications. This regimen is very similar to the chemotherapy regimen proposed for the next relapsed AML trial within the Children's Oncology Group (COG). If this trial proves to be safe and active, it will provide the foundation and smooth transition to larger statistically powered nationwide phase II clinical trials by COG.

A Phase I Study of 5-Azacytidine in Combination With Chemotherapy for Children With Relapsed or Refractory ALL or AML

To evaluate the side effects of giving Azacytidine before and during chemotherapy using the standard drugs Fludarabine, Cytarabine, IT Cytarabine (AML patients) and IT methotrexate (ALL patients)

CR is defined as a bone marrow with \< 5% blast by morphology, no evidence of extramedullary disease, and recovery of peripheral counts (ANC ≥ 1000/μl and platelet counts ≥ 100,000/μl). CR with incomplete count recovery (CRi) was defined as CR without recovery of ANC and/or platelets. Partial response (PR) was defined as complete disappearance of circulating blasts and a decrease of at least 50% of blasts in the bone marrow. Progressive disease (PD) was defined as an increase of at least 25% in the absolute number of bone marrow or circulating blasts, development of new sites of extramedullary disease, or other laboratory or clinical evidence of progression of disease. Stable disease (SD) referred to patient who did not satisfy the criteria for either CR, CRi, PR or PD.

Gateway for Cancer Research

Any patients in the AML arm who are evaluable for toxicity. Any patient who experiences a DLT after receiving at least one dose of AZA on study will be considered evaluable for toxicity of AZA. Patients who do not experience a DLT must receive at least 80% of the prescribed course of AZA in the first cycle (i.e., must receive at least 4 of the planned 5 doses of AZA between days 1 to 5) to be evaluable for toxicity of AZA. Patients not evaluable for toxicity of AZA will be replaced.

AML Cohort: 75 mg/m2/Day

Any patients in the ALL arm who are evaluable for toxicity. Any patient who experiences a DLT after receiving at least one dose of AZA on study will be considered evaluable for toxicity of AZA. Patients who do not experience a DLT must receive at least 80% of the prescribed course of AZA in the first cycle (i.e., must receive at least 4 of the planned 5 doses of AZA between days 1 to 5) to be evaluable for toxicity of AZA. Patients not evaluable for toxicity of AZA will be replaced.

ALL Cohort: 75 mg/m2/Day

This arm is comprised of the AML and ALL participants that completed Dose Level 1 75 mg/m2/day

Dose Level 1 75 mg/m2/Day

CNS Negative (better outcome): absence of blasts on a cytospin preparation in cerebral spinal fluid (CSF) or clinical signs of CNS leukemia

CNS Positive (worse outcome): presence of any number of blasts on a cytospin preparation in CSF or clinical signs of CNS leukemia

Central Nervous System (CNS) Status

Measurement of bone marrow response

M2 (better outcome): Marrow has between 5% and 25% leukemic blasts M3 (worse outcome): Marrow has more than 25% leukemic blasts

Bone Marrow Status (at study entry)

# Prior treatment regimens

Prior Hematopoetic Stem Cell Transplantation (HSCT)

White blood cell (WBC) Count

Peripheral blasts

Bone marrow blasts

Number that completed therapy and were analyzed

Roy Leong, BA, CCRP

# of patients with DLT

# of patients without DLT

# of patients not evaluable

Evaluable Participants who received azacytidine @ 75 mg/m2/day (Dose Level 1)

Dose 75 mg/m2/Day Azacytidine Diagnosed With AML

Evaluable ALL participants who received azacytidine @ 75 mg/m2/day (Dose Level 1)

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
AC220 for Children With Relapsed/Refractory ALL or AML	NCT01411267	Lymphoblastic L... Myelogenous Leu...	AC220 Cytarabine Etoposide Methotrexate	1 Month - 21 Years	Therapeutic Advances in Childhood Leukemia Consortium
A Study Of Panobinostat In Children With Refractory Hematologic Malignancies	NCT01321346	Lymphoblastic L... Myelogenous Leu... Hodgkin's Disea... Non-Hodgkin's L...	Panobinostat Cytarabine Panobinostat	8 Years - 21 Years	Therapeutic Advances in Childhood Leukemia Consortium
AC220 for Children With Relapsed/Refractory ALL or AML	NCT01411267	Lymphoblastic L... Myelogenous Leu...	AC220 Cytarabine Etoposide Methotrexate	1 Month - 21 Years	Therapeutic Advances in Childhood Leukemia Consortium
AC220 for Children With Relapsed/Refractory ALL or AML	NCT01411267	Lymphoblastic L... Myelogenous Leu...	AC220 Cytarabine Etoposide Methotrexate	1 Month - 21 Years	Therapeutic Advances in Childhood Leukemia Consortium
A Phase I Study of 5-Azacytidine in Combination With Chemotherapy for Children With Relapsed or Refractory ALL or AML	NCT01861002	Lymphoblastic L... Myelogenous Leu...	Azacytidine Fludarabine Cytarabine Intrathecal (IT... Intrathecal Met...	1 Year - 21 Years	Therapeutic Advances in Childhood Leukemia Consortium

Spots Global Cancer Trial Database for myelogenous leukemia, acute, childhood