An Open-Label Feasibility Study to Assess the Safety and Pharmacokinetics of Enasidenib in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (R/R-AML) With an Isocitrate Dehydrogenase-2 (IDH2) Mutation
Phase: PHASE2
Conditions: Recurrent Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
Sponsor: Children's Oncology Group

Trial Summary Report 
Date: 05/31/2023 
Record Verification Date: 03/17/2023 
 
 
 
Trial Identification 
Trial Category 
Complete 
Trial Type 
Interventional 
NCI Trial Identifier 
NCI-2019-07902 
Other Trial Identifiers 
    Lead Organization Identifier 
ADVL18P1 
    ClinicalTrials.gov Identifier 
NCT04203316 
    DCP Identifier 
No Data Available 
    CTEP Identifier 
ADVL18P1 
    CCR Identifier 
No Data Available 
Amendment Number 
4 
Amendment Date 
12/15/2022 
 
 
 
General Trial Details 
Type 
Interventional 
Official Title 
An Open-Label Feasibility Study to Assess the Safety and 
Pharmacokinetics of Enasidenib in Pediatric Patients with 
Relapsed/Refractory Acute Myeloid Leukemia (R/R-AML) with an 
Isocitrate Dehydrogenase-2 (IDH2) Mutation 
Brief Title 
Enasidenib for the Treatment of Relapsed or Refractory Acute 
Myeloid Leukemia Patients with an IDH2 Mutation 
Acronym 
No Data Available 
Brief Summary 
This trial studies the side effects of enasidenib and to see how well it works in treating patients 
with acute myeloid leukemia that has come back after treatment (relapsed) or has been difficult to 
treat with chemotherapy (refractory). Patients must also have a specific genetic change, also 
called a mutation, in a protein called IDH2. Enasidenib may stop the growth of cancer cells by 
blocking the mutated IDH2 protein, which is needed for cell growth. 
Detailed Description 
PRIMARY OBJECTIVES: 
I. To determine the safety of treatment with enasidenib mesylate (enasidenib) administered at 
continuous daily oral dosing for a 28-day cycle up to 12 cycles in pediatric patients with 
IDH2-mutant relapsed/refractory (R/R)-acute myeloid leukemia (AML).  
II. To characterize the plasma pharmacokinetic (PK) profile of enasidenib in pediatric patients with 
IDH2-mutant R/R-AML. 
 
SECONDARY OBJECTIVES: 
I. To investigate the pharmacodynamic (PD) relationship of oncogenic metabolite 

2-hydroxyglutarate (2-HG) to enasidenib treatment in pediatric patients with IDH2-mutant 
R/R-AML.  
II. To describe the clinical activity of enasidenib in pediatric patients with IDH2-mutant R/R-AML. 
 
OUTLINE: 
Patients receive enasidenib orally (PO) once daily (QD) on days 1-28. Treatment repeats every 28 
days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients 
also undergo bone marrow aspiration and/or biopsy and collection of blood on study. 
 
After completion of study treatment, patients are followed up at 30 days, then periodically up to 1 
year. 
Sponsor 
Children's Oncology Group 
Lead Organization 
Children's Oncology Group 
Principal Investigator 
Zarnegar-Lumley, Sara 
Responsible Party 
Sponsor 
Overall Official 
Zarnegar-Lumley, Sara (Principal Investigator), Children's 
Oncology Group 
 
 
 
Status/Dates 
Current Trial Status 
Active as of 01/30/2023 
Trial Start Date 
03/30/2020-Actual 
Primary Completion Date 
12/31/2030-Anticipated 
Trial Completion Date 
12/31/2030-Anticipated 
 
 
 
Regulatory Information 
Studies a U.S. FDA-regulated 
Drug Product 
No Data Available 
Studies a U.S. FDA-regulated 
Device Product 
No Data Available 
Product Exported from the U.S No Data Available 
FDA Regulated Intervention? 
Yes 
Section 801? 
Yes 
DMC Appointed? 
No 
IND/IDE Study? 
Yes 
 
 
 
Human Subject Safety 
Board Approval Status 
Submitted, approved 
Board Approval Number 
12/15/2022 
 
 
 

IND/IDE 
Type 
Grantor 
Number 
Holder Type Holder 
Availability of 
Expanded 
Access 
Expanded 
Access 
Record 
IND 
CDER 
146610 
Organization  
Unknown 
 
 
 
 
NIH Grants 
Funding Mechanism 
NIH Institution Code 
Serial Number 
NCI Division/Program 
Code 
U10 
CA 
180886 
CTEP 
 
 
 
Data Table 4 Information 
Funding Category 
National 
Funding Sponsor/Source 
Children's Oncology Group 
 
Anatomic Site Code 
Myeloid and Monocytic Leukemia 
 
 
 
Collaborators 
Name 
Role 
National Cancer Institute 
Funding Source 
 
 
 
Disease/Condition 
Name 
Recurrent Acute Myeloid Leukemia 
Refractory Acute Myeloid Leukemia 
 
 
 
Trial Design 
Type 
Interventional 
Primary Purpose 
Treatment 
Pragmatic Trial 
No 
Phase 
II 
Pilot Study? 
No 
Interventional Study Model 
Single Group 
Model Description 
No Data Available 
Number of Arms 
1 
Masking 
No Masking 
Masking Description 
No Data Available 

Allocation 
NA 
Target Enrollment 
10 
 
 
 
Eligibility Criteria 
Accepts Healthy Volunteers? 
No 
Sex 
All 
Minimum Age 
24 Months 
Maximum Age 
21 Years 
 
Inclusion Criteria 
• Patients must be >= 24 months and < 21 years of age at the time of study enrollment 
• Patient must have AML with an IDH2 mutation identified from a peripheral blood or bone marrow 
sample at the time of diagnosis and/or relapsed/refractory disease 
• Patient must have bone marrow assessment (aspiration or biopsy) with > 5% leukemic blasts by 
morphology and/or flow cytometry in at least one of the following clinical scenarios: 
* Second or greater relapse after chemotherapy or hematopoietic stem cell transplant (HSCT) 
* Refractory after >= 2 attempts at induction therapy 
• Relapsed patients 
* Must not have received prior re-induction therapy for this relapse 
* Each block of chemotherapy (i.e., cytarabine, daunorubicin and etoposide [ADE], cytarabine 
and mitoxantrone [MA]) is a separate re-induction attempt 
* Donor lymphocyte infusion (DLI) is considered a re-induction attempt 
• Refractory patients 
* Each attempt at induction therapy may include up to two chemotherapy courses 
• Karnofsky >= 50% for patients > 16 years of age and Lansky >= 50 for patients =< 16 years of 
age. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will 
be considered ambulatory for the purpose of assessing the performance score 
• Patient’s current disease state must be one for which there is no known curative therapy or 
therapy proven to prolong survival with an acceptable quality of life 
• Evaluation of cerebrospinal fluid (CSF) is only required if there is a clinical suspicion of central 
nervous system (CNS) involvement by leukemia during eligibility screening. Should a patient be 
found to have CNS2 or CNS3 status by CSF prior to eligibility screening, patient may receive 
intrathecal chemotherapy > 72 hours prior to starting study drug. CNS1 status must be 
established before starting study drug 
• Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy 
and must meet the following minimum duration from prior anti-cancer directed therapy prior to 
enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood 
count criteria, the patient is considered to have recovered adequately 
* Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive. The 
duration of this interval must be discussed with the study chair and the study-assigned research 
coordinator prior to enrollment 
** >= 14 days must have elapsed after the completion of other cytotoxic therapy with the 
exception of hydroxyurea. Additionally, patients must have fully recovered from all acute toxic 
effects of prior therapy. NOTE: Cytoreduction with hydroxyurea must be discontinued >= 24 
hours prior to the start of protocol therapy 
** Intrathecal chemotherapy must be completed >= 72 hours prior to the start of the first cycle of 
treatment 
* Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced 
platelet or absolute neutrophil count [ANC] counts): >= 7 days after the last dose of agent. The 
duration of this interval must be discussed with the study chair and the study-assigned research 
coordinator prior to enrollment 

* Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity 
related to prior antibody therapy must be recovered to grade =< 1 
* Corticosteroids: If used to modify immune adverse events related to prior therapy, >= 14 days 
must have elapsed since last dose of corticosteroid 
* Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor 
(e.g., pegfilgrastim) or 7 days for short-acting growth factor. For agents that have known adverse 
events occurring beyond 7 days after administration, this period must be extended beyond the 
time during which adverse events are known to occur. The duration of this interval must be 
discussed with the study chair and the study research coordinator 
* Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days 
after the completion of interleukins, interferon or cytokines (other than hematopoietic growth 
factors) 
* Stem cell Infusions (with or without total body irradiation [TBI]): 
** Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion 
including donor lymphocyte infusion (DLI) or boost infusion:  
*** >= 60 days after infusion for bone marrow or stem cell transplant and  
*** >= 4 weeks after infusion for any stem cell infusion including DLI or boost infusion 
*** There must be no evidence of graft versus host disease (GVHD) 
** Autologous stem cell infusion including boost infusion: >= 42 days 
* Cellular Therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified 
T cells, natural killer [NK] cells, dendritic cells, etc.) 
* XRT/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after 
TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone 
marrow (BM) radiation 
* Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I-metaiodobenzylguanidine 
[MIBG]): >= 42 days after systemically administered radiopharmaceutical therapy 
* Study-specific limitations on prior therapy: small molecule investigational agents: >= 14 days or 
> 5 half-lives must have elapsed from the last dose of the agent, whichever is greater 
• Platelet count >= 20,000/mm^3 (may receive platelet transfusions) 
• Hemoglobin >= 8.0 g/dL at baseline (may receive red blood cell [RBC] transfusions) 
• Creatinine clearance or radioisotope glomerular filtration rate [GFR] >= 70 ml/min/1.73 m^2 or a 
serum creatinine based on age/gender as follows: 
* Age: Maximum serum creatinine (mg/dL) 
** 2 to < 6 years: 0.8 (male and female) 
** 6 to < 10 years: 1 (male and female) 
** 10 to < 13 years: 1.2 (male and female) 
** 13 to < 16 years: 1.5 (male); 1.4 (female) 
** >= 16 years: 1.7 (male); 1.4 (female) 
• Bilirubin (sum of conjugated + unconjugated) =< 1.5 x upper limit of normal (ULN) for age 
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 225 U/L. 
For the purpose of this study, the ULN for SGPT is 45 U/L 
• Serum albumin >= 2 g/dL 
• Left ventricular ejection fraction of >= 50% by echocardiogram 
• Regulatory Requirements 
* All patients and/or their parents or legal authorized representatives must sign a written 
informed consent. Assent, when appropriate, will be obtained according to institutional 
guidelines 
* All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) 
requirements for human studies must be met 
 
 
Exclusion Criteria 
• AML associated with Down syndrome or t(15;17) is not eligible for study 
• Pregnant or breast-feeding women will not be entered on this study due to risks of fetal and 
teratogenic adverse events as seen in animal/human studies. Pregnancy tests must be obtained 

in girls who are post-menarchal. Males or females of reproductive potential may not participate 
unless they have agreed to use an effective contraceptive method for the duration of study 
therapy and for 2 months after the last dose of enasidenib. Abstinence is an acceptable method 
of birth control. It is not known if enasidenib is present in breast milk. Breastfeeding is not 
recommended during therapy or for at least 30 days after the last dose of enasidenib 
• Concomitant Medications: 
* Corticosteroids: Patients receiving corticosteroids who have not been on a stable or 
decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible. If used to 
modify immune adverse events related to prior therapy, >= 14 days must have elapsed since 
last dose of corticosteroid. The use of corticosteroids to manage the side effect of IDH 
inhibitor-associated differentiation syndrome (IDH-DS), is permitted on study 
* Investigational drugs: Patients who are currently receiving another investigational drug are not 
eligible  
* Anti-cancer agents: Patients who are currently receiving other anti-cancer agents are not 
eligible (except leukemia patients receiving hydroxyurea, which may be continued until 24 hours 
prior to start of protocol therapy; the use of hydroxyurea to manage the side effect of IDH-DS, is 
permitted on study) 
* Anti-GVHD agents post-transplant: Patients who are receiving cyclosporine, tacrolimus or 
other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible 
for this trial 
• Patients must be able to swallow intact tablets whole or use the alternate enasidenib 
formulation. 
* Patients with known hypersensitivity to any of the components of enasidenib are not eligible. 
* Patients with prior exposure to enasidenib or another IDH2 inhibitor are not eligible. 
* Patients taking the following drugs will be excluded from study entry unless these drugs are 
discontinued or patients are transferred to a medically acceptable alternative > 5 half-lives 
before the first dose of enasidenib. 
** Drugs with a narrow therapeutic range that are sensitive substrates of the following 
cytochrome P450 (CYP) enzymes: CYP2C8 (e.g. paclitaxel), 2C9 (e.g. phenytoin and warfarin), 
2C19 (e.g. s-mephenytoin), 2D6 (e.g. thioridazine), and 1A2 (e.g. theophylline and tizanidine). 
** Breast cancer resistant protein (BCRP) transporter-sensitive substrate rosuvastatin 
• Patients with the following leukemia complications are not eligible for this trial: 
* No intrathecal chemotherapy is permitted on study. Prior to study enrollment, cerebrospinal 
fluid (CSF) evaluation is only required if there is a clinical suspicion for CNS leukemia. Clinical 
signs of CNS leukemia (such as facial nerve palsy, brain/eye involvement or hypothalamic 
syndrome) are not eligible for this trial 
* Immediately life-threatening, severe complications of leukemia including uncontrolled bleeding, 
pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation 
• Patients who have received a prior solid organ transplantation are not eligible 
• Infection: Patients who have an uncontrolled infection or patients with known human 
immunodeficiency virus (HIV) or active hepatitis B or C are not eligible 
• Patients who in the opinion of the investigator may not be able to comply with the safety 
monitoring requirements of the study are not eligible 
 
 
 
 
 
Intervention(s) 
Type 
Name 
Alternate Name 
Description 
Drug 
Enasidenib 
1446502-11-9, AG-221, 
CC-90007 Free Base, 
ENASIDENIB 
Given PO 
Drug 
Enasidenib Mesylate 
1650550-25-6, 
Given PO 

2-Methyl-1-[(4-[6-(trifluo
romethyl)pyridin-2-yl]-6-
{[2-(trifluoromethyl)pyrid
in-4-yl]amino}-1,3,5-tria
zin-2-yl)amino]propan-2
-ol Methanesulfonate, 
2-Propanol, 
2-Methyl-1-((4-(6-(trifluo
romethyl)-2-pyridinyl)-6-
((2-(trifluoromethyl)-4-py
ridinyl)amino)-1,3,5-triaz
in-2-yl)amino)-, 
Methanesulfonate (1:1), 
AG-221 Mesylate, 
CC-90007, 
ENASIDENIB 
MESYLATE, 
Enasidenib 
Methanesulfonate, 
Idhifa 
Procedure/Surgery 
Bone Marrow Aspiration Bone Marrow 
Aspiration, bone 
marrow aspiration 
Undergo bone marrow 
aspiration 
Procedure/Surgery 
Bone Marrow Biopsy 
Biopsy of Bone Marrow, 
Biopsy, Bone Marrow, 
bone marrow biopsy, 
Bone Marrow Biopsy 
Undergo bone marrow 
biopsy 
Procedure/Surgery 
Biospecimen Collection Biological Sample 
Collection, Biological 
Sample Collection, 
Biospecimen Collected, 
Biospecimen Collection, 
Specimen Collection 
Undergo collection of 
blood 
 
 
 
Arm/Group(s) 
 
Type 
Experimental 
Label 
Treatment (enasidenib) 
Description 
Patients receive enasidenib PO QD on days 
1-28. Treatment repeats every 28 days for up to 
12 cycles in the absence of disease progression 
or unacceptable toxicity. Patients also undergo 
bone marrow aspiration and/or biopsy and 
collection of blood on study. 
Intervention(s) 

Type 
Name 
Alternate Name 
Description 
Drug 
Enasidenib 
1446502-11-9, AG-221, 
CC-90007 Free Base, 
ENASIDENIB 
Given PO 
Drug 
Enasidenib Mesylate 
1650550-25-6, 
2-Methyl-1-[(4-[6-(trifluo
romethyl)pyridin-2-yl]-6-
{[2-(trifluoromethyl)pyrid
in-4-yl]amino}-1,3,5-tria
zin-2-yl)amino]propan-2
-ol Methanesulfonate, 
2-Propanol, 
2-Methyl-1-((4-(6-(trifluo
romethyl)-2-pyridinyl)-6-
((2-(trifluoromethyl)-4-py
ridinyl)amino)-1,3,5-triaz
in-2-yl)amino)-, 
Methanesulfonate (1:1), 
AG-221 Mesylate, 
CC-90007, 
ENASIDENIB 
MESYLATE, 
Enasidenib 
Methanesulfonate, 
Idhifa 
Given PO 
Procedure/Surgery 
Bone Marrow Aspiration Bone Marrow 
Aspiration, bone 
marrow aspiration 
Undergo bone marrow 
aspiration 
Procedure/Surgery 
Bone Marrow Biopsy 
Biopsy of Bone Marrow, 
Biopsy, Bone Marrow, 
bone marrow biopsy, 
Bone Marrow Biopsy 
Undergo bone marrow 
biopsy 
Procedure/Surgery 
Biospecimen Collection Biological Sample 
Collection, Biological 
Sample Collection, 
Biospecimen Collected, 
Biospecimen Collection, 
Specimen Collection 
Undergo collection of 
blood 
 
 
 
Primary Outcome Measures 
Title 
Description 
Time Frame 
Incidence of dose limiting 
toxicities of enasidenib 
Frequencies (%) of patients with 
a dose limiting toxicity stratified 
by dose level. 
Up to 1 year after last dose of 
study drug 
Area under the plasma 
A descriptive analysis of the 
Up to 120 days 

concentration versus time curve 
of enasidenib 
area under the plasma 
concentration versus time curve 
of enasidenib including median, 
minimum and maximum by dose 
level. 
Total plasma clearance of 
enasidenib 
A descriptive analysis of the 
total plasma clearance of 
enasidenib including median, 
minimum and maximum by dose 
level. 
Up to 120 days 
Elimination half-life of 
enasidenib 
A descriptive analysis of the 
elimination half-life of 
enasidenib including median, 
minimum and maximum by dose 
level. 
Up to 120 days 
Maximum concentration of 
enasidenib 
A descriptive analysis of the 
maximum concentration of 
enasidenib including median, 
minimum and maximum by dose 
level. 
Up to 120 days 
 
 
 
Secondary Outcome Measures 
Title 
Description 
Time Frame 
Plasma 2-HG levels of 
enasidenib 
A descriptive analysis of the 
plasma 2-HG levels of 
enasidenib including median, 
minimum and maximum by dose 
level. 
Up to 120 days 
Overall Response Rate of 
enasidenib 
Frequency (%) of patients with 
at least partial response by dose 
level. 
Up to 2 years 
Composite complete remission 
rate (complete remission [CR]/ 
CR with incomplete hematologic 
recovery [CRi]) 
Will be reported in a table as 
frequency of response (%) for 
total overall response rate 
(ORR) by dose level. 
Up to 1 year after last dose of 
study drug 
Time to response of enasidenib Median time to response with 
95% confidence interval. 
From the date of first dose to the 
date of first documented 
response, assessed up to 1 year 
after last dose of study drug 
Time to complete remission of 
enasidenib 
Median time to remission with 
95% confidence interval. 
From the date of first dose to the 
date of first documented CR, 
assessed up to 1 year after last 
dose of study drug 
Duration of response of 
enasidenib 
Median duration of response 
with 95% confidence interval. 
From the date of first 
documented response to the 

date of first documented 
confirmed disease 
progression/relapse, or death, 
whichever occurs first, assessed 
up to 1 year 
Duration of complete response 
of enasidenib 
Median duration of remission 
with 95% confidence interval. 
From the date of first 
documented CR to the date of 
first documented confirmed 
disease progression/relapse, or 
death, whichever occurs first, 
assessed up to 1 year after last 
dose of study drug 
Event-free survival of 
enasidenib 
Median time to event with 95% 
confidence interval. 
From the date of first dose to the 
date of documented confirmed 
disease progression/relapse, or 
death, whichever occurs first, 
assessed up to 1 year after last 
dose of study drug 
Overall survival of enasidenib 
Median time to death with 95% 
confidence interval. 
From the patient’s first dose to 
the date of the death, or the last 
date the patient was known to 
be alive, assessed up to 1 year 
after last dose of study drug 
 
 
 
Markers 
Marker Name 
Evaluation Type Assay Type 
Biomarker Use Biomarker 
Purpose 
Specimen Type 
2-HG (2HG; 
2-HG; 
Alpha-Hydroxyg
lutarate; 
2-Hydroxypenta
nedioic Acid; 
Alpha-Hydroxyg
lutaric Acid; 
Pentanedioic 
Acid, 
2-Hydroxy-; 
2-Hydroxyglutar
ic Acid; 
2-Hydroxyglutar
ate; 
2-Hydroxy-Pent
anedioic Acid) 
Level/Quantity Unspecified 
Integrated 
Response 
Assessment 
Blood 
Blasts 5-25 
Level/Quantity Unspecified 
Integrated 
Response 
Bone Marrow 

Percent of Bone 
Marrow 
Nucleated Cells 
Assessment 
Blasts More 
than 5 Percent 
of Bone Marrow 
Nucleated Cells 
(Blasts Greater 
than 5 Percent 
of Bone Marrow 
Nucleated 
Cells; Blasts 
Over 5 Percent 
of Bone Marrow 
Nucleated 
Cells) 
Level/Quantity Flow Cytometry Integral 
Eligibility 
Criterion - 
Inclusion 
Bone Marrow 
Blasts Under 5 
Percent of Bone 
Marrow 
Nucleated Cells 
(Blasts Less 
than 5 Percent 
of Bone Marrow 
Nucleated 
Cells) 
Level/Quantity Unspecified 
Integrated 
Response 
Assessment 
Bone Marrow 
Bone Marrow 
Blasts 
Decreased by 
50 Percent or 
More 
Compared to 
Pretreatment 
Level 
(Decrease of 
Pretreatment 
Bone Marrow 
Blast 
Percentage by 
at Least 50 
Percent; 
Decrease of 
Pretreatment 
Bone Marrow 
Blast 
Percentage by 
at Least 50) 
Level/Quantity Unspecified 
Integrated 
Response 
Assessment 
Bone Marrow 
IDH2 Gene 
Genetic 
Unspecified 
Integral 
Eligibility 
Blood, Bone 

Mutation 
(Isocitrate 
Dehydrogenase 
(NADP(+)) 2, 
Mitochondrial 
Gene Mutation; 
IDH2 Mutation; 
Isocitrate 
Dehydrogenase 
1 (NADP+), 
Mitochondrial 
Gene Mutation) 
Analysis 
Criterion - 
Inclusion 
Marrow 
 
 
 
Participating Sites 
PO ID 
Facility 
Contact 
Recruitment Status 
& Date(s) 
Investigator(s) 
66945 
Alfred I duPont 
Hospital for 
Children, 
Wilmington, DE 
19803 USA 
Site Public 
Contact, email: 
Allison.bruce@ne
mours.org, phone: 
302-651-5572 
Active as of 
02/01/2023 
• Scott Bradfield - 
Principal 
Investigator 
 
29135 
Arkansas 
Children's Hospital, 
Little Rock, AR 
72202-3591 USA 
Site Public 
Contact, email: , 
phone: 
501-364-7373 
Active as of 
02/01/2023 
• David Becton - 
Principal 
Investigator 
 
133247 
C S Mott Children's 
Hospital, Ann 
Arbor, MI 48109 
USA 
Site Public 
Contact, email: , 
phone: 
800-865-1125 
Active as of 
02/01/2023 
• Rajen Mody - 
Principal 
Investigator 
 
9858 
Centre Hospitalier 
Universitaire 
Sainte-Justine, 
Montreal, Quebec 
H3T 1C5 CAN 
Site Public 
Contact, email: 
yvan.samson@um
ontreal.ca, phone: 
514-345-4931 
Temporarily 
Closed to Accrual 
as of 08/31/2022 
• Yvan Samson - 
Principal 
Investigator 
 
58509 
Children's Hospital 
Colorado, Aurora, 
CO 80045 USA 
Site Public 
Contact, email: 
josh.b.gordon@ns
mtp.kp.org, phone: 
303-764-5056 
Active as of 
02/01/2023 
• Margaret Macy - 
Principal 
Investigator 
 
25745 
Children's Hospital 
of Alabama, 
Birmingham, AL 
35233 USA 
Site Public 
Contact, email: 
oncologyresearch
@peds.uab.edu, 
phone: 
205-638-9285 
Active as of 
02/01/2023 
• Matthew Kutny - 
Principal 
Investigator 
 

196933 
Children's Hospital 
of Pittsburgh of 
UPMC, Pittsburgh, 
PA 15224 USA 
Site Public 
Contact, email: 
jean.tersak@chp.e
du, phone: 
412-692-8570 
Active as of 
02/01/2023 
• Andrew 
Bukowinski - 
Principal 
Investigator 
 
230502 
Children's Hospital 
of San Antonio, 
San Antonio, TX 
78207 USA 
Site Public 
Contact, email: 
bridget.medina@c
hristushealth.org, 
phone: 
210-704-2894 
Active as of 
05/16/2023 
• Timothy Griffin - 
Principal 
Investigator 
 
238382 
Children's Hospital 
of The King's 
Daughters, Norfolk, 
VA 23507 USA 
Site Public 
Contact, email: 
CCBDCresearch@
chkd.org, phone: 
757-668-7243 
Active as of 
02/01/2023 
• Eric Lowe - 
Principal 
Investigator 
 
141741 
Children's Mercy 
Hospitals and 
Clinics, Kansas 
City, MO 64108 
USA 
Site Public 
Contact, email: 
rryan@cmh.edu, 
phone: 
816-302-6808 
Active as of 
02/01/2023 
• Kevin Ginn - 
Principal 
Investigator 
 
65938 
Children's National 
Medical Center, 
Washington, DC 
20010 USA 
Site Public 
Contact, email: , 
phone: 
202-884-2549 
Active as of 
02/01/2023 
• AeRang Kim - 
Principal 
Investigator 
 
182911 
Cincinnati 
Children's Hospital 
Medical Center, 
Cincinnati, OH 
45229 USA 
Site Public 
Contact, email: 
cancer@cchmc.org
, phone: 
513-636-2799 
Active as of 
02/01/2023 
• Joseph Pressey - 
Principal 
Investigator 
 
160485 
Hackensack 
University Medical 
Center, 
Hackensack, NJ 
07601 USA 
Site Public 
Contact, email: , 
phone: 
201-996-2879 
Active as of 
02/01/2023 
• Jing Chen - 
Principal 
Investigator 
 
125217 
Johns Hopkins 
University/Sidney 
Kimmel Cancer 
Center, Baltimore, 
MD 21287 USA 
Site Public 
Contact, email: 
jhcccro@jhmi.edu, 
phone: 
410-955-8804 
Active as of 
02/01/2023 
• Alan Friedman - 
Principal 
Investigator 
 
65850 
MedStar 
Georgetown 
University Hospital, 
Washington, DC 
20007 USA 
Site Public 
Contact, email: , 
phone: 
202-444-2223 
Closed to Accrual 
as of 11/17/2022 
• Nina 
Kadan-Lottick - 
Principal 
Investigator 
 
71176 
Nicklaus Children's 
Hospital, Miami, FL 
Site Public 
Contact, email: , 
Active as of 
05/30/2023 
• Ziad Khatib - 
Principal 

33155 USA 
phone: 
888-624-2778 
Investigator 
 
105447 
Riley Hospital for 
Children, 
Indianapolis, IN 
46202 USA 
Site Public 
Contact, email: , 
phone: 
800-248-1199 
Active as of 
02/01/2023 
• Sandeep Batra - 
Principal 
Investigator 
 
60830 
Rocky Mountain 
Hospital for 
Children-Presbyteri
an Saint Luke's 
Medical Center, 
Denver, CO 80218 
USA 
Site Public 
Contact, email: , 
phone: 
303-839-6000 
Active as of 
02/01/2023 
• Jennifer Clark - 
Principal 
Investigator 
 
221716 
Saint Jude 
Children's 
Research Hospital, 
Memphis, TN 
38105 USA 
Site Public 
Contact, email: 
referralinfo@stjude
.org, phone: 
888-226-4343 
Active as of 
02/01/2023 
• Jeffrey Rubnitz - 
Principal 
Investigator 
 
173533 
State University of 
New York Upstate 
Medical University, 
Syracuse, NY 
13210 USA 
Site Public 
Contact, email: , 
phone: 
315-464-5476 
Active as of 
02/01/2023 
• Philip 
Monteleone - 
Principal 
Investigator 
 
70710 
University of 
Florida Health 
Science Center - 
Gainesville, 
Gainesville, FL 
32610 USA 
Site Public 
Contact, email: 
cancer-center@ufl.
edu, phone: 
352-273-8010 
Closed to Accrual 
as of 02/01/2023 
• William Slayton - 
Principal 
Investigator 
 
190601 
University of 
Oklahoma Health 
Sciences Center, 
Oklahoma City, OK 
73104 USA 
Site Public 
Contact, email: 
ou-clinical-trials@o
uhsc.edu, phone: 
405-271-8777 
Active as of 
02/21/2023 
• Rene 
McNall-Knapp - 
Principal 
Investigator 
 
226789 
UT 
Southwestern/Sim
mons Cancer 
Center-Dallas, 
Dallas, TX 75390 
USA 
Site Public 
Contact, email: 
canceranswerline
@UTSouthwestern
.edu, phone: 
214-648-7097 
Active as of 
02/01/2023 
• Kathleen 
(Wiertel) Ludwig 
- Principal 
Investigator 
 
221306 
Vanderbilt 
University/Ingram 
Cancer Center, 
Nashville, TN 
37232 USA 
Site Public 
Contact, email: , 
phone: 
800-811-8480 
Active as of 
02/01/2023 
• Sara 
Zarnegar-Lumley 
- Principal 
Investigator