Trial Outcomes & Findings for Alisertib in Treating Young Patients With Recurrent or Refractory Solid Tumors or Leukemia (NCT NCT01154816)

NCT ID: NCT01154816

Last Updated: 2020-05-13

Results Overview

For patients with recurrent solid tumors a patient who experienced a complete or partial response according to RECIST version 1.1 criteria is considered a responder. For patients with recurrent acute lymphoblastic leukemia a patient who experiences a bone marrow evaluation with \< 5% blast cells on morphological evaluation of bone marrow will be considered a responder. For patients with recurrent acute myelogenous leukemia a patient who experiences a complete remission or complete remission with partial recovery of platelet count according to the AML International Working Group Criteria will be considered a responder.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 118 participants
• Primary outcome timeframe: From first dose of alisertib through 6 cycles of protocol therapy or until removal from protocol therapy whichever occurred first.
• Results posted on: 2020-05-13

Participant Flow

Participant milestones

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma Experimental: Arm 3	Recurrent Osteosarcoma Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma Experimental: Arm 6	Childhood Hepatoblastoma Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia Experimental: Arm 11	Rhaboid Malignancy Experimental: Arm 12
Overall Study STARTED	20	12	10	10	10	8	6	7	10	10	11	4
Overall Study COMPLETED	0	2	0	0	0	0	0	0	1	0	0	0
Overall Study NOT COMPLETED	20	10	10	10	10	8	6	7	9	10	11	4

Reasons for withdrawal

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma Experimental: Arm 3	Recurrent Osteosarcoma Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma Experimental: Arm 6	Childhood Hepatoblastoma Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia Experimental: Arm 11	Rhaboid Malignancy Experimental: Arm 12
Overall Study Withdrawal by Subject	1	0	0	1	0	0	0	0	1	3	0	0
Overall Study Progressive Disease	13	8	9	9	8	8	5	6	8	6	10	3
Overall Study Physician Decision	1	0	1	0	2	0	0	0	0	1	1	1
Overall Study Adverse Event	5	2	0	0	0	0	1	1	0	0	0	0

Baseline Characteristics

Alisertib in Treating Young Patients With Recurrent or Refractory Solid Tumors or Leukemia

Baseline characteristics by cohort

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=20 Participants Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll n=12 Participants Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma n=10 Participants Experimental: Arm 3	Recurrent Osteosarcoma n=10 Participants Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor n=10 Participants Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma n=8 Participants Experimental: Arm 6	Childhood Hepatoblastoma n=6 Participants Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor n=7 Participants Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors n=10 Participants Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia n=10 Participants Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia n=11 Participants Experimental: Arm 11	Rhaboid Malignancy n=4 Participants Experimental: Arm 12	Total n=118 Participants Total of all reporting groups
Age, Continuous	8 Years n=5 Participants	8 Years n=7 Participants	12 Years n=5 Participants	17 Years n=4 Participants	14 Years n=21 Participants	16 Years n=10 Participants	5 Years n=115 Participants	11 Years n=24 Participants	10 Years n=42 Participants	13 Years n=42 Participants	10 Years n=42 Participants	5 Years n=42 Participants	11 Years n=36 Participants
Sex: Female, Male Female	10 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants	3 Participants n=4 Participants	3 Participants n=21 Participants	4 Participants n=10 Participants	1 Participants n=115 Participants	4 Participants n=24 Participants	7 Participants n=42 Participants	3 Participants n=42 Participants	6 Participants n=42 Participants	2 Participants n=42 Participants	52 Participants n=36 Participants
Sex: Female, Male Male	10 Participants n=5 Participants	10 Participants n=7 Participants	3 Participants n=5 Participants	7 Participants n=4 Participants	7 Participants n=21 Participants	4 Participants n=10 Participants	5 Participants n=115 Participants	3 Participants n=24 Participants	3 Participants n=42 Participants	7 Participants n=42 Participants	5 Participants n=42 Participants	2 Participants n=42 Participants	66 Participants n=36 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	3 Participants n=4 Participants	1 Participants n=21 Participants	2 Participants n=10 Participants	3 Participants n=115 Participants	2 Participants n=24 Participants	4 Participants n=42 Participants	2 Participants n=42 Participants	4 Participants n=42 Participants	1 Participants n=42 Participants	27 Participants n=36 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	15 Participants n=5 Participants	10 Participants n=7 Participants	7 Participants n=5 Participants	7 Participants n=4 Participants	8 Participants n=21 Participants	5 Participants n=10 Participants	3 Participants n=115 Participants	4 Participants n=24 Participants	5 Participants n=42 Participants	8 Participants n=42 Participants	5 Participants n=42 Participants	3 Participants n=42 Participants	80 Participants n=36 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants	0 Participants n=4 Participants	1 Participants n=21 Participants	1 Participants n=10 Participants	0 Participants n=115 Participants	1 Participants n=24 Participants	1 Participants n=42 Participants	0 Participants n=42 Participants	2 Participants n=42 Participants	0 Participants n=42 Participants	11 Participants n=36 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	0 Participants n=115 Participants	0 Participants n=24 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=36 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	0 Participants n=115 Participants	0 Participants n=24 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=36 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	0 Participants n=115 Participants	0 Participants n=24 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=36 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants	2 Participants n=4 Participants	0 Participants n=21 Participants	1 Participants n=10 Participants	1 Participants n=115 Participants	0 Participants n=24 Participants	2 Participants n=42 Participants	2 Participants n=42 Participants	2 Participants n=42 Participants	0 Participants n=42 Participants	14 Participants n=36 Participants
Race (NIH/OMB) White	15 Participants n=5 Participants	7 Participants n=7 Participants	7 Participants n=5 Participants	7 Participants n=4 Participants	8 Participants n=21 Participants	3 Participants n=10 Participants	3 Participants n=115 Participants	4 Participants n=24 Participants	5 Participants n=42 Participants	5 Participants n=42 Participants	2 Participants n=42 Participants	3 Participants n=42 Participants	69 Participants n=36 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants	0 Participants n=21 Participants	0 Participants n=10 Participants	0 Participants n=115 Participants	0 Participants n=24 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=42 Participants	0 Participants n=36 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants	4 Participants n=7 Participants	2 Participants n=5 Participants	1 Participants n=4 Participants	2 Participants n=21 Participants	4 Participants n=10 Participants	2 Participants n=115 Participants	3 Participants n=24 Participants	3 Participants n=42 Participants	3 Participants n=42 Participants	7 Participants n=42 Participants	1 Participants n=42 Participants	35 Participants n=36 Participants
Region of Enrollment Canada	1 participants n=5 Participants	2 participants n=7 Participants	1 participants n=5 Participants	0 participants n=4 Participants	0 participants n=21 Participants	0 participants n=10 Participants	1 participants n=115 Participants	1 participants n=24 Participants	1 participants n=42 Participants	0 participants n=42 Participants	1 participants n=42 Participants	0 participants n=42 Participants	8 participants n=36 Participants
Region of Enrollment United States	18 participants n=5 Participants	9 participants n=7 Participants	9 participants n=5 Participants	10 participants n=4 Participants	10 participants n=21 Participants	8 participants n=10 Participants	5 participants n=115 Participants	6 participants n=24 Participants	8 participants n=42 Participants	10 participants n=42 Participants	10 participants n=42 Participants	4 participants n=42 Participants	107 participants n=36 Participants
Region of Enrollment United Kingdom	1 participants n=5 Participants	1 participants n=7 Participants	0 participants n=5 Participants	0 participants n=4 Participants	0 participants n=21 Participants	0 participants n=10 Participants	0 participants n=115 Participants	0 participants n=24 Participants	0 participants n=42 Participants	0 participants n=42 Participants	0 participants n=42 Participants	0 participants n=42 Participants	2 participants n=36 Participants

PRIMARY outcome

Timeframe: From first dose of alisertib through 6 cycles of protocol therapy or until removal from protocol therapy whichever occurred first.

Population: Of the 118 enrolled patients, two were not evaluable for the primary outcome measure. Neither received protocol therapy. One was enrolled in arm 11, one was enrolled in Arm 1.

For patients with recurrent solid tumors a patient who experienced a complete or partial response according to RECIST version 1.1 criteria is considered a responder. For patients with recurrent acute lymphoblastic leukemia a patient who experiences a bone marrow evaluation with < 5% blast cells on morphological evaluation of bone marrow will be considered a responder. For patients with recurrent acute myelogenous leukemia a patient who experiences a complete remission or complete remission with partial recovery of platelet count according to the AML International Working Group Criteria will be considered a responder.

Outcome measures

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=19 Participants Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll n=12 Participants Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma n=10 Participants Experimental: Arm 3	Recurrent Osteosarcoma n=10 Participants Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor n=10 Participants Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma n=8 Participants Experimental: Arm 6	Childhood Hepatoblastoma n=6 Participants Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor n=7 Participants Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors n=10 Participants Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia n=10 Participants Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia n=10 Participants Experimental: Arm 11	Rhaboid Malignancy n=4 Participants Experimental: Arm 12
Number of Participants With Overall Response	0 Patients	1 Patients	0 Patients	0 Patients	0 Patients	0 Patients	0 Patients	0 Patients	1 Patients	0 Patients	0 Patients	0 Patients

SECONDARY outcome

Timeframe: Up to 24 months

Population: 116 patients contributed 360 patient-cycles to the analysis.

The number of patient-cycles in which the adverse event considered grade 3 or higher AE according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 considered by the treating physician to be possibly, probably or definitely related to alisertib.

Outcome measures

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=116 Participants Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma Experimental: Arm 3	Recurrent Osteosarcoma Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma Experimental: Arm 6	Childhood Hepatoblastoma Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia Experimental: Arm 11	Rhaboid Malignancy Experimental: Arm 12
Number of Patients Cycles With Grade 3 or Higher Adverse Event Allergic Reaction	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Alopecia	27 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Anal Mucocitis	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Anemia	141 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Anorexia	8 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Aspartate Aminotransferase Increase	15 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Back Pain	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Blood Bilirubin Increase	7 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Blurred Vision	2 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Bullous Dermatitis	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Colitis	2 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Depression	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Diarrhea	8 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Dizziness	15 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Dry Mouth	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Enterocolitis	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Erectile Dysfunction	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Fatigue	14 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Febrile Neutropenia	13 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Other Gastrointestinal Disorders	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event GGT Increase	2 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Headache	3 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Hepatic Hemhorrage	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Hyperkalemia	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Hypersomnia	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Hyperuricemia	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Lymphocyte Count Decrease	65 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Lymphocyte Count Increase	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Mucosal Infection	2 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Oral Mucocitis	35 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Nausea	11 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Decreased Neutrophil Count	208 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Oral Pain	12 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Otitis Media	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Pain in an Extremity	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Palmar-Plantar Erythrodysesthesia Syndrome	10 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Respiratory Failure	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Serum Amylase Increase	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Skin Infection	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Somnolence	2 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Sore Throat	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Tumor Lysis Syndrome	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Upper Respiratory Infection	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Urticaria	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Urinary Tract Infection	4 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Vomiting	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event White Blood Cell Decrease	163 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Abdominal Pain	3 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Prolonged Partial Thromboplatin Time	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Acute Kidney Injury	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Agitation	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Alanine Aminotransferase Elevation	25 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Confusion	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Dehydration	5 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Euphoria	3 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Eye Pain	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Hypoalbuminemia	7 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Hypocalcemia	2 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Hypokalemia	9 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Hyponatremia	3 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Hypophosphatemia	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Other Infections	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event INR Increase	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Keratitis	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Lethargy	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Lung Infection	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Photophobia	4 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Platelet Count Decrease	92 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Pruritis	4 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—
Number of Patients Cycles With Grade 3 or Higher Adverse Event Psychiatric Disorders	1 Patient-cycle	—	—	—	—	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: day 1 of protocol therapy

Population: Of all eligible patients enrolled, 32 provided a prior to the first day of administration sample for analysis.

Serum concentration of alisertib prior to the first day of administration in nanograms/milliliter.

Outcome measures

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=32 Participants Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma Experimental: Arm 3	Recurrent Osteosarcoma Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma Experimental: Arm 6	Childhood Hepatoblastoma Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia Experimental: Arm 11	Rhaboid Malignancy Experimental: Arm 12
Serum Concentration of Alisertib Prior to the First Day of Administration	0 ng/ml Standard Deviation 0	—	—	—	—	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: day 1 of protocol therapy

Population: Of all eligible patients enrolled, 16 provided a first day of administration one hour after administration infusion sample for analysis.

Serum concentration of alisertib on the first day of administration one hour after administration in nanograms/milliliter.

Outcome measures

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=16 Participants Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma Experimental: Arm 3	Recurrent Osteosarcoma Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma Experimental: Arm 6	Childhood Hepatoblastoma Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia Experimental: Arm 11	Rhaboid Malignancy Experimental: Arm 12
Serum Concentration of Alisertib on the First Day of Administration One Hour After Administration	1874.0625 ng/ml Standard Deviation 1131.87375	—	—	—	—	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: day 1 of protocol therapy

Population: Of all eligible patients enrolled, 16 provided a first day of administration three hours after administration sample for analysis.

Serum concentration of alisertib on the first day of administration three hours after administration in nanograms/milliliter.

Outcome measures

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=16 Participants Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma Experimental: Arm 3	Recurrent Osteosarcoma Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma Experimental: Arm 6	Childhood Hepatoblastoma Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia Experimental: Arm 11	Rhaboid Malignancy Experimental: Arm 12
Serum Concentration of Alisertib on the First Day of Administration Three Hours After Administration	1863.9375 ng/ml Standard Deviation 588.116822	—	—	—	—	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: day 1 of protocol therapy

Population: Of all eligible patients enrolled, 16 provided a first day of administration six hours after administration sample for analysis.

Serum concentration of alisertib on the first day of administration six hours after administration in nanograms/milliliter.

Outcome measures

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=16 Participants Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma Experimental: Arm 3	Recurrent Osteosarcoma Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma Experimental: Arm 6	Childhood Hepatoblastoma Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia Experimental: Arm 11	Rhaboid Malignancy Experimental: Arm 12
Serum Concentration of Alisertib on the First Day of Administration Six Hours After Administration	1234 ng/ml Standard Deviation 512.203475	—	—	—	—	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: day 4 of protocol therapy

Population: Of all eligible patients enrolled, 30 provided a fourth day of administration prior to the administration of the day 4 dose sample for analysis.

Serum concentration of alisertib on the fourth day of administration prior to the administration of the day 4 dose in nanograms/milliliter.

Outcome measures

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=30 Participants Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma Experimental: Arm 3	Recurrent Osteosarcoma Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma Experimental: Arm 6	Childhood Hepatoblastoma Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia Experimental: Arm 11	Rhaboid Malignancy Experimental: Arm 12
Serum Concentration of Alisertib on the Fourth Day of Administration Prior to the Administration of the Day 4 Dose	1123.73333 ng/ml Standard Deviation 715.013235	—	—	—	—	—	—	—	—	—	—	—

SECONDARY outcome

Timeframe: day 7 of protocol therapy

Population: Of all eligible patients enrolled, 31 provided a seventh day of administration prior to the administration of the day 7 dose sample for analysis.

Serum concentration of alisertib on the seventh day of administration prior to the administration of the day 7 dose in nanograms/milliliter.

Outcome measures

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=31 Participants Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma Experimental: Arm 3	Recurrent Osteosarcoma Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma Experimental: Arm 6	Childhood Hepatoblastoma Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia Experimental: Arm 11	Rhaboid Malignancy Experimental: Arm 12
Serum Concentration of Alisertib on the Seventh Day of Administration Prior to the Administration of the Day 7 Dose.	1097.50645 ng/ml Standard Deviation 1012.34016	—	—	—	—	—	—	—	—	—	—	—

Adverse Events

Recurrent Neuroblastoma With Measurable Disease at Enrollment

Serious events: 7 serious events

Other events: 15 other events

Deaths: 0 deaths

Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll

Serious events: 5 serious events

Other events: 11 other events

Deaths: 0 deaths

Previously Treated Childhood Rhabdomyosarcoma

Serious events: 4 serious events

Other events: 8 other events

Deaths: 0 deaths

Recurrent Osteosarcoma

Serious events: 7 serious events

Other events: 9 other events

Deaths: 0 deaths

Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor

Serious events: 5 serious events

Other events: 9 other events

Deaths: 0 deaths

Recurrent Childhood Soft Tissue Sarcoma

Serious events: 3 serious events

Other events: 6 other events

Deaths: 0 deaths

Childhood Hepatoblastoma

Serious events: 6 serious events

Other events: 3 other events

Deaths: 0 deaths

Recurrent Childhood Germ Cell Tumor

Serious events: 1 serious events

Other events: 5 other events

Deaths: 0 deaths

Recurrent Wilms Tumor and Other Childhood Kidney Tumors

Serious events: 3 serious events

Other events: 9 other events

Deaths: 0 deaths

Recurrent Childhood Acute Lympohblastic Leukemia

Serious events: 8 serious events

Other events: 10 other events

Deaths: 0 deaths

Recurrent Childhood Acute Myeloid Leukemia

Serious events: 5 serious events

Other events: 9 other events

Deaths: 0 deaths

Rhaboid Malignancy

Serious events: 2 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=20 participants at risk Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll n=12 participants at risk Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma n=10 participants at risk Experimental: Arm 3	Recurrent Osteosarcoma n=10 participants at risk Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor n=10 participants at risk Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma n=8 participants at risk Experimental: Arm 6	Childhood Hepatoblastoma n=6 participants at risk Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor n=7 participants at risk Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors n=10 participants at risk Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia n=10 participants at risk Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia n=11 participants at risk Experimental: Arm 11	Rhaboid Malignancy n=4 participants at risk Experimental: 12
Gastrointestinal disorders Abdominal pain	5.0% 1/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	10.0% 1/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Activated partial thromboplastin time prolonged	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Psychiatric disorders Agitation	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	14.3% 1/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Alanine aminotransferase increased	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Immune system disorders Allergic reaction	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Blood and lymphatic system disorders Anemia	0.00% 0/20	0.00% 0/12	10.0% 1/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Infections and infestations Anorectal infection	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Investigations Aspartate aminotransferase increased	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Blood bilirubin increased	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Musculoskeletal and connective tissue disorders Bone pain	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Cardiac disorders Cardiac disorders - Other, specify	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Infections and infestations Catheter related infection	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Gastrointestinal disorders Colitis	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
General disorders Death NOS	0.00% 0/20	0.00% 0/12	10.0% 1/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Dehydration	5.0% 1/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Psychiatric disorders Depression	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Diarrhea	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Dry mouth	0.00% 0/20	0.00% 0/12	10.0% 1/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Dysphagia	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	12.5% 1/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Dyspnea	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Enterocolitis	0.00% 0/20	0.00% 0/12	10.0% 1/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Infections and infestations Enterocolitis infectious	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Reproductive system and breast disorders Erectile dysfunction	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Blood and lymphatic system disorders Febrile neutropenia	5.0% 1/20	8.3% 1/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	50.0% 5/10	36.4% 4/11	0.00% 0/4
General disorders Fever	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Gastrointestinal disorders Gastrointestinal disorders - Other, specify	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Nervous system disorders Headache	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Vascular disorders Hematoma	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Hepatobiliary disorders Hepatic hemorrhage	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Hyperkalemia	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Hypersomnia	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Hyperuricemia	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Hypoalbuminemia	0.00% 0/20	0.00% 0/12	10.0% 1/10	30.0% 3/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	25.0% 1/4
Metabolism and nutrition disorders Hypocalcemia	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Metabolism and nutrition disorders Hypoglycemia	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Hypokalemia	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	20.0% 2/10	9.1% 1/11	25.0% 1/4
Metabolism and nutrition disorders Hyponatremia	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	10.0% 1/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	25.0% 1/4
Metabolism and nutrition disorders Hypophosphatemia	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Vascular disorders Hypotension	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Hypoxia	5.0% 1/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Infections and infestations Infections and infestations - Other, specify	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Investigations INR increased	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Intracranial hemorrhage	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Eye disorders Keratitis	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Infections and infestations Lung infection	0.00% 0/20	0.00% 0/12	10.0% 1/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	9.1% 1/11	0.00% 0/4
Investigations Lymphocyte count decreased	0.00% 0/20	0.00% 0/12	10.0% 1/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Mucositis oral	15.0% 3/20	8.3% 1/12	0.00% 0/10	20.0% 2/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	20.0% 2/10	0.00% 0/11	0.00% 0/4
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other, specify	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	12.5% 1/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Musculoskeletal and connective tissue disorders Myositis	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Nausea	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Musculoskeletal and connective tissue disorders Neck pain	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Neutrophil count decreased	0.00% 0/20	0.00% 0/12	10.0% 1/10	10.0% 1/10	10.0% 1/10	12.5% 1/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
General disorders Non-cardiac chest pain	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
General disorders Pain	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	12.5% 1/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	12.5% 1/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Platelet count decreased	5.0% 1/20	8.3% 1/12	10.0% 1/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/20	0.00% 0/12	10.0% 1/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/20	16.7% 2/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Pulmonary edema	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	12.5% 1/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Seizure	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Infections and infestations Sepsis	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Infections and infestations Sinusitis	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Infections and infestations Skin infection	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	20.0% 2/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Tumor lysis syndrome	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumor pain	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Infections and infestations Upper respiratory infection	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Urine output decreased	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations White blood cell decreased	0.00% 0/20	0.00% 0/12	10.0% 1/10	10.0% 1/10	0.00% 0/10	12.5% 1/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4

Other adverse events

Measure	Recurrent Neuroblastoma With Measurable Disease at Enrollment n=20 participants at risk Experimental: Arm 1	Recurrent Neuroblastoma Only MIBG Evaluable Disease at Enroll n=12 participants at risk Experimental: Arm 2	Previously Treated Childhood Rhabdomyosarcoma n=10 participants at risk Experimental: Arm 3	Recurrent Osteosarcoma n=10 participants at risk Experimental: Arm 4	Recurrent Ewing Sarcoma /Peripheral Neuroectodermal Tumor n=10 participants at risk Experimental: Arm 5	Recurrent Childhood Soft Tissue Sarcoma n=8 participants at risk Experimental: Arm 6	Childhood Hepatoblastoma n=6 participants at risk Experimental: Arm 7	Recurrent Childhood Germ Cell Tumor n=7 participants at risk Experimental: Arm 8	Recurrent Wilms Tumor and Other Childhood Kidney Tumors n=10 participants at risk Experimental: Arm 9	Recurrent Childhood Acute Lympohblastic Leukemia n=10 participants at risk Experimental: Arm 10	Recurrent Childhood Acute Myeloid Leukemia n=11 participants at risk Experimental: Arm 11	Rhaboid Malignancy n=4 participants at risk Experimental: 12
Gastrointestinal disorders Abdominal distension	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Gastrointestinal disorders Abdominal pain	0.00% 0/20	0.00% 0/12	20.0% 2/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	10.0% 1/10	0.00% 0/11	25.0% 1/4
Investigations Activated partial thromboplastin time prolonged	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Renal and urinary disorders Acute kidney injury	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Investigations Alanine aminotransferase increased	20.0% 4/20	25.0% 3/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	14.3% 1/7	10.0% 1/10	20.0% 2/10	0.00% 0/11	0.00% 0/4
Skin and subcutaneous tissue disorders Alopecia	30.0% 6/20	8.3% 1/12	20.0% 2/10	10.0% 1/10	30.0% 3/10	0.00% 0/8	0.00% 0/6	28.6% 2/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Gastrointestinal disorders Anal mucositis	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Blood and lymphatic system disorders Anemia	45.0% 9/20	58.3% 7/12	50.0% 5/10	50.0% 5/10	30.0% 3/10	12.5% 1/8	33.3% 2/6	14.3% 1/7	60.0% 6/10	70.0% 7/10	63.6% 7/11	50.0% 2/4
Metabolism and nutrition disorders Anorexia	10.0% 2/20	8.3% 1/12	0.00% 0/10	20.0% 2/10	30.0% 3/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	25.0% 1/4
Investigations Aspartate aminotransferase increased	15.0% 3/20	16.7% 2/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	33.3% 2/6	0.00% 0/7	0.00% 0/10	20.0% 2/10	0.00% 0/11	0.00% 0/4
Musculoskeletal and connective tissue disorders Back pain	5.0% 1/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Investigations Blood bilirubin increased	0.00% 0/20	0.00% 0/12	10.0% 1/10	20.0% 2/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	20.0% 2/10	18.2% 2/11	0.00% 0/4
Eye disorders Blurred vision	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Skin and subcutaneous tissue disorders Bullous dermatitis	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Infections and infestations Catheter related infection	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Musculoskeletal and connective tissue disorders Chest wall pain	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
General disorders Chills	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Gastrointestinal disorders Colitis	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Concentration impairment	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Psychiatric disorders Confusion	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Creatinine increased	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Dehydration	0.00% 0/20	8.3% 1/12	20.0% 2/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Gastrointestinal disorders Diarrhea	15.0% 3/20	8.3% 1/12	0.00% 0/10	20.0% 2/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	25.0% 1/4
Nervous system disorders Dizziness	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	14.3% 1/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Dyspepsia	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Dysphagia	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Dyspnea	5.0% 1/20	0.00% 0/12	0.00% 0/10	20.0% 2/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Ear and labyrinth disorders Ear pain	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Infections and infestations Enterocolitis infectious	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Epistaxis	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Psychiatric disorders Euphoria	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Eye disorders Eye pain	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
General disorders Fatigue	15.0% 3/20	8.3% 1/12	10.0% 1/10	20.0% 2/10	30.0% 3/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	9.1% 1/11	25.0% 1/4
Blood and lymphatic system disorders Febrile neutropenia	5.0% 1/20	8.3% 1/12	20.0% 2/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	9.1% 1/11	0.00% 0/4
General disorders Fever	0.00% 0/20	25.0% 3/12	10.0% 1/10	10.0% 1/10	20.0% 2/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Injury, poisoning and procedural complications Fracture	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
General disorders Gait disturbance	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Gastrointestinal disorders - Other, specify	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Musculoskeletal and connective tissue disorders Generalized muscle weakness	0.00% 0/20	0.00% 0/12	0.00% 0/10	20.0% 2/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Investigations GGT increased	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	30.0% 3/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Headache	5.0% 1/20	8.3% 1/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	12.5% 1/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Ear and labyrinth disorders Hearing impaired	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Metabolism and nutrition disorders Hypercalcemia	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Hyperglycemia	0.00% 0/20	0.00% 0/12	10.0% 1/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Hypermagnesemia	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Hypoalbuminemia	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	20.0% 2/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Hypocalcemia	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	20.0% 2/10	9.1% 1/11	0.00% 0/4
Metabolism and nutrition disorders Hypokalemia	5.0% 1/20	8.3% 1/12	20.0% 2/10	10.0% 1/10	10.0% 1/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	40.0% 4/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Hyponatremia	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Metabolism and nutrition disorders Hypophosphatemia	5.0% 1/20	16.7% 2/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	20.0% 2/10	18.2% 2/11	0.00% 0/4
Vascular disorders Hypotension	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	18.2% 2/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Hypoxia	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Infections and infestations Infections and infestations - Other, specify	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Injury, poisoning and procedural complications Injury, poisoning and procedural complications - Other, specify	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations INR increased	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Lethargy	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Lymphocyte count decreased	50.0% 10/20	33.3% 4/12	30.0% 3/10	50.0% 5/10	50.0% 5/10	37.5% 3/8	16.7% 1/6	14.3% 1/7	20.0% 2/10	40.0% 4/10	36.4% 4/11	0.00% 0/4
Investigations Lymphocyte count increased	0.00% 0/20	0.00% 0/12	10.0% 1/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	9.1% 1/11	0.00% 0/4
Infections and infestations Mucosal infection	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Mucositis oral	15.0% 3/20	33.3% 4/12	30.0% 3/10	10.0% 1/10	30.0% 3/10	12.5% 1/8	16.7% 1/6	14.3% 1/7	20.0% 2/10	10.0% 1/10	27.3% 3/11	0.00% 0/4
Gastrointestinal disorders Nausea	15.0% 3/20	0.00% 0/12	20.0% 2/10	20.0% 2/10	20.0% 2/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Investigations Neutrophil count decreased	75.0% 15/20	83.3% 10/12	60.0% 6/10	70.0% 7/10	40.0% 4/10	12.5% 1/8	33.3% 2/6	57.1% 4/7	70.0% 7/10	20.0% 2/10	54.5% 6/11	50.0% 2/4
Gastrointestinal disorders Oral pain	0.00% 0/20	8.3% 1/12	20.0% 2/10	10.0% 1/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	14.3% 1/7	10.0% 1/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Infections and infestations Otitis media	0.00% 0/20	8.3% 1/12	10.0% 1/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
General disorders Pain	0.00% 0/20	8.3% 1/12	10.0% 1/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	20.0% 2/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/20	8.3% 1/12	0.00% 0/10	20.0% 2/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	14.3% 1/7	0.00% 0/10	20.0% 2/10	0.00% 0/11	0.00% 0/4
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrome	5.0% 1/20	16.7% 2/12	10.0% 1/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Peripheral motor neuropathy	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	10.0% 1/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Peripheral sensory neuropathy	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Eye disorders Photophobia	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	20.0% 2/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Platelet count decreased	55.0% 11/20	41.7% 5/12	40.0% 4/10	40.0% 4/10	30.0% 3/10	12.5% 1/8	33.3% 2/6	28.6% 2/7	50.0% 5/10	70.0% 7/10	54.5% 6/11	25.0% 1/4
Respiratory, thoracic and mediastinal disorders Pleural effusion	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	14.3% 1/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Productive cough	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Renal and urinary disorders Proteinuria	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	16.7% 1/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Psychiatric disorders Psychiatric disorders - Other, specify	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Skin and subcutaneous tissue disorders Rash maculo-papular	5.0% 1/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Infections and infestations Scrotal infection	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Seizure	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	12.5% 1/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Serum amylase increased	0.00% 0/20	0.00% 0/12	10.0% 1/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Cardiac disorders Sinus tachycardia	0.00% 0/20	0.00% 0/12	10.0% 1/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Infections and infestations Skin infection	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Somnolence	5.0% 1/20	0.00% 0/12	0.00% 0/10	20.0% 2/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	10.0% 1/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Sore throat	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	12.5% 1/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Nervous system disorders Syncope	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Reproductive system and breast disorders Testicular hemorrhage	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Infections and infestations Upper respiratory infection	0.00% 0/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	33.3% 2/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Renal and urinary disorders Urinary retention	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Infections and infestations Urinary tract infection	5.0% 1/20	8.3% 1/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	12.5% 1/8	0.00% 0/6	14.3% 1/7	20.0% 2/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Skin and subcutaneous tissue disorders Urticaria	5.0% 1/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Gastrointestinal disorders Vomiting	5.0% 1/20	8.3% 1/12	0.00% 0/10	0.00% 0/10	10.0% 1/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations Weight gain	0.00% 0/20	0.00% 0/12	0.00% 0/10	0.00% 0/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	25.0% 1/4
Investigations Weight loss	0.00% 0/20	0.00% 0/12	0.00% 0/10	10.0% 1/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Respiratory, thoracic and mediastinal disorders Wheezing	0.00% 0/20	0.00% 0/12	0.00% 0/10	20.0% 2/10	0.00% 0/10	0.00% 0/8	0.00% 0/6	0.00% 0/7	0.00% 0/10	0.00% 0/10	0.00% 0/11	0.00% 0/4
Investigations White blood cell decreased	65.0% 13/20	66.7% 8/12	50.0% 5/10	70.0% 7/10	70.0% 7/10	0.00% 0/8	33.3% 2/6	57.1% 4/7	60.0% 6/10	40.0% 4/10	36.4% 4/11	50.0% 2/4

Additional Information

Results Reporting Coordinator

Children's Oncology Group

Phone: 626-447-0064

Email: resultsreportingcoordinator@childrensoncologygroup.org

Results disclosure agreements

• Principal investigator is a sponsor employee Must obtain prior Sponsor approval.
• Publication restrictions are in place

Restriction type: OTHER