Trial Outcomes & Findings for Sorafenib Tosylate in Treating Younger Patients With Relapsed or Refractory Rhabdomyosarcoma, Wilms Tumor, Liver Cancer, or Thyroid Cancer (NCT NCT01502410)

Last Updated: 2018-06-26

Results Overview

Response rates will be calculated as the number of evaluable patients who are responders. Response Evaluation Criteria In Solid Tumors (RECIST) criteria: Complete Response (CR): Disappearance of all target lesions, Partial Response (PR): At least a 30% decrease in the sum of the LD of target lesions, taking as reference the baseline sum LD, Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum LD since the treatment started and Progressive Disease (PD): At least a 20% increase in the sum of the LD of target lesions, taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

20 participants

Primary outcome timeframe

6 cycles (168 days)

Results posted on

2018-06-26

Participant Flow

Only Rhabdomyosarcoma and Wilms Tumor patients are reported as there were no accruals on Hepatocellular carcinoma and no accruals on Thyroid carcinoma.

Participant milestones

Participant milestones
Measure	Group 1 Relapsed/Refractory Rhabdomyosarcoma Patients with relapsed or refractory rhabdomyosarcoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Group 2 Relapsed/Refractory Wilms Tumor Patients with relapsed or refractory Wilms tumor receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies
Overall Study STARTED	10	10
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	10	10

Reasons for withdrawal

Reasons for withdrawal
Measure	Group 1 Relapsed/Refractory Rhabdomyosarcoma Patients with relapsed or refractory rhabdomyosarcoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Group 2 Relapsed/Refractory Wilms Tumor Patients with relapsed or refractory Wilms tumor receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies
Overall Study Adverse Event	1	2
Overall Study Lack of Efficacy	7	7
Overall Study Physician Decision	0	1
Overall Study Withdrawal by Subject	2	0

Baseline Characteristics

Sorafenib Tosylate in Treating Younger Patients With Relapsed or Refractory Rhabdomyosarcoma, Wilms Tumor, Liver Cancer, or Thyroid Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Group 1 Relapsed/Refractory Rhabdomyosarcoma n=10 Participants Patients with relapsed or refractory rhabdomyosarcoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Group 2 Relapsed/Refractory Wilms Tumor n=10 Participants Patients with relapsed or refractory Wilms tumor receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Group 3 Relapsed/Refractory Hepatocellular Carcinoma Patients with relapsed or refractory hepatocellular carcinoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Group 4 Papillary Thyroid Carcinoma Patients with relapsed or refractory papillary thyroid carcinoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Total n=20 Participants Total of all reporting groups
Age, Categorical <=18 years	7 Participants n=5 Participants	10 Participants n=7 Participants	—	—	17 Participants n=21 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants	0 Participants n=7 Participants	—	—	3 Participants n=21 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Age, Continuous	12 years n=5 Participants	10.5 years n=7 Participants	—	—	11 years n=21 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	5 Participants n=7 Participants	—	—	9 Participants n=21 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	5 Participants n=7 Participants	—	—	11 Participants n=21 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	2 Participants n=7 Participants	—	—	2 Participants n=21 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	10 Participants n=5 Participants	8 Participants n=7 Participants	—	—	18 Participants n=21 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	—	—	1 Participants n=21 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants	0 Participants n=7 Participants	—	—	3 Participants n=21 Participants
Race (NIH/OMB) White	7 Participants n=5 Participants	7 Participants n=7 Participants	—	—	14 Participants n=21 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	—	—	0 Participants n=21 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	2 Participants n=7 Participants	—	—	2 Participants n=21 Participants
Region of Enrollment United States	9 participants n=5 Participants	10 participants n=7 Participants	—	—	19 participants n=21 Participants
Region of Enrollment Canada	1 participants n=5 Participants	0 participants n=7 Participants	—	—	1 participants n=21 Participants

PRIMARY outcome

Timeframe: 6 cycles (168 days)

Population: Only Rhabdomyosarcoma and Wilms Tumor patients are included in this analysis as there were no accruals on Hepatocellular carcinoma and no accruals on Thyroid carcinoma.

Outcome measures

Outcome measures
Measure	Group 1 Relapsed/Refractory Rhabdomyosarcoma n=10 Participants Patients with relapsed or refractory rhabdomyosarcoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Group 2 Relapsed/Refractory Wilms Tumor n=10 Participants Patients with relapsed or refractory Wilms tumor receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies
Objective Response by RECIST Criteria v 1.1 Responder	0 participants	0 participants
Objective Response by RECIST Criteria v 1.1 Non-Responder	10 participants	10 participants

SECONDARY outcome

Timeframe: Six months after enrollment

Population: All eligible patients are included in this analysis for both Rhabdomyosarcoma and Wilms Tumor. However, there were no accruals on the disease arms for Hepatocellular carcinoma and Papillary Thyroid carcinoma and were not included in this analysis.

Percent probability of being progression free six months following enrollment. Progression-free interval (PFI) will be calculated as the date of enrollment until the end PFI date, where that date is calculated as the date of disease progression, date of death, date of removal of all tumors by surgery or last patient contact, whichever occurs first.

Outcome measures

Outcome measures
Measure	Group 1 Relapsed/Refractory Rhabdomyosarcoma n=10 Participants Patients with relapsed or refractory rhabdomyosarcoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Group 2 Relapsed/Refractory Wilms Tumor n=10 Participants Patients with relapsed or refractory Wilms tumor receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies
Progression-free Survival According to RECIST Version 1.1	10 percentage of probability Interval 0.57 to 36.0	23 percentage of probability Interval 3.5 to 52.0

SECONDARY outcome

Timeframe: six cycles of chemotherapy; expected to be 126 days of treatment

Population: All eligible patients diagnosed with Rhabdomyosarcoma or Wilms Tumor are combined in this analysis.

Each patient is classified as having experienced grade 3 or higher CTC version 4 toxicity if at any time during protocol therapy such an event is observed for the individual.

Outcome measures

Outcome measures
Measure	Group 1 Relapsed/Refractory Rhabdomyosarcoma n=20 Participants Patients with relapsed or refractory rhabdomyosarcoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Group 2 Relapsed/Refractory Wilms Tumor Patients with relapsed or refractory Wilms tumor receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies
The Number of Patients Who Experience at Least One Grade 3 or Higher CTC Version 4 Toxicity,	14 Participants	—

SECONDARY outcome

Timeframe: Prior to administration of Sorafenib (baseline), day 15, day 56, day 112 and day 168

Population: Baseline sample was available for only 9 participants.

The trough sorafenib concentration is evaluated at baseline (prior to administration of Sorafenib) and 12 hours after administration of Sorafenib on day 15, day 56, day 112 and day 168 in micrograms/ml.

Outcome measures

Outcome measures
Measure	Group 1 Relapsed/Refractory Rhabdomyosarcoma n=9 Participants Patients with relapsed or refractory rhabdomyosarcoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Group 2 Relapsed/Refractory Wilms Tumor Patients with relapsed or refractory Wilms tumor receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies
Pharmacokinetic (PK) Parameters of Sorafenib Tosylate Baseline	0 micrograms/ml Interval 0.0 to 0.0	—
Pharmacokinetic (PK) Parameters of Sorafenib Tosylate Day 15 of protocol therapy	6.51 micrograms/ml Interval 0.09 to 13.64	—
Pharmacokinetic (PK) Parameters of Sorafenib Tosylate Day 56 of protocol therapy	6.99 micrograms/ml Interval 3.33 to 10.1	—
Pharmacokinetic (PK) Parameters of Sorafenib Tosylate Day 112 of protocol therapy	4.10 micrograms/ml Interval 4.1 to 4.1	—
Pharmacokinetic (PK) Parameters of Sorafenib Tosylate Day 168 of protocol therapy	5.20 micrograms/ml Interval 5.2 to 5.2	—

SECONDARY outcome

Timeframe: Prior to the administration of sorafenib (baseline) and day 15 of protocol therapy

Population: Change in VEGF and VEGFR-2 are the responses of the patient's organs to the drug. As such, these measures are unrelated to the particular disease with which the patient was diagnosed, and were combined for analysis.

Serum VEGF and VEGF receptor 2 Concentration is evaluated at baseline and at day 15 of protocol therapy in picograms/ml.

Outcome measures

Outcome measures
Measure	Group 1 Relapsed/Refractory Rhabdomyosarcoma n=9 Participants Patients with relapsed or refractory rhabdomyosarcoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies	Group 2 Relapsed/Refractory Wilms Tumor Patients with relapsed or refractory Wilms tumor receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity. sorafenib tosylate: Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28 pharmacological study: Optional correlative studies laboratory biomarker analysis: Optional correlative studies
Change in VEGF and VEGFR-2 VEGF Baseline	155.75 picograms/ml Interval 28.01 to 452.22	—
Change in VEGF and VEGFR-2 VEGF Day 15	209.92 picograms/ml Interval 70.1 to 470.38	—
Change in VEGF and VEGFR-2 VEGF-R2 Baseline	9594.04 picograms/ml Interval 651.8 to 15576.39	—
Change in VEGF and VEGFR-2 VEG-R2 Day 15	7948.48 picograms/ml Interval 5569.73 to 11637.18	—

SECONDARY outcome

Timeframe: At baseline

Population: No patients were enrolled with PTC (papillary thyroid carcinoma) so there are no patients who can contribute to this outcome measure.

Descriptive statistics including mean, median, standard deviation, and range will be calculated for baseline for patients with PTC, TG and TG antibody, and presence of BRAF mutation or RET/PTC rearrangement.

Outcome measures

Outcome data not reported

Adverse Events

Group 1 Relapsed/Refractory Rhabdomyosarcoma

Serious events: 4 serious events

Other events: 5 other events

Deaths: 0 deaths

Group 2 Relapsed/Refractory Wilms Tumor

Serious events: 3 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Group 1 Relapsed/Refractory Rhabdomyosarcoma n=10 participants at risk Patients with relapsed or refractory rhabdomyosarcoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity.	Group 2 Relapsed/Refractory Wilms Tumor n=10 participants at risk Patients with relapsed or refractory Wilms tumor receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity.
Metabolism and nutrition disorders Anorexia	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Musculoskeletal and connective tissue disorders Back pain	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
General disorders Fatigue	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue disorder - Other	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Musculoskeletal and connective tissue disorders Neck pain	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
General disorders Pain	20.0% 2/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Cardiac disorders Pericardial effusion	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumor pain	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.

Other adverse events

Additional Information

Results Reporting Coordinator

Children's Oncology Group

Phone: 352-273-0558

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60

Measure	Group 1 Relapsed/Refractory Rhabdomyosarcoma n=10 participants at risk Patients with relapsed or refractory rhabdomyosarcoma receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity.	Group 2 Relapsed/Refractory Wilms Tumor n=10 participants at risk Patients with relapsed or refractory Wilms tumor receive sorafenib tosylate PO BID on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity.
Gastrointestinal disorders Abdominal pain	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Investigations Alkaline phosphatase increased	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Respiratory, thoracic and mediastinal disorders Atelectasis	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Investigations Blood bilirubin increased	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Gastrointestinal disorders Diarrhea	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Respiratory, thoracic and mediastinal disorders Dyspnea	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	20.0% 2/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Metabolism and nutrition disorders Hypoalbuminemia	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Metabolism and nutrition disorders Hypocalcemia	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Metabolism and nutrition disorders Hypokalemia	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Metabolism and nutrition disorders Hypophosphatemia	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Investigations Lipase increased	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Infections and infestations Lung infection	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Investigations Lymphocyte count decreased	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Investigations Neutrophil count decreased	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Infections and infestations Otitis externa	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
General disorders Pain	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrome	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Investigations Platelet count decreased	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Respiratory, thoracic and mediastinal disorders Pleural effusion	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Renal and urinary disorders Proteinuria	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Skin and subcutaneous tissue disorders Rash maculo-papular	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Respiratory, thoracic and mediastinal disorders Respiratory, thoracic and mediastinal disorders - Other	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Nervous system disorders Seizure	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.
Infections and infestations Upper respiratory infection	0.00% 0/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.	10.0% 1/10 SAE field contains NCI Common Terminology Criteria for Adverse Events (CTCAEs) submitted via expedited reporting (NCI AdEERs / CAeRs). The AE field contains grade 3 and higher CTCAEs reported on study excluding those that were reported as SAEs. Only Grade 3 and higher Adverse Events were collected/assessed in this study.