Trial · NCT01502410

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

NCT01502410, Phase 2, Completed, National Cancer Institute (NCI); results first posted 2015-05-15

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Sorafenib Tosylate in Treating Younger Patients With Relapsed or Refractory Rhabdomyosarcoma, Wilms Tumor, Liver Cancer, or Thyroid Cancer. Trials, record NCT01502410. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01502410

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Sorafenib Tosylate in Treating Younger Patients With Relapsed or Refractory Rhabdomyosarcoma, Wilms Tumor, Liver Cancer, or Thyroid Cancer
NCT number: NCT01502410
Status: Completed
Phases: Phase 2
Enrollment: 20 participants, actual
Lead sponsor: National Cancer Institute (NCI)
Conditions: Childhood Hepatocellular Carcinoma, Papillary Thyroid Cancer, Previously Treated Childhood Rhabdomyosarcoma, Recurrent Childhood Liver Cancer, Recurrent Childhood Rhabdomyosarcoma, Recurrent Thyroid Cancer, Recurrent Wilms Tumor and Other Childhood Kidney Tumors
Interventions: sorafenib tosylate, pharmacological study, laboratory biomarker analysis
First posted: 2011-12-30
Last updated: 2018-06-26
Results first posted: 2015-05-15

Source ClinicalTrials.gov

Registered outcomes6

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Objective Response by RECIST Criteria v 1.1	6 cycles (168 days)	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.
Secondary	Progression-free Survival According to RECIST Version 1.1	Six months after enrollment	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.
Secondary	The Number of Patients Who Experience at Least One Grade 3 or Higher CTC Version 4 Toxicity,	six cycles of chemotherapy; expected to be 126 days of treatment	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.
Secondary	Pharmacokinetic (PK) Parameters of Sorafenib Tosylate	Prior to administration of Sorafenib (baseline), day 15, day 56, day 112 and day 168	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.
Secondary	Change in VEGF and VEGFR-2	Prior to the administration of sorafenib (baseline) and day 15 of protocol therapy	Serum VEGF and VEGF receptor 2 Concentration is evaluated at baseline and at day 15 of protocol therapy in picograms/ml.
Secondary	Presence of BRAF Mutation or RET/PTC Rearrangement	At baseline	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.

Source ClinicalTrials.gov

Outcomes6

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Objective Response by RECIST Criteria v 1.1	6 cycles (168 days)	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.	participants	NUMBER
SECONDARY	Progression-free Survival According to RECIST Version 1.1	Six months after enrollment	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.	percentage of probability	NUMBER
SECONDARY	The Number of Patients Who Experience at Least One Grade 3 or Higher CTC Version 4 Toxicity,	six cycles of chemotherapy; expected to be 126 days of treatment	All eligible patients diagnosed with Rhabdomyosarcoma or Wilms Tumor are combined in this analysis.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Pharmacokinetic (PK) Parameters of Sorafenib Tosylate	Prior to administration of Sorafenib (baseline), day 15, day 56, day 112 and day 168	Baseline sample was available for only 9 participants.	micrograms/ml	MEAN
SECONDARY	Change in VEGF and VEGFR-2	Prior to the administration of sorafenib (baseline) and day 15 of protocol therapy	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.	picograms/ml	MEAN
SECONDARY	Presence of BRAF Mutation or RET/PTC Rearrangement	At baseline	No patients were enrolled with PTC (papillary thyroid carcinoma) so there are no patients who can contribute to this outcome measure.

Source ClinicalTrials.gov

Results by group16

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Objective Response by RECIST Criteria v 1.1	Group 1 Relapsed/Refractory Rhabdomyosarcoma	Non-Responder	10		participants
Objective Response by RECIST Criteria v 1.1	Group 2 Relapsed/Refractory Wilms Tumor	Non-Responder	10		participants
Objective Response by RECIST Criteria v 1.1	Group 1 Relapsed/Refractory Rhabdomyosarcoma	Responder	0		participants
Objective Response by RECIST Criteria v 1.1	Group 2 Relapsed/Refractory Wilms Tumor	Responder	0		participants
Progression-free Survival According to RECIST Version 1.1	Group 1 Relapsed/Refractory Rhabdomyosarcoma		10	95% Confidence Interval 0.57 to 36	percentage of probability
Progression-free Survival According to RECIST Version 1.1	Group 2 Relapsed/Refractory Wilms Tumor		23	95% Confidence Interval 3.5 to 52	percentage of probability
The Number of Patients Who Experience at Least One Grade 3 or Higher CTC Version 4 Toxicity,	Rhabdomyosarcoma and Wilms Tumor		14		Participants
Pharmacokinetic (PK) Parameters of Sorafenib Tosylate	Rhabdomyosarcoma and Wilms Tumor	Baseline	0	Full Range 0 to 0	micrograms/ml
Pharmacokinetic (PK) Parameters of Sorafenib Tosylate	Rhabdomyosarcoma and Wilms Tumor	Day 15 of protocol therapy	6.51	Full Range 0.09 to 13.64	micrograms/ml
Pharmacokinetic (PK) Parameters of Sorafenib Tosylate	Rhabdomyosarcoma and Wilms Tumor	Day 56 of protocol therapy	6.99	Full Range 3.33 to 10.10	micrograms/ml

1 to 10 of 16Source ClinicalTrials.gov

See all 16 results by group

Participant flow12

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Group 1 Relapsed/Refractory Rhabdomyosarcoma	10
Overall Study	STARTED	Group 2 Relapsed/Refractory Wilms Tumor	10
Overall Study	STARTED	Group 3 Relapsed/Refractory Hepatocellular Carcinoma	0
Overall Study	STARTED	Group 4 Papillary Thyroid Carcinoma	0
Overall Study	COMPLETED	Group 1 Relapsed/Refractory Rhabdomyosarcoma	0
Overall Study	COMPLETED	Group 2 Relapsed/Refractory Wilms Tumor	0
Overall Study	COMPLETED	Group 3 Relapsed/Refractory Hepatocellular Carcinoma	0
Overall Study	COMPLETED	Group 4 Papillary Thyroid Carcinoma	0
Overall Study	NOT COMPLETED	Group 1 Relapsed/Refractory Rhabdomyosarcoma	10
Overall Study	NOT COMPLETED	Group 2 Relapsed/Refractory Wilms Tumor	10

1 to 10 of 12Source ClinicalTrials.gov

See all 12 participant flow

Adverse events72

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Abdominal pain	Gastrointestinal disorders	Group 1 Relapsed/Refractory Rhabdomyosarcoma	0	10
other	Abdominal pain	Gastrointestinal disorders	Group 2 Relapsed/Refractory Wilms Tumor	1	10
other	Alkaline phosphatase increased	Investigations	Group 1 Relapsed/Refractory Rhabdomyosarcoma	1	10
other	Alkaline phosphatase increased	Investigations	Group 2 Relapsed/Refractory Wilms Tumor	0	10
other	Atelectasis	Respiratory, thoracic and mediastinal disorders	Group 1 Relapsed/Refractory Rhabdomyosarcoma	1	10
other	Atelectasis	Respiratory, thoracic and mediastinal disorders	Group 2 Relapsed/Refractory Wilms Tumor	0	10
other	Blood bilirubin increased	Investigations	Group 1 Relapsed/Refractory Rhabdomyosarcoma	1	10
other	Blood bilirubin increased	Investigations	Group 2 Relapsed/Refractory Wilms Tumor	0	10
other	Diarrhea	Gastrointestinal disorders	Group 1 Relapsed/Refractory Rhabdomyosarcoma	1	10
other	Diarrhea	Gastrointestinal disorders	Group 2 Relapsed/Refractory Wilms Tumor	0	10

1 to 10 of 72Source ClinicalTrials.gov

See all 72 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Group 1 Relapsed/Refractory Rhabdomyosarcoma
Total, all-cause mortality	Group 2 Relapsed/Refractory Wilms Tumor
Total, other adverse events	Group 1 Relapsed/Refractory Rhabdomyosarcoma	5	10
Total, other adverse events	Group 2 Relapsed/Refractory Wilms Tumor	6	10
Total, serious adverse events	Group 1 Relapsed/Refractory Rhabdomyosarcoma	4	10
Total, serious adverse events	Group 2 Relapsed/Refractory Wilms Tumor	3	10

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants1

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

Project	Title	Principal investigators	Organization	Funder	Fiscal years
U10CA098543	Children's Oncology Group Chair's Grant	ADAMSON, PETER C.	CHILDREN'S HOSP OF PHILADELPHIA	NATIONAL CANCER INSTITUTE	2003 to 2013

Source NIH RePORTER

Trial comparisons1

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison
Group 1 Relapsed/Refractory Rhabdomyosarcoma vs Group 2 Relapsed/Refractory Wilms Tumor: serious adverse events

Source Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT01502410

GET https://conductscience.org/api/trials/v1/NCT01502410/events?page=2

API reference For AI assistants