Sorafenib Tosylate in Treating Younger Patients With Relapsed or Refractory Rhabdomyosarcoma, Wilms Tumor, Liver Cancer, or Thyroid Cancer
A Phase II Study of the Raf Kinase and Receptor Tyrosine Kinase Inhibitor Sorafenib in Children and Young Adults With Relapsed/Refractory Rhabdomyosarcoma, Wilms Tumor, Hepatocellular Carcinoma, and Papillary Thyroid Carcinoma
5 other identifiers
interventional
20
3 countries
92
Brief Summary
This phase II trial studies how well sorafenib tosylate works in treating younger patients with relapsed or refractory rhabdomyosarcoma, Wilms tumor, liver cancer, or thyroid cancer. Sorafenib tosylate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jan 2012
92 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 29, 2011
CompletedFirst Posted
Study publicly available on registry
December 30, 2011
CompletedStudy Start
First participant enrolled
January 1, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2014
CompletedResults Posted
Study results publicly available
May 15, 2015
CompletedJune 26, 2018
June 1, 2018
2.4 years
December 29, 2011
April 14, 2015
June 20, 2018
Conditions
Outcome Measures
Primary Outcomes (1)
Objective Response by RECIST Criteria v 1.1
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.
6 cycles (168 days)
Secondary Outcomes (5)
Progression-free Survival According to RECIST Version 1.1
Six months after enrollment
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
Pharmacokinetic (PK) Parameters of Sorafenib Tosylate
Prior to administration of Sorafenib (baseline), day 15, day 56, day 112 and day 168
Change in VEGF and VEGFR-2
Prior to the administration of sorafenib (baseline) and day 15 of protocol therapy
Presence of BRAF Mutation or RET/PTC Rearrangement
At baseline
Study Arms (4)
Group 1 Relapsed/Refractory Rhabdomyosarcoma
EXPERIMENTALPatients 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
EXPERIMENTALPatients 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
EXPERIMENTALPatients 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
EXPERIMENTALPatients 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
Interventions
Given PO dosage 200 mg/m2/dose (max dose:400 mg/dose) given every 12 hours on days 1-28
Optional correlative studies
Optional correlative studies
Eligibility Criteria
You may qualify if:
- Patients must have had histologic verification of one of the malignancies listed below at original diagnosis or at relapse:
- Rhabdomyosarcoma (RMS)
- Wilms tumor
- Hepatocellular carcinoma (HCC)
- Papillary thyroid carcinoma (PTC)
- Patients must have relapsed or refractory disease (RMS, Wilms tumor, HCC, PTC)
- Patients must have radiographically measurable disease; measurable disease is defined as the presence of at least one lesion on magnetic resonance imaging (MRI) or computed tomography (CT) scan that can be accurately measured with the longest diameter a minimum of 10 mm in at least one dimension (CT scan slice thickness no greater than 5 mm)
- The following do not qualify as measurable disease:
- Malignant fluid collections (e.g., ascites, pleural effusions)
- Bone marrow infiltration
- Lesions only detected by nuclear medicine studies (e.g., bone, gallium, or positron emission tomography \[PET\] scans)
- Elevated tumor markers in plasma or cerebrospinal fluid(CSF)
- Previously radiated lesions that have not demonstrated clear progression post radiation
- Leptomeningeal lesions that do not meet the requirements noted above
- Patients with HCC must be relapsed or refractory to conventional chemotherapy
- +50 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (92)
Children's Hospital of Alabama
Birmingham, Alabama, 35233, United States
University of Alabama at Birmingham
Birmingham, Alabama, 35294, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205, United States
Southern California Permanente Medical Group
Downey, California, 90242, United States
Miller Children's Hospital
Long Beach, California, 90806, United States
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Children's Hospital Central California
Madera, California, 93636-8762, United States
Childrens Hospital of Orange County
Orange, California, 92868-3874, United States
Rady Children's Hospital - San Diego
San Diego, California, 92123, United States
University of California San Francisco Medical Center-Parnassus
San Francisco, California, 94143, United States
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
Denver, Colorado, 80218, United States
Connecticut Children's Medical Center
Hartford, Connecticut, 06106, United States
Alfred I duPont Hospital for Children
Wilmington, Delaware, 19803, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
Lee Memorial Health System
Fort Myers, Florida, 33901, United States
Nemours Children's Clinic - Jacksonville
Jacksonville, Florida, 32207, United States
Florida Hospital
Orlando, Florida, 32803, United States
Nemours Children's Clinic - Orlando
Orlando, Florida, 32806, United States
Nemours Children's Clinic - Pensacola
Pensacola, Florida, 32504, United States
All Children's Hospital
St. Petersburg, Florida, 33701, United States
Saint Joseph Children's Hospital of Tampa
Tampa, Florida, 33607, United States
Children's Healthcare of Atlanta - Egleston
Atlanta, Georgia, 30322, United States
University of Hawaii
Honolulu, Hawaii, 96813, United States
Saint Luke's Mountain States Tumor Institute
Boise, Idaho, 83712, United States
Lurie Children's Hospital-Chicago
Chicago, Illinois, 60611, United States
University of Illinois
Chicago, Illinois, 60612, United States
University of Chicago
Chicago, Illinois, 60637, United States
Saint Jude Midwest Affiliate
Peoria, Illinois, 61602, United States
Southern Illinois University
Springfield, Illinois, 62702, United States
Riley Hospital for Children
Indianapolis, Indiana, 46202, United States
University of Kentucky
Lexington, Kentucky, 40536, United States
Kosair Children's Hospital
Louisville, Kentucky, 40202, United States
Tulane University Health Sciences Center
New Orleans, Louisiana, 70112, United States
Sinai Hospital of Baltimore
Baltimore, Maryland, 21215, United States
Mark O Hatfield-Warren Grant Magnuson Clinical Center
Bethesda, Maryland, 20892, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, 02115, United States
Dana-Farber Harvard Cancer Center
Boston, Massachusetts, 02115, United States
Wayne State University/Karmanos Cancer Institute
Detroit, Michigan, 48201, United States
Bronson Methodist Hospital
Kalamazoo, Michigan, 49007, United States
University of Minnesota Medical Center-Fairview
Minneapolis, Minnesota, 55455, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
University of Mississippi Medical Center
Jackson, Mississippi, 39216, United States
The Childrens Mercy Hospital
Kansas City, Missouri, 64108, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
Children's Hospital and Medical Center of Omaha
Omaha, Nebraska, 68114, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Morristown Memorial Hospital
Morristown, New Jersey, 07962, United States
UMDNJ - Robert Wood Johnson University Hospital
New Brunswick, New Jersey, 08903, United States
Overlook Hospital
Summit, New Jersey, 07902, United States
University of New Mexico Cancer Center
Albuquerque, New Mexico, 87106, United States
Roswell Park Cancer Institute
Buffalo, New York, 14263, United States
Columbia University Medical Center
New York, New York, 10032, United States
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065, United States
University of Rochester
Rochester, New York, 14642, United States
State University of New York Upstate Medical University
Syracuse, New York, 13210, United States
Montefiore Medical Center - Moses Campus
The Bronx, New York, 10467-2490, United States
University of North Carolina
Chapel Hill, North Carolina, 27599, United States
Carolinas Medical Center
Charlotte, North Carolina, 28203, United States
Novant Health Presbyterian Medical Center
Charlotte, North Carolina, 28204, United States
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157, United States
Children's Hospital Medical Center of Akron
Akron, Ohio, 44308, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Rainbow Babies and Childrens Hospital
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Dayton Children's Hospital
Dayton, Ohio, 45404, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Oncology Group
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
BI-LO Charities Children's Cancer Center
Greenville, South Carolina, 29605, United States
Greenville Cancer Treatment Center
Greenville, South Carolina, 29605, United States
East Tennessee Childrens Hospital
Knoxville, Tennessee, 37916, United States
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, 37232, United States
Driscoll Children's Hospital
Corpus Christi, Texas, 78411, United States
Medical City Dallas Hospital
Dallas, Texas, 75230, United States
University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Childrens Hospital-King's Daughters
Norfolk, Virginia, 23507, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, 99204, United States
Midwest Children's Cancer Center
Milwaukee, Wisconsin, 53226, United States
Sydney Children's Hospital
Randwick, New South Wales, 2031, Australia
Princess Margaret Hospital for Children
Perth, Western Australia, 6008, Australia
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4, Canada
IWK Health Centre
Halifax, Nova Scotia, B3J 3G9, Canada
McMaster Children's Hospital at Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5, Canada
Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
The Montreal Children's Hospital of the MUHC
Montreal, Quebec, H3H 1P3, Canada
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5, Canada
Centre Hospitalier Universitaire de Quebec
Ste-Foy, Quebec, G1V 4G2, Canada
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Results Reporting Coordinator
- Organization
- Children's Oncology Group
Study Officials
- PRINCIPAL INVESTIGATOR
AeRang Kim, MD
Children's Oncology Group
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- NIH
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 29, 2011
First Posted
December 30, 2011
Study Start
January 1, 2012
Primary Completion
June 1, 2014
Study Completion
June 1, 2014
Last Updated
June 26, 2018
Results First Posted
May 15, 2015
Record last verified: 2018-06