Sorafenib Tosylate in Treating Younger Patients With Relapsed or Refractory Rhabdomyosarcoma, Wilms Tumor, Liver Cancer, or Thyroid Cancer
NCT ID: NCT01502410
Last Updated: 2018-06-26
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
20 participants
INTERVENTIONAL
2012-01-31
2014-06-30
Brief Summary
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Detailed Description
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I. To determine the objective response rate to sorafenib tosylate (sorafenib) in children with relapsed or refractory rhabdomyosarcoma, Wilms tumor, hepatocellular carcinoma (HCC), or papillary thyroid carcinoma (PTC).
SECONDARY OBJECTIVES:
I. To further define and describe the toxicities of sorafenib administered on an oral, twice-daily continuous schedule.
II. To further characterize the pharmacokinetics of sorafenib in children with refractory cancer.
III. To estimate the progression-free survival on sorafenib for rhabdomyosarcoma, Wilms tumor, and hepatocellular carcinoma and compare to a group of patients enrolled on selected closed Phase II studies of Children Oncology Group (COG).
IV. To assess the biologic activity of sorafenib on vascular endothelial growth factor (VEGF) and soluble vascular endothelial growth factor receptor-2 (VEGFR-2) in peripheral blood samples. (Exploratory) V. To evaluate the presence of BRAF mutations and RET/PTC rearrangements in patients with PTC. (Exploratory)
OUTLINE: This is a multicenter study. Patients are stratified according to diagnosis (rhabdomyosarcoma vs Wilms tumor vs hepatocellular carcinoma vs papillary thyroid carcinoma).
Patients receive sorafenib tosylate orally (PO) twice daily (BID) on days 1-28. Treatment repeats every 28 days for up to 24 courses in the absence of disease progression or unacceptable toxicity.
Patients undergo blood sample collection at baseline and periodically during study for pharmacokinetic studies, and VEGF and VEGFR-2 analysis by ELISA. Previously collected formalin-fixed paraffin-embedded tissue samples, from patients with papillary thyroid carcinoma, are also analyzed for BRAF mutation and RET/PTC rearrangements by PCR.
After completion of study treatment, patients are followed up for up to 5 years.
Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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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
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
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
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
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
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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
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* Patients with PTC must be refractory to radioactive iodine (RAI)
* Patient's current disease state must be one for which there is no known curative therapy or therapy proven to prolong survival with an acceptable quality of life
* Patients with known metastasis to the brain will be excluded from trial participation unless treated surgically or with radiotherapy and stable with no recurrent lesions for at least 3 months
* Rhabdomyosarcoma and Wilms strata: patients must be ≥ 24 months and ≤ 30 years of age at study enrollment
* Hepatocellular carcinoma (HCC): patients must be ≥ 24 months and \< 18 years of age at study enrollment
* Papillary thyroid carcinoma (PTC): patients must be ≥ 24 months and ≤ 21 years of age at study enrollment
* Patients must have a Lansky or Karnofsky performance status score of ≥ 50%, corresponding to ECOG categories 0, 1, or 2
* Use Karnofsky for patients \> 16 years of age and Lansky for patients ≤ 16 years of age
* Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
* Peripheral absolute neutrophil count (ANC) ≥ 1,000/μL
* Platelet count ≥ 75,000/μL (transfusion independent, defined as not receiving platelet transfusions within a 7-day period prior to enrollment)
* Hemoglobin 8.0 g/dL (may receive red blood cell\[RBC\] transfusions)
* Creatinine clearance or radioisotope glomerular filtration rate(GFR) 70 mL/min OR a serum creatinine based on age/gender as follows:
* 0.8 mg/dL (2 to \< 6 years of age)
* 1.0 mg/dL (6 to \< 10 years of age)
* 1.2 mg/dL (10 to \< 13 years of age)
* 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to \< 16 years of age)
* 1.7 mg/dL (male) or 1.4 mg/dL (female) (≥ 16 years of age)
* Total bilirubin ≤ 1.5 times upper limit of normal (ULN) for age
* SGPT (ALT) ≤ 135 U/L (for the purpose of this study, the ULN for SGPT is 45 U/L)
* PT, PTT, and INR \< 1.5 times ULN
* Normal serum lipase and amylase (per institutional normal values)
* No evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry \> 94% if there is clinical indication for determination
* A blood pressure (BP) ≤ the 95\^th percentile for age, height, and gender; and not receiving medication for treatment of hypertension
* Patients who are pregnant or breast-feeding are not eligible
* Negative pregnancy tests must be obtained in girls who are post-menarchal
* Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method beginning at the signing of the informed consent until at least 30 days after the last dose of the study drug
* Patients with clinical symptoms of hepatic encephalopathy or ascites are not eligible
* Patients who have an uncontrolled infection are not eligible
* Patients with evidence of bleeding diathesis are not eligible
* Patients with known Gilbert syndrome are not eligible
* Patients who, in the opinion of the investigator, may not be able to comply with the safety-monitoring requirements of the study are not eligible
* No concurrent chemotherapy, radiation therapy, immunomodulating agents, or other investigational agents
* Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study
* Patients with solid tumors must not have received myelosuppressive chemotherapy within 3 weeks of enrollment onto this study (6 weeks if prior nitrosourea)
* At least 7 days must have elapsed since the completion of therapy with a growth factor (at least 14 days must have elapsed after receiving pegfilgrastim)
* At least 7 days must have elapsed since completion of therapy with a biologic agent;
* For agents that have known adverse events occurring beyond 7 days after administration, this period prior to enrollment must be extended beyond the time during which adverse events are known to occur
* At least 3 half-lives must have elapsed since prior therapy that included a monoclonal antibody
* At least 2 weeks must have elapsed since local palliative radiotherapy (XRT) (small port); ≥ 3 months must have elapsed if prior craniospinal XRT was received, if ≥ 50% of the pelvis was irradiated, or if TBI was received; ≥ 6 weeks must have elapsed if other substantial bone marrow irradiation was given
* No evidence of active graft-vs-host disease and ≥ 2 months must have elapsed since transplant (stem cell transplant or rescue without total-body irradiation)
* For patients with papillary thyroid carcinoma (PTC) only: ≥ 3 weeks from prior radioiodine (RAI) treatment
* Patients requiring corticosteroids that have not been on a stable or decreasing dose of corticosteroid for 7 days prior to enrollment are not eligible
* Patients who are currently receiving another investigational drug are not eligible
* Patients who are currently receiving other anti-cancer agents are not eligible
* Patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post transplant are not eligible for this trial
* Patients who take cytochrome P450 enzyme-inducing anti-epileptic drugs (phenytoin, carbamazepine, or phenobarbital), rifampin, grapefruit juice, or St. Johns wort will not be eligible for the trial
* Patients who have received prior treatment with sorafenib are not eligible
* Patients must not be on therapeutic anti-coagulation;
* Prophylactic anticoagulation (i.e., low-dose warfarin) of venous or arterial devices is allowed provided that the requirements for prothrombin time(PT), partial thromboplastin time(PTT), and international normalized ratio(INR) are met
2 Years
30 Years
ALL
No
Sponsors
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National Cancer Institute (NCI)
NIH
Responsible Party
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Principal Investigators
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AeRang Kim, MD
Role: PRINCIPAL_INVESTIGATOR
Children's Oncology Group
Locations
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Children's Hospital of Alabama
Birmingham, Alabama, United States
University of Alabama at Birmingham
Birmingham, Alabama, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
Southern California Permanente Medical Group
Downey, California, United States
Miller Children's Hospital
Long Beach, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital Central California
Madera, California, United States
Childrens Hospital of Orange County
Orange, California, United States
Rady Children's Hospital - San Diego
San Diego, California, United States
University of California San Francisco Medical Center-Parnassus
San Francisco, California, United States
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
Denver, Colorado, United States
Connecticut Children's Medical Center
Hartford, Connecticut, United States
Alfred I duPont Hospital for Children
Wilmington, Delaware, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Lee Memorial Health System
Fort Myers, Florida, United States
Nemours Children's Clinic - Jacksonville
Jacksonville, Florida, United States
Florida Hospital
Orlando, Florida, United States
Nemours Children's Clinic - Orlando
Orlando, Florida, United States
Nemours Children's Clinic - Pensacola
Pensacola, Florida, United States
All Children's Hospital
St. Petersburg, Florida, United States
Saint Joseph Children's Hospital of Tampa
Tampa, Florida, United States
Children's Healthcare of Atlanta - Egleston
Atlanta, Georgia, United States
University of Hawaii
Honolulu, Hawaii, United States
Saint Luke's Mountain States Tumor Institute
Boise, Idaho, United States
Lurie Children's Hospital-Chicago
Chicago, Illinois, United States
University of Illinois
Chicago, Illinois, United States
University of Chicago
Chicago, Illinois, United States
Saint Jude Midwest Affiliate
Peoria, Illinois, United States
Southern Illinois University
Springfield, Illinois, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
University of Kentucky
Lexington, Kentucky, United States
Kosair Children's Hospital
Louisville, Kentucky, United States
Tulane University Health Sciences Center
New Orleans, Louisiana, United States
Sinai Hospital of Baltimore
Baltimore, Maryland, United States
Mark O Hatfield-Warren Grant Magnuson Clinical Center
Bethesda, Maryland, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
Dana-Farber Harvard Cancer Center
Boston, Massachusetts, United States
Wayne State University/Karmanos Cancer Institute
Detroit, Michigan, United States
Bronson Methodist Hospital
Kalamazoo, Michigan, United States
University of Minnesota Medical Center-Fairview
Minneapolis, Minnesota, United States
Mayo Clinic
Rochester, Minnesota, United States
University of Mississippi Medical Center
Jackson, Mississippi, United States
The Childrens Mercy Hospital
Kansas City, Missouri, United States
Washington University School of Medicine
St Louis, Missouri, United States
Children's Hospital and Medical Center of Omaha
Omaha, Nebraska, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
Morristown Memorial Hospital
Morristown, New Jersey, United States
UMDNJ - Robert Wood Johnson University Hospital
New Brunswick, New Jersey, United States
Overlook Hospital
Summit, New Jersey, United States
University of New Mexico Cancer Center
Albuquerque, New Mexico, United States
Roswell Park Cancer Institute
Buffalo, New York, United States
Columbia University Medical Center
New York, New York, United States
Memorial Sloan-Kettering Cancer Center
New York, New York, United States
University of Rochester
Rochester, New York, United States
State University of New York Upstate Medical University
Syracuse, New York, United States
Montefiore Medical Center - Moses Campus
The Bronx, New York, United States
University of North Carolina
Chapel Hill, North Carolina, United States
Carolinas Medical Center
Charlotte, North Carolina, United States
Novant Health Presbyterian Medical Center
Charlotte, North Carolina, United States
Wake Forest University Health Sciences
Winston-Salem, North Carolina, United States
Children's Hospital Medical Center of Akron
Akron, Ohio, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Rainbow Babies and Childrens Hospital
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Dayton Children's Hospital
Dayton, Ohio, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
Oregon Health and Science University
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Children's Oncology Group
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
BI-LO Charities Children's Cancer Center
Greenville, South Carolina, United States
Greenville Cancer Treatment Center
Greenville, South Carolina, United States
East Tennessee Childrens Hospital
Knoxville, Tennessee, United States
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, United States
Driscoll Children's Hospital
Corpus Christi, Texas, United States
Medical City Dallas Hospital
Dallas, Texas, United States
University of Texas Southwestern Medical Center
Dallas, Texas, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
Baylor College of Medicine
Houston, Texas, United States
Childrens Hospital-King's Daughters
Norfolk, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, United States
Midwest Children's Cancer Center
Milwaukee, Wisconsin, United States
Sydney Children's Hospital
Randwick, New South Wales, Australia
Princess Margaret Hospital for Children
Perth, Western Australia, Australia
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
IWK Health Centre
Halifax, Nova Scotia, Canada
McMaster Children's Hospital at Hamilton Health Sciences
Hamilton, Ontario, Canada
Hospital for Sick Children
Toronto, Ontario, Canada
The Montreal Children's Hospital of the MUHC
Montreal, Quebec, Canada
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
Centre Hospitalier Universitaire de Quebec
Ste-Foy, Quebec, Canada
Countries
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Other Identifiers
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NCI-2012-00106
Identifier Type: REGISTRY
Identifier Source: secondary_id
COG-ADVL1121
Identifier Type: OTHER
Identifier Source: secondary_id
CDR0000721611
Identifier Type: OTHER
Identifier Source: secondary_id
ADVL1121
Identifier Type: OTHER
Identifier Source: secondary_id
ADVL1121
Identifier Type: OTHER
Identifier Source: secondary_id
NCI-2012-00106
Identifier Type: -
Identifier Source: org_study_id
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