Home
Search
Study Topics
Glossary
|
|
|
|
|
|
Sponsored by: |
National Cancer Institute (NCI) |
---|---|
Information provided by: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT00727233 |
RATIONALE: Sorafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.
PURPOSE: This phase I trial is studying the side effects and best dose of sorafenib in treating young patients with neurofibromatosis type 1 and plexiform neurofibromas that cannot be removed by surgery.
Condition | Intervention | Phase |
---|---|---|
Neurofibromatosis Type 1 (nf1) Precancerous/Nonmalignant Condition |
Drug: sorafenib tosylate |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment |
Official Title: | Phase I Trial of the Raf Kinase and Receptor Tyrosine Kinase Inhibitor Sorafenib (BAY 43-9006, Nexavar®) in Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas |
Estimated Enrollment: | 30 |
Study Start Date: | July 2008 |
Estimated Primary Completion Date: | December 2010 (Final data collection date for primary outcome measure) |
OBJECTIVES:
Primary
Secondary
OUTLINE: This is a multicenter study.
Patients receive oral sorafenib tosylate twice daily on days 1-28. Patients with no documented disease progression or maximum radiographic response within the past 2 years continue treatment for up to 6 months in the absence of disease progression or unacceptable toxicity. All other patients continue treatment for as long as benefit is shown.
After completion of study therapy, patients are followed at 30 days.
Ages Eligible for Study: | 3 Years to 18 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
DISEASE CHARACTERISTICS:
Histologically confirmed* neurofibromatosis 1 (NF1) and plexiform neurofibromas (PNs) that are inoperable AND have the potential to cause significant morbidity including, but not limited to, any of the following:
Must have either positive genetic testing for NF1 OR at least one other diagnostic criterion (according to NIH Consensus conference criteria), including any of the following:
Measurable disease, defined as ≥ 1 unidimensionally measurable PN ≥ 3 cm
PATIENT CHARACTERISTICS:
Creatinine clearance or radioisotope glomerular filtration rate ≥ 60mL/ min OR serum creatinine normal based on age as follows:
PRIOR CONCURRENT THERAPY:
At least 7 days since prior and no concurrent cytochrome P450 enzyme-inducing antiepileptic drugs, including any of the following:
United States, Alabama | |
Lurleen Wallace Comprehensive Cancer at University of Alabama - Birmingham | Recruiting |
Birmingham, Alabama, United States, 35294 | |
Contact: Clinical Trials Office - Lurleen Wallace Comprehensive Cancer 205-934-0309 | |
United States, Maryland | |
Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office | Recruiting |
Bethesda, Maryland, United States, 20892-1182 | |
Contact: Clinical Trials Office - Warren Grant Magnusen Clinical Center 888-NCI-1937 |
Principal Investigator: | Brigitte C. Widemann, MD | NCI - Pediatric Oncology Branch |
Study ID Numbers: | CDR0000601122, NCI-08-C-0180, NCI-P06074 |
Study First Received: | July 31, 2008 |
Last Updated: | January 14, 2009 |
ClinicalTrials.gov Identifier: | NCT00727233 |
Health Authority: | Unspecified |
neurofibromatosis type 1 (NF1) plexiform neurofibroma |
Precancerous Conditions Neurodegenerative Diseases Neurofibromatosis type 1 Neurofibromatosis 1 Neoplastic Syndromes, Hereditary Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn Neurofibroma Neuromuscular Diseases |
Peripheral Nervous System Diseases Neurofibromatoses Peripheral Nervous System Neoplasms Neurofibroma, Plexiform Sorafenib Nerve Sheath Neoplasms Nervous System Neoplasms Neurocutaneous Syndromes |
Neoplasms Neoplasms by Histologic Type Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Therapeutic Uses |
Nervous System Diseases Neoplasms, Nerve Tissue Enzyme Inhibitors Protein Kinase Inhibitors Pharmacologic Actions |