Medical Treatment of "High-Risk" Neurofibromas
This trial is active, not recruiting.
|Treatments||peg-interferon alpha-2b, celecoxib (celebrex), temozolomide (temodar), vincristine sulfate (oncovin)|
|Sponsor||Spectrum Health Hospitals|
|Start date||October 2008|
|End date||October 2017|
|Trial size||20 participants|
|Trial identifier||NCT00846430, 2008-260|
Patients with neurofibromatosis type 1 (NF1) commonly develop non-cancerous tumors called plexiform neurofibromas. These tumors can be defined as "high-risk" when they result in severe pain, physical disability, organ dysfunction and/or become life-threatening. Presently, there is no effective medical therapy to offer patients with "high-risk" plexiform neurofibromas, and surgery does not provide lasting help. This study will evaluate the effectiveness of two treatment combinations in patients with "high-risk" plexiform neurofibromas.
|Endpoint classification||safety/efficacy study|
|Intervention model||crossover assignment|
Tumor response based on evaluation of symptom assessment, tumor measurements, and MRI studies - Toxicity of treatment combinations based upon laboratory studies and physical examination
time frame: Monthly physical exam first three months and then every three months after, MRI's will occur at baseline, 6, 12 and 24 months.
Psychological toxicity based upon psychological evaluations - Improved quality of life based upon physical examination and performance scales
time frame: Psychological evaluation at baseline, 3, 12, and 24 months
Male or female participants from 2 years up to 30 years old.
Inclusion Criteria: - "High-Risk" Plexiform Neurofibromas associated with a diagnosis of NF1 - 2-30 years old (minimum bodyweight of 10 kilograms) - Adequate renal function Exclusion Criteria: - Previously untreated active optic glioma - History of any previous allergy to study medications - History of ischemic vascular disease - Pregnancy / Breast feeding
|Official title||Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies|
|Principal investigator||Albert S Cornelius, MD|
|Description||The study's design involves treating eligible patients with a combination of celecoxib and pegylated interferon alpha-2b. If the patients have at least a partial response after six months, they may continue on the same treatment for up to two years. If the patient experiences less than a partial response, or has progressive disease after six months of therapy, then vincristine and temozolomide will be added to the celecoxib and interferon alpha-2b backbone. Response to treatment will be assessed after a minimum of six months, presuming the patient has not experienced progressive disease. Total duration of therapy on study is two years for any individual treatment plan.|
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