Medical Treatment of "High-Risk" Neurofibromas
| Status: | Completed |
|---|---|
| Conditions: | Cancer, Other Indications |
| Therapuetic Areas: | Oncology, Other |
| Healthy: | No |
| Age Range: | 2 - 30 |
| Updated: | 7/11/2015 |
| Start Date: | October 2008 |
| End Date: | December 2010 |
| Contact: | Sara Finton, RN |
| Email: | sara.finton@spectrum-health.org |
| Phone: | 616-391-9365 |
Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies
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.
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.
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.
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.
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
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