NCT00846430

Medical Treatment of "High-Risk" Neurofibromas

Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Corewell Health West · Academic / Other
• Sex: All
• Age: 2 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed 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.

Conditions

• Neurofibromatosis 1

Interventions

Type	Name	Description
DRUG	Peg-Interferon alpha-2b	age and weight dependant
DRUG	Celecoxib (Celebrex)	age and weight dependant
DRUG	Temozolomide (temodar)	age and weight dependant
DRUG	Vincristine Sulfate (Oncovin)	age and weight dependant

Timeline

• Start date: 2008-10-01
• Primary completion: 2017-04-01
• Completion: 2017-05-01
• First posted: 2009-02-18
• Last updated: 2023-08-30
• Results posted: 2023-08-30

Locations

1 site across 1 country: United States