Medical Treatment of "High-Risk" Neurofibromas

Spectrum Health Hospitals

Status and phase: Completed; Phase 2

Conditions: Neurofibromatosis 1

Treatments: Drug: Temozolomide (temodar); Drug: Peg-Interferon alpha-2b; Drug: Celecoxib (Celebrex); Drug: Vincristine Sulfate (Oncovin)

Study type: Interventional

Funder types: Other

Identifiers

NCT00846430

2008-260

Details and patient eligibility

About

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.

Full 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.

Enrollment: 9 patients

Sex: All

Ages: 2 to 30 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 1 patient group

Open-Label Intervention

Experimental group

Description:

This is a phase II single arm study with sequential treatments available by response where all participants begin therapy with a combination of celecoxib and interferon alpha-2b (CI, treatment-1). Response to CI therapy will be assessed at six months by clinical and radiographic evaluations. Those patients who have achieved a partial response (improvement in pain, improvement in functioning, or ≥50% reduction in tumor size) or complete response (resolution of pain, and normalization of functioning with a ≥ 90% reduction in tumor size) will continue with the same CI therapy for up-to two years on study.

Treatment:

Drug: Peg-Interferon alpha-2b

Drug: Temozolomide (temodar)

Drug: Celecoxib (Celebrex)

Drug: Vincristine Sulfate (Oncovin)