Medical Treatment of "High-Risk" Neurofibromas

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

9 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-10-31

Study Completion Date

2017-05-31

Brief Summary

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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.

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Detailed Description

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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

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Neurofibromatosis 1

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Open-Label Intervention

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.

Group Type EXPERIMENTAL

Peg-Interferon alpha-2b

Intervention Type DRUG

age and weight dependant

Celecoxib (Celebrex)

Intervention Type DRUG

age and weight dependant

Temozolomide (temodar)

Intervention Type DRUG

age and weight dependant

Vincristine Sulfate (Oncovin)

Intervention Type DRUG

age and weight dependant

Interventions

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Peg-Interferon alpha-2b

age and weight dependant

Intervention Type DRUG

Celecoxib (Celebrex)

age and weight dependant

Intervention Type DRUG

Temozolomide (temodar)

age and weight dependant

Intervention Type DRUG

Vincristine Sulfate (Oncovin)

age and weight dependant

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

2 Years

Maximum Eligible Age

30 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No