Clinical trial NCT00846430: Medical Treatment of "High-Risk" Neurofibromas
Official title: Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Neurofibromatosis 1
Keywords: Neurofibromas
Neurofibromatosis 1
Celecoxib
Celebrex
Peg-Interferon alpha-2b
PEG-Intron
Temozolomide
Temodar
Vincristine Sulfate
Oncovin
Lead sponsor: Spectrum Health Hospitals
Sponsor class: OTHER
First posted: 2009-02-18
Enrollment: 9
Sex: ALL
Minimum age (years): 2
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Eligibility criteria: 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

Primary outcomes:
measure: Improvement of Symptoms and Pain
timeFrame: Monthly physical exam first three months and then every three months after, for up to 36 months
measure: At Least 50% Shrinkage in Tumor Measurements by Physical Examination
timeFrame: Monthly physical exam first three months and then every three months after, for up to 36 months
measure: Response by MRI Measurements
timeFrame: evaluated 6, 12 and 24 months compared to baseline
Secondary outcomes:
measure: No Reported Psychological Toxicity Based Upon Psychological Evaluations
timeFrame: Psychological evaluation at 24 months
Other outcomes:
Locations:
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital
state: Michigan
Study officials:
affiliation: Helen DeVos Children's Hospital
name: Albert S Cornelius, MD
role: PRINCIPAL_INVESTIGATOR
References: