This trial is active, not recruiting.

Condition neurofibromatosis 1
Treatments peg-interferon alpha-2b, celecoxib (celebrex), temozolomide (temodar), vincristine sulfate (oncovin)
Phase phase 2
Sponsor Spectrum Health Hospitals
Start date October 2008
End date October 2017
Trial size 20 participants
Trial identifier NCT00846430, 2008-260


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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model crossover assignment
Masking open label
Primary purpose treatment
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.
peg-interferon alpha-2b
age and weight dependant
celecoxib (celebrex)
age and weight dependant
temozolomide (temodar)
age and weight dependant
vincristine sulfate (oncovin)
age and weight dependant

Primary Outcomes

Tumor response based on evaluation of symptom assessment, tumor measurements, and MRI studies - Toxicity of treatment combinations based upon laboratory studies and physical examination
time frame: Monthly physical exam first three months and then every three months after, MRI's will occur at baseline, 6, 12 and 24 months.

Secondary Outcomes

Psychological toxicity based upon psychological evaluations - Improved quality of life based upon physical examination and performance scales
time frame: Psychological evaluation at baseline, 3, 12, and 24 months

Eligibility Criteria

Male or female participants from 2 years up to 30 years old.

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

Additional Information

Official title Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies
Principal investigator Albert S Cornelius, MD
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.
Trial information was received from ClinicalTrials.gov and was last updated in May 2015.
Information provided to ClinicalTrials.gov by Spectrum Health Hospitals.