Medical Treatment of "High-Risk" Neurofibromas

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Albert Cornelius, Spectrum Health Hospitals Identifier:
First received: January 16, 2009
Last updated: May 20, 2015
Last verified: May 2015
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.

Condition Intervention Phase
Neurofibromatosis 1
Drug: Peg-Interferon alpha-2b
Drug: Celecoxib (Celebrex)
Drug: Temozolomide (temodar)
Drug: Vincristine Sulfate (Oncovin)
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies

Resource links provided by NLM:

Further study details as provided by Spectrum Health Hospitals:

Primary Outcome Measures:
  • 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. ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Estimated Enrollment: 20
Study Start Date: October 2008
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: October 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 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.
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

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.

Ages Eligible for Study:   2 Years to 30 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00846430

United States, Michigan
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States, 49503
Sponsors and Collaborators
Spectrum Health Hospitals
Principal Investigator: Albert S Cornelius, MD Helen DeVos Children's Hospital
  More Information

No publications provided

Responsible Party: Albert Cornelius, MD, Spectrum Health Hospitals Identifier: NCT00846430     History of Changes
Other Study ID Numbers: 2008-260, 2008-260
Study First Received: January 16, 2009
Last Updated: May 20, 2015
Health Authority: United States: Institutional Review Board

Keywords provided by Spectrum Health Hospitals:
Neurofibromatosis 1
Peg-Interferon alpha-2b
Vincristine Sulfate

Additional relevant MeSH terms:
Nerve Sheath Neoplasms
Neurofibromatosis 1
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Neoplasms by Histologic Type
Neoplasms, Nerve Tissue
Neoplastic Syndromes, Hereditary
Nervous System Diseases
Nervous System Neoplasms
Neurocutaneous Syndromes
Neurodegenerative Diseases
Neuromuscular Diseases
Peripheral Nervous System Diseases
Peripheral Nervous System Neoplasms
Peginterferon alfa-2b
Alkylating Agents
Analgesics, Non-Narcotic
Anti-Infective Agents
Anti-Inflammatory Agents
Anti-Inflammatory Agents, Non-Steroidal
Antimitotic Agents processed this record on November 25, 2015