Medical Treatment of "High-Risk" Neurofibromas

The recruitment status of this study is unknown because the information has not been verified recently.
Verified September 2009 by Spectrum Health Hospitals.
Recruitment status was  Recruiting
Information provided by:
Spectrum Health Hospitals Identifier:
First received: January 16, 2009
Last updated: September 22, 2009
Last verified: September 2009

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 2010
Estimated Primary Completion Date: October 2010 (Final data collection date for primary outcome measure)
Intervention Details:
    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

Contact: Sara Finton, RN 616-391-9365
Contact: Heidi Smith, RN 616-391-9364

United States, Michigan
Helen DeVos Children's Hospital Recruiting
Grand Rapids, Michigan, United States, 49503
Principal Investigator: Albert S. Cornelius, MD         
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, Helen DeVos Childrens Hospital Identifier: NCT00846430     History of Changes
Other Study ID Numbers: 2008-260, 2008-260
Study First Received: January 16, 2009
Last Updated: September 22, 2009
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:
Neurofibromatosis 1
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Neoplasms by Histologic Type
Neoplasms, Nerve Tissue
Neoplastic Syndromes, Hereditary
Nerve Sheath Neoplasms
Nervous System Diseases
Nervous System Neoplasms
Neurocutaneous Syndromes
Neurodegenerative Diseases
Neuromuscular Diseases
Peripheral Nervous System Diseases
Peripheral Nervous System Neoplasms
Peginterferon alfa-2b
Anti-Infective Agents
Antimitotic Agents
Antineoplastic Agents
Antineoplastic Agents, Phytogenic
Antiviral Agents
Immunologic Factors
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs processed this record on March 26, 2015