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
Sponsor:
Information provided by:
Spectrum Health Hospitals
ClinicalTrials.gov Identifier:
NCT00846430
First received: January 16, 2009
Last updated: September 22, 2009
Last verified: September 2009
  Purpose

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.

  Eligibility

Ages Eligible for Study:   2 Years to 30 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00846430

Contacts
Contact: Sara Finton, RN 616-391-9365 sara.finton@spectrum-health.org
Contact: Heidi Smith, RN 616-391-9364 heidi.smith@spectrum-health.org

Locations
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
Investigators
Principal Investigator: Albert S Cornelius, MD Helen DeVos Children's Hospital
  More Information

No publications provided

Responsible Party: Albert Cornelius, Helen DeVos Childrens Hospital
ClinicalTrials.gov 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:
Neurofibromas
Neurofibromatosis 1
Celecoxib
Celebrex
Peg-Interferon alpha-2b
PEG-Intron
Temozolomide
Temodar
Vincristine Sulfate
Oncovin

Additional relevant MeSH terms:
Neurofibromatoses
Neurofibromatosis 1
Neurofibroma
Osteitis Fibrosa Cystica
Nerve Sheath Neoplasms
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Nervous System Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Bone Diseases, Endocrine
Bone Diseases
Musculoskeletal Diseases
Interferon-alpha
Interferon Alfa-2a
Interferon Alfa-2b
Interferons
Peginterferon alfa-2b
Reaferon
Temozolomide
Vincristine
Celecoxib

ClinicalTrials.gov processed this record on April 17, 2014