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Medical Treatment of "High-Risk" Neurofibromas

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified May 2015 by Albert Cornelius, Spectrum Health Hospitals.
Recruitment status was:  Active, not recruiting
Sponsor:
ClinicalTrials.gov Identifier:
NCT00846430
First Posted: February 18, 2009
Last Update Posted: May 21, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Albert Cornelius, Spectrum Health Hospitals
  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
Intervention Model: Crossover Assignment
Masking: None (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 Albert Cornelius, 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. ]

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 ]

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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 30 Years   (Child, Adult)
Sexes Eligible for Study:   All
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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00846430


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

Responsible Party: Albert Cornelius, MD, Spectrum Health Hospitals
ClinicalTrials.gov Identifier: NCT00846430     History of Changes
Other Study ID Numbers: 2008-260
First Submitted: January 16, 2009
First Posted: February 18, 2009
Last Update Posted: May 21, 2015
Last Verified: May 2015

Keywords provided by Albert Cornelius, Spectrum Health Hospitals:
Neurofibromas
Neurofibromatosis 1
Celecoxib
Celebrex
Peg-Interferon alpha-2b
PEG-Intron
Temozolomide
Temodar
Vincristine Sulfate
Oncovin

Additional relevant MeSH terms:
Neurofibromatoses
Neurofibroma
Nerve Sheath Neoplasms
Neurofibromatosis 1
Neoplasms, Nerve Tissue
Neoplasms by Histologic Type
Neoplasms
Neoplastic Syndromes, Hereditary
Neurocutaneous Syndromes
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Neoplasms
Nervous System Neoplasms
Peripheral Nervous System Diseases
Neuromuscular Diseases
Temozolomide
Interferons
Vincristine
Interferon-alpha
Peginterferon alfa-2b
Celecoxib
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents, Phytogenic