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Completed

NCT Number: NCT00846430

Medical Treatment of "High-Risk" Neurofibromas

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.

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Key information

Conditions

Neurofibromatosis 1 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Neoplasms Neoplasms by Histologic Type Neoplasms, Nerve Tissue Neoplastic Syndromes, Hereditary Nerve Sheath Neoplasms Nervous System Diseases Nervous System Neoplasms Neurocutaneous Syndromes Neurodegenerative Diseases Neurofibroma Neurofibromatoses Neuromuscular Diseases Peripheral Nervous System Diseases Peripheral Nervous System Neoplasms

Age range

2 year–30 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Peg-Interferon alpha-2b

Drug

age and weight dependant

celecoxib (Celebrex)

Drug

age and weight dependant

Temozolomide (temodar)

Drug

age and weight dependant

Vincristine Sulfate (Oncovin)

Drug

age and weight dependant

Primary outcomes

  1. Improvement of Symptoms and Pain

    Time frame: Monthly physical exam first three months and then every three months after, for up to 36 months

    Subjects will be evaluated for pain and Quality of Life scores

  2. At Least 50% Shrinkage in Tumor Measurements by Physical Examination

    Time frame: Monthly physical exam first three months and then every three months after, for up to 36 months

  3. Response by MRI Measurements

    Time frame: evaluated 6, 12 and 24 months compared to baseline

    partial response by RICST criteria is defined as >50% tumor shrinkage

Secondary outcomes

  1. No Reported Psychological Toxicity Based Upon Psychological Evaluations

    Time frame: Psychological evaluation at 24 months

    Psychological toxicity defined as suicidal ideation

Sponsors and collaborators

Lead sponsor

Corewell Health West

Other

Registry information

Official study title

Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies

Important dates

Study start
2008
Primary completion
2017
Study completion
2017
First posted
Feb 18, 2009
Registry last updated
Aug 30, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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