Medical Treatment of "High-Risk" Neurofibromas

NCT00846430 | Completed Phase 2 Results

• 1 other identifier: 2008-260
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Neurofibromatosis 1

Keywords

Neurofibromas; Neurofibromatosis 1; Celecoxib; Celebrex; Peg-Interferon alpha-2b; PEG-Intron; Temozolomide; Temodar; Vincristine Sulfate; Oncovin

Outcome Measures

Primary Outcomes (3)

• Improvement of Symptoms and Pain

  Subjects will be evaluated for pain and Quality of Life scores

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

• At Least 50% Shrinkage in Tumor Measurements by Physical Examination

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

• Response by MRI Measurements

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

  evaluated 6, 12 and 24 months compared to baseline

Secondary Outcomes (1)

• No Reported Psychological Toxicity Based Upon Psychological Evaluations

  Psychological evaluation at 24 months

Study Arms (1)

Open-Label Intervention

EXPERIMENTAL

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; Drug: Celecoxib (Celebrex); Drug: Temozolomide (temodar); Drug: Vincristine Sulfate (Oncovin)

Interventions

Peg-Interferon alpha-2b DRUG

age and weight dependant

Open-Label Intervention

Celecoxib (Celebrex) DRUG

age and weight dependant

Open-Label Intervention

Temozolomide (temodar) DRUG

age and weight dependant

Open-Label Intervention

Vincristine Sulfate (Oncovin) DRUG

age and weight dependant

Open-Label Intervention

Eligibility Criteria

• Age: 2 Years - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• "High-Risk" Plexiform Neurofibromas associated with a diagnosis of NF1
• years old (minimum bodyweight of 10 kilograms)
• Adequate renal function

You may not qualify if:

• Previously untreated active optic glioma
• History of any previous allergy to study medications
• History of ischemic vascular disease
• Pregnancy / Breast feeding

Sponsors & Collaborators

• Corewell Health West: lead

Study Sites (1)

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibroma

Interventions

peginterferon alfa-2b; Celecoxib; Temozolomide; Vincristine

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Benzenesulfonamides; Sulfonamides; Amides; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Sulfones; Sulfur Compounds; Pyrazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Dacarbazine; Triazenes; Imidazoles; Vinca Alkaloids; Secologanin Tryptamine Alkaloids; Indole Alkaloids; Alkaloids; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Indolizidines; Indolizines

Results Point of Contact

• Title: Dr. Albert Cornelius
• Organization: Corewell Health

albert.cornelius@helendevoschildrens.org

616-391-2086

Study Officials

• Albert S Cornelius, MD

  Helen DeVos Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: January 16, 2009
• First Posted: February 18, 2009
• Study Start: October 1, 2008
• Primary Completion: April 1, 2017
• Study Completion: May 1, 2017
• Last Updated: August 30, 2023
• Results First Posted: August 30, 2023

Record last verified: 2023-08

Locations

US (1)