Medical Treatment of "High-Risk" Neurofibromas

• ClinicalTrials.gov Identifier: NCT00846430
• Recruitment Status: Completed
• First Posted: February 18, 2009
• Last Update Posted: December 15, 2022
• Sponsor: Spectrum Health Hospitals
• Information provided by (Responsible Party): Albert Cornelius, Spectrum Health Hospitals

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Neurofibromatosis 1	Drug: Peg-Interferon alpha-2b; Drug: Celecoxib (Celebrex); Drug: Temozolomide (temodar); Drug: Vincristine Sulfate (Oncovin)	Phase 2

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 11 participants
• Allocation: N/A
• Intervention Model: Single Group 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
• Actual Study Start Date: October 2008
• Actual Primary Completion Date: April 2017
• Actual Study Completion Date: May 2017

Arms and Interventions

Arm

Intervention/treatment

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

Outcome Measures

Primary Outcome Measures :

1. 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 :

1. 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 ]

Eligibility Criteria

• 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

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
• Other Study ID Numbers: 2008-260; 2008-260 ( Other Identifier: Spectrum Health )
• First Posted: February 18, 2009
• Last Update Posted: December 15, 2022
• Last Verified: April 2017

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; Neurofibromatosis 1; Neurofibroma; 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; Genetic Diseases, Inborn; Peripheral Nervous System Diseases; Neuromuscular Diseases; Peripheral Nervous System Neoplasms; Nervous System Neoplasms; Interferons; Interferon-alpha; Interferon alpha-2; Peginterferon alfa-2b; Celecoxib; Vincristine; Temozolomide; Antineoplastic Agents; Antiviral Agents; Anti-Infective Agents; Antineoplastic Agents, Phytogenic; Tubulin Modulators; Antimitotic Agents