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

Tracking Information

• First Submitted Date: January 16, 2009
• First Posted Date: February 18, 2009
• Last Update Posted Date: December 15, 2022
• Actual Study Start Date: October 2008
• Actual Primary Completion Date: April 2017 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: February 16, 2009): 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. ]
• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures (submitted: February 16, 2009): 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 ]
• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Medical Treatment of "High-Risk" Neurofibromas
• Official Title: Medical Treatment of "High-Risk" Neurofibromas in Patients With Type 1 Neurofibromatosis: A Clinical Trial of Sequential Medical Therapies
• 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.
• 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 Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Neurofibromatosis 1

Intervention

• 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

Study Arms

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.

Interventions:

• Drug: Peg-Interferon alpha-2b
• Drug: Celecoxib (Celebrex)
• Drug: Temozolomide (temodar)
• Drug: Vincristine Sulfate (Oncovin)

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: December 13, 2022): 11
• Original Estimated Enrollment (submitted: February 16, 2009): 20
• Actual Study Completion Date: May 2017
• Actual Primary Completion Date: April 2017 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 2 Years to 30 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00846430
• Other Study ID Numbers: 2008-260; 2008-260 ( Other Identifier: Spectrum Health )
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Albert Cornelius, Spectrum Health Hospitals
• Original Responsible Party: Albert Cornelius, Helen DeVos Childrens Hospital
• Current Study Sponsor: Spectrum Health Hospitals
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Albert S Cornelius, MD; Helen DeVos Children's Hospital

• PRS Account: Spectrum Health Hospitals
• Verification Date: April 2017