Medical Treatment of "High-Risk" Neurofibromas

Neurofibromatosis 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00846430
• Other study ID #: 2008-260
• Secondary ID: 2008-260
• Status: Completed
• Phase: Phase 2
• Start date: October 2008
• Est. completion date: May 2017

Study information

• Verified date: August 2023
• Source: Spectrum Health Hospitals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: May 2017
• Est. primary completion date: April 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 30 Years

Eligibility

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

Related Conditions & MeSH terms

• Neurofibroma
• Neurofibromatoses
• Neurofibromatosis 1

Intervention

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

Locations

Country	Name	City	State
United States	Helen DeVos Children's Hospital	Grand Rapids	Michigan

Sponsors (1)

Lead Sponsor	Collaborator
Spectrum Health Hospitals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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	No Reported Psychological Toxicity Based Upon Psychological Evaluations	Psychological toxicity defined as suicidal ideation	Psychological evaluation at 24 months