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Medical Treatment of "High-Risk" Neurofibromas

Primary Purpose

Neurofibromatosis 1

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Peg-Interferon alpha-2b
Celecoxib (Celebrex)
Temozolomide (temodar)
Vincristine Sulfate (Oncovin)
Sponsored by
Spectrum Health Hospitals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Neurofibromatosis 1 focused on measuring Neurofibromas, Neurofibromatosis 1, Celecoxib, Celebrex, Peg-Interferon alpha-2b, PEG-Intron, Temozolomide, Temodar, Vincristine Sulfate, Oncovin

Eligibility Criteria

2 Years - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Helen DeVos Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Open-Label Intervention

Arm Description

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.

Outcomes

Primary Outcome Measures

Improvement of Symptoms and Pain
Subjects will be evaluated for pain and Quality of Life scores
At Least 50% Shrinkage in Tumor Measurements by Physical Examination
Response by MRI Measurements
partial response by RICST criteria is defined as >50% tumor shrinkage

Secondary Outcome Measures

No Reported Psychological Toxicity Based Upon Psychological Evaluations
Psychological toxicity defined as suicidal ideation

Full Information

First Posted
January 16, 2009
Last Updated
August 7, 2023
Sponsor
Spectrum Health Hospitals
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1. Study Identification

Unique Protocol Identification Number
NCT00846430
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
October 2008 (Actual)
Primary Completion Date
April 2017 (Actual)
Study Completion Date
May 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Spectrum Health Hospitals

4. Oversight

Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neurofibromatosis 1
Keywords
Neurofibromas, Neurofibromatosis 1, Celecoxib, Celebrex, Peg-Interferon alpha-2b, PEG-Intron, Temozolomide, Temodar, Vincristine Sulfate, Oncovin

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Open-Label Intervention
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Peg-Interferon alpha-2b
Intervention Description
age and weight dependant
Intervention Type
Drug
Intervention Name(s)
Celecoxib (Celebrex)
Intervention Description
age and weight dependant
Intervention Type
Drug
Intervention Name(s)
Temozolomide (temodar)
Intervention Description
age and weight dependant
Intervention Type
Drug
Intervention Name(s)
Vincristine Sulfate (Oncovin)
Intervention Description
age and weight dependant
Primary Outcome Measure Information:
Title
Improvement of Symptoms and Pain
Description
Subjects will be evaluated for pain and Quality of Life scores
Time Frame
Monthly physical exam first three months and then every three months after, for up to 36 months
Title
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
Title
Response by MRI Measurements
Description
partial response by RICST criteria is defined as >50% tumor shrinkage
Time Frame
evaluated 6, 12 and 24 months compared to baseline
Secondary Outcome Measure Information:
Title
No Reported Psychological Toxicity Based Upon Psychological Evaluations
Description
Psychological toxicity defined as suicidal ideation
Time Frame
Psychological evaluation at 24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Albert S Cornelius, MD
Organizational Affiliation
Helen DeVos Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Helen DeVos Children's Hospital
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49503
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Medical Treatment of "High-Risk" Neurofibromas

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