Medical Treatment of "High-Risk" Neurofibromas
Recruitment status was Recruiting
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Neurofibromatosis 1 |
Drug: Peg-Interferon alpha-2b Drug: Celecoxib (Celebrex) Drug: Temozolomide (temodar) Drug: Vincristine Sulfate (Oncovin) |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: 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 |
- 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. ] [ Designated as safety issue: No ]
- 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 ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 20 |
| Study Start Date: | October 2008 |
| Estimated Study Completion Date: | December 2010 |
| Estimated Primary Completion Date: | October 2010 (Final data collection date for primary outcome measure) |
-
Drug: Peg-Interferon alpha-2b
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.
Eligibility| Ages Eligible for Study: | 2 Years to 30 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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| Contact: Sara Finton, RN | 616-391-9365 | sara.finton@spectrum-health.org |
| Contact: Heidi Smith, RN | 616-391-9364 | heidi.smith@spectrum-health.org |
| United States, Michigan | |
| Helen DeVos Children's Hospital | Recruiting |
| Grand Rapids, Michigan, United States, 49503 | |
| Principal Investigator: Albert S. Cornelius, MD | |
| Principal Investigator: | Albert S Cornelius, MD | Helen DeVos Children's Hospital |
More Information
No publications provided
| Responsible Party: | Albert Cornelius, Helen DeVos Childrens Hospital |
| ClinicalTrials.gov Identifier: | NCT00846430 History of Changes |
| Other Study ID Numbers: | 2008-260, 2008-260 |
| Study First Received: | January 16, 2009 |
| Last Updated: | September 22, 2009 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by Spectrum Health Hospitals:
|
Neurofibromas Neurofibromatosis 1 Celecoxib Celebrex Peg-Interferon alpha-2b |
PEG-Intron Temozolomide Temodar Vincristine Sulfate Oncovin |
Additional relevant MeSH terms:
|
Neurofibroma Neurofibromatoses Neurofibromatosis 1 Osteitis Fibrosa Cystica Nerve Sheath Neoplasms Neoplasms, Nerve Tissue Neoplasms by Histologic Type Neoplasms Peripheral Nervous System Neoplasms Nervous System Neoplasms Nervous System Diseases Peripheral Nervous System Diseases Neuromuscular Diseases Neoplastic Syndromes, Hereditary Neurocutaneous Syndromes |
Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Bone Diseases, Endocrine Bone Diseases Musculoskeletal Diseases Interferon-alpha Interferon Alfa-2a Interferon Alfa-2b Interferons Peginterferon alfa-2b Reaferon Temozolomide Vincristine Celecoxib |
ClinicalTrials.gov processed this record on June 17, 2013