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

2008-260

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.

2008-10-01

2017-04-01

2017-05-01

Completed

Early phase I

9

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

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 9, 'type': 'ACTUAL'}}

2023-08-30