View clinical trials related to Neurofibromatosis 1.
Filter by:RATIONALE: Drugs used in chemotherapy, such as vinblastine and carboplatin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more tumor cells. PURPOSE: This phase I trial is studying the side effects and best dose of vinblastine when given together with carboplatin in treating young patients with newly diagnosed or recurrent low-grade glioma.
This phase II trial is studying how well AZD2171 works in treating patients with neurofibromatosis type 1 and plexiform neurofibroma and/or neurofibroma near the spine. AZD2171 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.
This study will explore the growth of dermal neurofibromas (skin tumors) in patients with neurofibromatosis type 1 (NF1). Investigators will try to learn: 1) how fast (or slow) these benign tumors grow in NF1, 2) how often new tumors appear and 3) what genes are involved in the growth of the tumors. Men and women between 20 and 50 years of age diagnosed with NF1 and their biological parents are eligible for this study. Patients with NF1 are evaluated at the NIH Clinical Center with the following tests and procedures: - Medical examination and drawing of family tree. - Photos of the back, abdomen and thigh in order to count the number of skin tumors. - Photos of the skin taken with a special camera (Primos camera) that takes very detailed pictures of a small area of skin. - Photos of the skin taken with a dermatoscope, which takes very detailed pictures of a small area of skin under high magnification. - Biopsy of at least one skin tumor and biopsy of a small piece of normal skin. - Blood sample collection for genetic testing of the gene NF1 and to establish a cell line. - Other medical tests (e.g., x-rays or MRI) if needed. Patients and their families will also have a genetic counseling session and an opportunity to ask questions about neurofibromatosis type 1. Patients return to the NIH after 3, 6, 12, 18 and 24 months for follow-up photographs and possibly blood samples. Biological parents of patients provide a blood sample for genetic testing.
RATIONALE: Drugs used in chemotherapy, such as doxorubicin, ifosfamide, and etoposide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Radiation therapy uses high-energy x-rays to kill tumor cells. Giving combination chemotherapy with or without radiation therapy before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. Giving combination chemotherapy after surgery may kill any tumor cells that remain after surgery. PURPOSE: This phase II trial is studying how well combination chemotherapy works in treating patients with stage III or stage IV malignant peripheral nerve sheath tumors.
The study is a multicenter four-year outcome study of the natural history of tibial dysplasia in patients with NF1 and selected patients without NF1. We will obtain information on the natural history, burden, functional and health status, health-related quality of life, and surgical interventions/outcomes of tibial dysplasia. The project will also establish a Core Facility (NOCF) for tissue samples for future studies.
Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder with an estimated prevalence of 1/2190 to 1/6711. Attention deficit hyperactivity disorder (ADHD) has been reported to be common in NF1. We, the researchers at Hospices Civils de Lyon, designed a randomized, double blind, placebo controlled, crossover trial with a total follow-up duration of 9 weeks to evaluate the effect of methylphenidate (MPH) on the improvement on the simplified parents Conners' Rating Scale. In a parallel exploratory study we will compare the nature of attention deficit disorders in NF1 children to 30 ADHD NF1-free controls. Children aged 7 to 12 years are eligible when their intelligence quotient (IQ) is between 80 and 120. Fifty subjects (25 for each period) were required for testing the primary study hypothesis.
This study may identify genes that predict the seriousness of neurofibromatosis type 1 (NF1). Finding these genes may explain why some people with NF1 have more medical problems than others. The study will also examine medical problems in NF1 that are rarely seen and are not well understood. Male and female patients with NF1 who have gone through puberty may be eligible for this study, as well as patients of any age who have unique or under-recognized disease features. Affected and unaffected family members, including parents, siblings, and more distant relatives, may also be enrolled. Candidates are screened with a discussion of medical history or review of medical records, or both. Participants undergo the following procedures: Patients with NF1 - Physical examination and family history - Photographs of the iris of each eye - Photographs of the back, abdomen and thigh to count skin tumors - Photographs of the face and body (with underwear on) to help track growth and appearance - Magnetic resonance imaging (MRI) of the spine (This test uses a magnetic field and radio waves to look for tumors and curvature of the spine. The patient lies still in the scanner, a narrow cylindrical device, wearing earplugs to muffle loud knocking sounds that occur during the scan. A contrast material called gadolinium is injected into a vein through a catheter to enhance the images.) - Blood draw for genetic studies - Possibly a skin biopsy (with the use of numbing medicine, removal of a small sample of skin tissue) to grow cells in the laboratory Patients with NF1 who have unique or under-recognized disease features - Physical examination and family history - Blood draw for genetic studies - Possibly a skin biopsy - Possibly additional tests, such as blood work, x-rays, photographs, MRIs, ultrasounds, or other tests Unaffected family members - Blood draw for genetic studies - Brief skin and eye examinations - Possibly a skin biopsy for cell culture Families are asked to give permission for researchers to recontact them for follow-up information, additional blood samples, or follow-up visit. ...
Background: Neurofibromatosis Type 1 (NF1) is an autosomal dominant, progressive genetic disorder characterized by diverse clinical manifestations. Patients with NF1 have an increased risk of developing tumors of the central and peripheral nervous system including plexiform neurofibromas, which are benign nerve sheath tumors that may cause severe morbidity and possible mortality. The histopathology of these tumors suggests that events connected with formation of fibroblasts might constitute a point of molecular vulnerability. Gene profile analysis demonstrates overexpression of fibroblast growth factor, epidermal growth factor, and platelet-derived growth factor in plexiform neurofibromas in patients with NF1. Pirfenidone is a novel antifibrotic agent that inhibits these and other growth factors. Clinical experience in adults has demonstrated that pirfenidone is effective in a variety of fibrosing conditions and pirfenidone is presently under study in a phase II trial for adults with progressive plexiform neurofibromas. A phase I trial of pirfenidone in children and young adults with NF1 and plexiform neurofibromas was completed, and has established the phase II dose (the dose resulting in a mean drug exposure [AUC] not more than 1 standard deviation below the mean drug exposure [AUC] in adults who received pirfenidone at the dose level demonstrating activity in fibrosing conditions). Pirfenidone has been well tolerated. Objectives: To determine whether pirfenidone increases the time to disease progression based on volumetric measurements in children and young adults with NF1 and growing plexiform neurofibromas. To define the objective response rate to pirfenidone in NF1-related plexiform neurofibromas. To describe and define the toxicities of pirfenidone. Eligibility: Individuals (greater than or equal to 3 years to less than or equal to 21 years of age) with a clinical diagnosis of NF1 and inoperable, measurable, and progressive plexiform neurofibromas that have the potential to cause substantial morbidity. Design: The phase II dose will be used in a single stage, single arm phase II trial The natural history of the growth of plexiform neurofibromas is unknown. For this reason, time to disease progression on the placebo arm of an ongoing National Cancer Institute (NCI) Pediatric Oncology Branch (POB) placebo-controlled, double-blind, cross-over phase II trial of the farnesyltransferase inhibitor R115777 for children and young adults with NF1 and progressive plexiform neurofibromas. Funding source - Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD)
RATIONALE: New imaging procedures such as fludeoxyglucose F 18 positron emission tomography (FDG-PET) and magnetic resonance (MR) perfusion imaging may improve the ability to detect disease progression, help doctors predict a patient's response to treatment, and help plan the most effective treatment. PURPOSE: This diagnostic trial is studying how well FDG-PET and MR perfusion imaging work in finding disease progression and determining response to treatment in patients with neurofibromatosis 1 and plexiform neurofibroma.
RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. Pirfenidone may slow the growth or prevent further development of plexiform neurofibromas. PURPOSE: Phase I trial to study the effectiveness of pirfenidone in treating young patients who have neurofibromatosis type 1 and plexiform neurofibroma.