Advertisement

Topics

Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas

2014-07-23 21:08:50 | BioPortfolio

Summary

This is a second Pilot Study to determine the efficacy of Gleevec® in neurofibromatosis (NF1) patients with plexiform neurofibromas using new response assessment modalities with the secondary goals of assessing Gleevec toxicity, and characterizing markers of response. The rationale for this study arises from the response of human and murine NF1 cells to Gleevec® in vitro, the response of a NF1 patient treated with Gleevec® for airway compression by a plexiform neurofibroma with a dramatic response not previously seen in NF1 therapy, and the experience in 37 NF1 patients treated with Gleevec® in the initial pilot study. Gleevec® will be dosed orally 400 mg/day for adult patients. If tolerated the dose will be increased to 600 mg/day after a minimum of two weeks and will be increased to 800 mg per day after an additional two weeks if tolerated (All escalations minimal at any time). Treatment will continue for 6 months with an option to continue for 24 months if the patient is deriving a clinical benefit.

Study Design

Allocation: Non-Randomized, Control: Active Control, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Conditions

Neurofibromatosis

Intervention

Gleevec

Location

Riley Hospital for Children
Indianapolis
Indiana
United States
46202

Status

Recruiting

Source

Indiana University

Results (where available)

View Results

Links

Published on BioPortfolio: 2014-07-23T21:08:50-0400

Clinical Trials [119 Associated Clinical Trials listed on BioPortfolio]

Effect of Antacids on Gleevec® in Healthy Volunteers

This is a research study that will investigate the effects of antacids (often used to treat stomach upset) on Gleevec® (a drug that is FDA-approved to treat some types of cancer) in healt...

Phase II Gleevec Idiopathic Hypereosinophilic Syndrome

The purpose of the trial is to determine the safety and efficacy of Gleevec" in idiopathic hypereosinophilic syndrome (HES) and to characterize the molecular basis for the therapeutic bene...

Homoharringtonine With Oral Gleevec in Chronic, Accelerated and Blast Phase Chronic Myeloid Leukemia (CML)

This will be an open label, multi-center study of up to 77 patients with CML in chronic, accelerated or blast phase who have developed resistance to or have failed previous treatment with ...

Effect of a Proton Pump Inhibitor on Gleevec® in Healthy Volunteers

This is a research study that will investigate the effects of proton pump inhibitors (often used to treat stomach upset) on Gleevec® (a drug that is FDA-approved to treat some types of ca...

Neurofibromatosis (NF) Registry Portal

The NF Registry is a database of patient-reported experiences of living with neurofibromatosis. It also serves as a referral source for clinical trials.

PubMed Articles [78 Associated PubMed Articles listed on BioPortfolio]

Germline mutation of CHEK2 in neurofibromatosis 1 and 2: Two case reports.

Neurofibromatosis, including type 1 and type 2, is inherited dominant disease that causes serious consequences. The genetic mechanism of these diseases has been described, but germline mutation of che...

The Rare Togetherness of Bladder Leiomyoma and Neurofibromatosis.

Neurofibromatosis Type 1 (Von Recklinghausen disease) is a common, autosomal dominant hereditary disorder characterized by involvement of multiple tissues derived from the neural crest. Urinary system...

Neurofibromatosis and Schwannomatosis.

Neurofibromatosis 1, neurofibromatosis 2, and schwannomatosis are a group of related classically inherited but often times sporadic tumor suppressor syndromes. Neuro-oncologists should recognize these...

Amusia and its electrophysiological correlates in neurofibromatosis type 1.

Auditory processing deficits are common in people with neurofibromatosis type 1 (NF1) and they often report difficulties in musical performance.

First report of the efficacy of vestibular rehabilitation in improving function in patients with Neurofibromatosis type 2: an observational cohort study in a clinical setting.

Patients with Neurofibromatosis type 2 often experience debilitating neuro-otological problems which affect their mobility and balance. This study examined the efficacy of a personalised program of ve...

Medical and Biotech [MESH] Definitions

A group of disorders characterized by an autosomal dominant pattern of inheritance with high rates of spontaneous mutation and multiple neurofibromas or neurilemmomas. NEUROFIBROMATOSIS 1 (generalized neurofibromatosis) accounts for approximately 95% of cases, although multiple additional subtypes (e.g., NEUROFIBROMATOSIS 2, neurofibromatosis 3, etc.) have been described. (From Neurochirurgie 1998 Nov;44(4):267-72)

Tumor suppressor genes located on the long arm of human chromosome 22. Mutation or loss of these genes causes NEUROFIBROMATOSIS 2.

Tumor suppressor genes located on the long arm of human chromosome 17 in the region 17q11.2. Mutation of these genes is thought to cause NEUROFIBROMATOSIS 1, Watson syndrome, and LEOPARD syndrome.

A protein found most abundantly in the nervous system. Defects or deficiencies in this protein are associated with NEUROFIBROMATOSIS 1, Watson syndrome, and LEOPARD syndrome. Mutations in the gene (GENE, NEUROFIBROMATOSIS 1) affect two known functions: regulation of ras-GTPase and tumor suppression.

An autosomal dominant disorder characterized by a high incidence of bilateral acoustic neuromas as well as schwannomas (NEURILEMMOMA) of other cranial and peripheral nerves, and other benign intracranial tumors including meningiomas, ependymomas, spinal neurofibromas, and gliomas. The disease has been linked to mutations of the NF2 gene (GENES, NEUROFIBROMATOSIS 2) on chromosome 22 (22q12) and usually presents clinically in the first or second decade of life.

More From BioPortfolio on "Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas"

Advertisement
Quick Search
Advertisement
Advertisement

 

Relevant Topic

Pediatrics
Pediatrics is the general medicine of childhood. Because of the developmental processes (psychological and physical) of childhood, the involvement of parents, and the social management of conditions at home and at school, pediatrics is a specialty. With ...


Searches Linking to this Trial