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Nilotinib (Tasigna) in the Treatment of Early Diffuse Systemic Sclerosis: An Open-Label, Pilot Clinical Trial

ABSTRACT: To assess the safety, efficacy, and molecular change associated with treatment of patients with early, diffuse cutaneous systemic sclerosis (dcSSc) with nilotinib (Tasigna™). In this open-label pilot trial 6 adult patients with early dcSSc received nilotinib. Primary endpoints were safety and change in modified Rodnan Skin Score (MRSS) after 6 months. Lesional skin biopsies at baseline, 6 and 12 months of treatment were assessed by histopathology, immunohistochemistry, and DNA microarray.

ORGANISM(S): Homo sapiens

SUBMITTER: Tammara Wood

PROVIDER: E-GEOD-65405 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress

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Nilotinib (Tasigna™) in the treatment of early diffuse systemic sclerosis: an open-label, pilot clinical trial.

Gordon Jessica K JK Martyanov Viktor V Magro Cynthia C Wildman Horatio F HF Wood Tammara A TA Huang Wei-Ti WT Crow Mary K MK Whitfield Michael L ML Spiera Robert F RF

Arthritis research & therapy 20150818

<h4>Introduction</h4>Tyrosine kinase inhibitors (TKI) are medications of interest in the treatment of Systemic Sclerosis (SSc) because of their ability to inhibit pathways involved in fibrosis. In this open-label pilot trial, our objectives were to assess the safety, efficacy, and molecular change associated with treatment of patients with diffuse cutaneous (dc)SSc with the TKI nilotinib (Tasigna™).<h4>Methods</h4>Ten adult patients with early dcSSc were treated with nilotinib. Primary endpoints ...[more]

PMID: 26283632

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Nilotinib (Tasigna) in the Treatment of Early Diffuse Systemic Sclerosis: An Open-Label, Pilot Clinical Trial

Project description:To assess the safety, efficacy, and molecular change associated with treatment of patients with early, diffuse cutaneous systemic sclerosis (dcSSc) with nilotinib (Tasigna™).

2015-08-27 | GSE65405 | GEO

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Systemic sclerosis skin disease trajectory during mycophenolate mofetil treatment

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2017-01-01 | GSE76885 | GEO

A phase 2, open label single arm study for evaluating safety & efficacy of apremilast in the treatment of cutaneous disease in patients with recalcitrant dermatomyositis.

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A pilot open label, single dose trial of fenobam in adults with fragile X syndrome.

Project description:A pilot open label, single dose trial of fenobam, an mGluR5 antagonist, was conducted to provide an initial evaluation of safety and pharmacokinetics in adult males and females with fragile X syndrome (FXS).Twelve subjects, recruited from two fragile X clinics, received a single oral dose of 50-150 mg of fenobam. Blood for pharmacokinetic testing, vital signs and side effect screening was obtained at baseline and numerous time points for 6 h after dosing. Outcome measures included prepulse inhibition (PPI) and a continuous performance test (CPT) obtained before and after dosing to explore the effects of fenobam on core phenotypic measures of sensory gating, attention and inhibition.There were no significant adverse reactions to fenobam administration. Pharmacokinetic analysis showed that fenobam concentrations were dose dependent but variable, with mean (SEM) peak values of 39.7 (18.4) ng/ml at 180 min after the 150 mg dose. PPI met a response criterion of an improvement of at least 20% over baseline in 6 of 12 individuals (4/6 males and 2/6 females). The CPT did not display improvement with treatment due to ceiling effects.Clinically significant adverse effects were not identified in this study of single dose fenobam across the range of dosages utilised. The positive effects seen in animal models of FXS treated with fenobam or other mGluR5 antagonists, the apparent lack of clinically significant adverse effects, and the potential beneficial clinical effects seen in this pilot trial support further study of the compound in adults with FXS.

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Molecular Signatures in Skin Associated with Clinical Improvement During Mycophenolate Treatment in Systemic Sclerosis

Project description:Skin gene expression signatures, including intrinsic subset, are associated with skin score/MRSS improvement during mycophenolate mofetil (MMF) treatment.

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Ethosuximide for Essential Tremor: An Open-Label Trial.

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