XXXX (Gleevec) in the treatment of @entity7087 : results of a 1-year, phase IIa, single-arm, open-label clinical trial.

@entity1 @entity272 @entity401 @entity669 @entity2213 @entity7087
OBJECTIVE: To assess the safety and effectiveness of @entity2213 in the treatment of @entity7087 ( @entity7087 ). METHODS: In this phase IIa, open-label, single-arm clinical trial, 30 @entity1 with @entity7087 were treated with @entity2213 400 mg daily. @entity1 were monitored monthly for safety assessments. @entity272 ( @entity272 ) were assessed every 3 months. Pulmonary function testing, chest radiography, echocardiography and skin biopsies were performed at baseline and after 12 months of treatment. RESULTS: Twenty-four @entity1 completed 12 months of therapy. 171 adverse events (AE) with possible relation to @entity2213 were identified; 97.6% were grade 1 or 2. Twenty-four serious AE were identified, two of which were attributed to study medication. @entity272 decreased by 6.6 points or 22.4% at 12 months (p=0.001). This change was evident starting at the 6-month time point (/\=-4.5; p<0.001) and was seen in @entity1 with both early and late-stage disease. Forced vital capacity (FVC) improved by 6.4% predicted (p=0.008), and the diffusion capacity remained stable. The improvement in FVC was significantly greater in @entity1 without @entity401 . Health-related quality of life measures improved or remained stable. Blinded dermatopathological analysis confirmed a significant decrease in skin thickness and improvement in skin morphology. CONCLUSIONS: Treatment with @entity2213 was tolerated by most @entity1 in this cohort. Although AE were common, most were mild to moderate. In this open-label experience, improvements in skin thickening and FVC were observed. Further investigation of @entity669 kinase inhibition for @entity7087 in a double-blind randomised placebo controlled trial is warranted. ClinicalTrials.gov, NCT00555581.
