5 results
1. Normalization of the abnormal serum TH parameters and thereby improving the clinical condition of the AHDS patients2. Observation of changes in cognitive and motor function.
• Determine the maximum tolerated dose (MTD) and/or recommended dose (RD) for further evaluation of XL092 when administered alone and in combination with ICIs to subjects with advanced solid tumors• Evaluate preliminary efficacy of XL092 when…
Part I:Primary Objective: Evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, as measured by the Gross Motor Function Measure (GMFM)-88 and Bayley Scales of Infant Development (BSID-III) Gross Motor Skill Domain…
Therapy:The investigational product, ACP-196, will be supplied as hardgelatin capsules for oral administration.Commercially available ibrutinib (IMBRUVICA®) will be used asthe reference therapy.Objectives: Primary Objective:To assess whether ACP-196…
This study will evaluate the efficacy and safety of multiple therapies that are selected using somatic alterations and potential predictive biomarkers identified via NGS assays in patients with solid tumors: