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.
The purpose of this study is to evaluate how safe telisotuzumab vedotin is and how telisotuzumab vedotin is tolerated as monotherapy and in combination with osimertinib.
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…
Primary Objective:• To evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total triiodothyronine (T3) concentrations, measured by liquid chromatography with tandem mass spectrometry (LC/MS/MS), and the requirement…
Phase 1• To determine the maximum tolerated dose (MTD) and RP2D of BLU 945 as monotherapy and in combination with osimertinib• To determine the safety and tolerability of BLU 945 as monotherapy and in combination with osimertinibPhase 2• To assess…