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.
Part 1PrimaryTo assess dose limiting toxicities (DLTs) of EDR to select an RP3D for the combination to be used in Part 2 of this study.Secondary• To evaluate the overall safety profile of EDR to select an RP3D for the combination to be used in Part…
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…
Primary:• To compare the efficacy of elranatamab vs lenalidomide Secondary:- To compare the efficacy of elranatamab vs lenalidomide- To determine the safety and tolerability of elranatamab- To evaluate the PK of elranatamab- To evaluate the…