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.
Primary Objective(s)Phase I (completed)The primary objective of the Phase I portion of the study is to determine the maximum tolerated dose (MTD) of the combination of melflufen and dexamethasone in patients with relapsed/refractory multiple myeloma…
Primary objective:The primary objective of this study is to assess the time to painful VOC resolution, measured from the first dose of sevuparine given to achievement of crises resolution, as compared to placebo. Secondary objectives: The secondary…
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…
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…