5 results
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…
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 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…
This entry-into-human (EIH) Phase Ia/Ib study aims to establish the safety,pharmacokinetics, immunogenicity, and pharmacodynamics of RO7284755 (alone or incombination with atezolizumab) and to evaluate its anti-tumor activity.
Primary Objective: To evaluate the long-term safety (including progression to acute myeloid leukemia (AML) and/or other malignancies/pre-malignancies) of luspatercept in subjects who have participated in other luspatercept clinical trials.Secondary…