4 results
To assess the safety of treatment with escalating dosages of VELCADE in combination with ZARNESTRA in subjects with Intermediate-2 or high risk MDS according to the IPSS classification.
Primary ObjectivesThe primary objectives of this study are as follows:• To evaluate the safety and tolerability of risdiplam.• To investigate the pharmacokinetics (PK) of risdiplam and metabolites as appropriate.Secondary ObjectiveThe secondary…
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the increase in Hb concentrations in pediatric subjects with PK deficiency who are not regularly transfused
To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the reduction in transfusion burden in pediatric subjects with pyruvate kinase deficiency (PK deficiency) who are regularly transfused.