2 results
Approved WMOCompleted
Primary objective:Determine the short- and long-term safety and tolerability of single and multiple doses of NI006 in subjects with ATTR-CM by evaluating adverse events (AE) and serious adverse events (SAE) and changes in laboratory parameters (…
Approved WMORecruiting
Phase 2:Primary ObjectivesTo determine the recommended Phase3 dose of mitapivat by evaluating the effect of 2 dose levels of mitapivat versus placebo on:• Anemia in subjects with sickle cell disease (SCD)• SafetySecondary ObjectivesTo evaluate the…