5 results
Primary ObjectivePart1: To evaluate the safety and tolerability of intravenous (IV) administration of AL001 over up to 96 weeks in asymptomatic and symptomatic carriersof a granulin (GRN) mutation causative of frontotemporal dementia (FTD) and in…
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…
To evaluate if hepcidin levels can predict response to iron therapy with either ferrous fumarate, ferric maltol, and intravenous iron in patients with IBD.
The primary objective of this study is: Part 1: To evaluate the efficacy of AL001 compared with placebo as measured by CDR® plus NACC FTLD-SB.Part 2: To assess the long-term safety and tolerability of AL001 in participants who have completed 96…
To determine the effect of treatment of postoperative iron deficiency anemia (IDA) with intravenous iron (IVI) on disability 90 days after cardiac surgery.