4 results
This study will evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of GDC-0134 in patients with amyotrophic lateral sclerosis (ALS). Safety Objective: To evaluate the safety of single and multiple…
This study will evaluate the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of GDC-0134 in patients with amyotrophic lateral sclerosis (ALS).The long term safety and tolerability of up to 48 weeks of GDC-0134 will…
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…
Primary:• To compare the efficacy of viltolarsen administered intravenously (IV) at weekly doses of 80 mg/kg over a 48-week treatment period vs. placebo controls in ambulant boys ages 4 to <8 years with DMD using the Time to Stand Test (…