4 results
Primary:* To demonstrate non-inferiority between the level of agreement in diagnosis (i.e. patient classification of normal, mild/moderate or severe ischemic disease based on the number of reversible perfusion segments) between sequential adenosine…
The trial is designed as an open-label pilot study. The main goal is to investigate if bezafibrate can reduce VLCFA in vivo in patient with X-ALD. If there is indeed a biochemical effect, a large follow-up study will be initiated with clinical…
The primary objective is to assess the effects of the combination of OCA and BZF on alkaline phosphatase(ALP) in comparison to BZF alone in subjects with primary biliary cholangitis (PBC)To assess the effects of the combination of OCA and BZF 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…