5 Clinical Trials for Various Conditions
The purpose of this Phase 1a, first in human, randomized, double-blind, placebo-controlled study is to evaluate the safety, tolerability, PK and PD profile of the orally administered IMR-687 in healthy adult subjects.
Sickle Cell Disease (SCD) is a rare disease occurring in an estimated 100,000 individuals, often poor and underserved, in the US. Silent and overt strokes contribute significantly to morbidity in adults with SCD, resulting in functional impairment, challenges with school and job performance, and premature death. Five NIH-funded randomized controlled trials have identified therapies to prevent silent and overt strokes in children with SCD, including monthly blood transfusion therapy (for preventing initial and recurrent strokes) and hydroxyurea (for preventing initial strokes). Despite the observation that at least 99% of children with SCD in high-income countries reach adulthood, and approximately 60% of adults will experience one or more strokes (\~50% with silent strokes and \~10% with overt strokes), no stroke trials have established therapeutic approaches for adults with SCD. For adults with SCD, inadequate evidence-based guidelines exist for secondary stroke prevention strategies. Applying stroke prevention strategies in children may not be effective for stroke prevention in adults with SCD, particularly given the high rate of co-morbidities. Identifying subgroups of adults with SCD and higher incidence coupled with the contribution of established stroke risk factors in the general population (smoking, diabetes, obesity, renal disease) will provide the requisite data required for the first-ever phase III clinical trials focused on secondary stroke prevention in adults.
The primary study hypothesis is that inhibition of factor Xa with rivaroxaban will reduce inflammation, coagulation and endothelial cell activation, and improve microvascular blood flow in patients with sickle cell disease (SCD) during the non-crisis, steady state. To test this hypothesis, this study will evaluate the effects of rivaroxaban on: * plasma markers of inflammation; * plasma markers of endothelial activation; * plasma markers of thrombin generation; and * microvascular blood flow assessed using laser Doppler velocimetry (LDV) of post-occlusive reactive hyperemia (PORH). In a cross-over design, subjects will receive rivaroxaban 20 mg/day and placebo for 4 weeks each, separated by a 2-week washout phase.
The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.
The purpose of the study is to determine the effectiveness of LOVAZA (fish oil capsules) to decrease inflammation in children and adolescents with Sickle Cell Disease (SCD). It has been found that besides the damage caused by sickle red blood cells themselves, the inflammatory response that occurs in SCD patients could potentially play a significant role in the occurrence of painful episodes or pain crises. The investigators will also study whether the subject/caregiver feels that there is an improvement in the child's quality of life by taking the medication. Besides the effect of LOVAZA on inflammation,the investigators are also testing whether the drug will have a beneficial effect on blood clotting ability (which is known to be increased in SCD) and on the anemia (low red blood cells) that is part of the disease entity.