3 Clinical Trials for Various Conditions
The purpose of this study is to determine if intravenous L-citrulline can abrogate an active vaso-occlusive crisis in sickle cell disease, resulting in decreased pain, reduction or elimination of opiate usage, and reduction or elimination of hospital admission. The applicant is developing intravenous L-citrulline (Turnobi™) for treatment of sickle cell disease (SCD). The current development program targets treatment of sickle cell-associated vaso-occlusive crisis (VOC) specifically. The aim of Part 1 is to identify the optimum dose regimens for the Part 2 of the trial which is a double-blind, placebo controlled adaptive 'pick-the-winner' design. This study will allow assignment of more subjects to the better treatment arm/s based on emerging data. The study, initially, will evaluate efficacy and tolerability of incremental doses of intravenous (IV) L-citrulline (Turnobi™) in patients with SCD while receiving standard of care therapy for VOC.
Background: Painful vasoocclusive crisis (VOC) occurs in people with sickle cell disease (SCD). People with VOC have many visits to the hospital. About 10 30 percent of these people will go on to develop acute chest syndrome (ACS). ACS can cause further ill health. It can also cause death. Researchers want to find ways to diagnose ACS more quickly. To do this, they want to use stored blood samples and scans from a study (the DeNOVO trial) that was closed in 2015. They want to see if scans and samples taken of people with VOC who later developed ACS could help diagnose ACS faster. The data of people in the DeNOVO study who did not develop ACS will serve as controls. Objectives: To look at data from the DeNOVO trial to find a way to diagnose ACS more quickly. Eligibility: People 10 85 years old who took part in NHLBI Protocol number 05-H-0019 (the DeNOVO trial). The trial lasted from 2004 to 2008. The study was closed in November 2015. Design: Scans and intact, frozen samples from a study that was closed in 2015 will be studied. No new participants will be enrolled. ...
This Phase 3 study will assess the safety and efficacy of a single dose of inclacumab, a P-selectin inhibitor, for a vaso-occlusive crisis (VOC) after an index VOC in participants with sickle cell disease (SCD). Participants will be randomized to receive either inclacumab or placebo.