A Single Dose Study of the Safety, Blood Levels and Biological Effects of Aes-103 Compared to Placebo in Subjects With Stable Sickle Cell Disease

• Status: Completed
• Trial end date: 2013-06-07

Summary

The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of Aes-103 (active ingredient 5-hydroxymethyl-2-furfural [5-HMF]) compared with placebo in subjects with stable sickle cell disease (SCD). Safety will be measured by monitoring adverse events (AEs), electrocardiograms (ECGs), vital signs, and laboratory values. Pharmacokinetics of Aes-103 will be measured over time in plasma, red blood cell hemolysate and binding of Aes-103 to hemoglobin. Pharmacodynamic effects will be assessed by measuring partial pressure of oxygen at which 50% of hemoglobin is saturated with oxygen (p50) while breathing normal air, blood oxygen levels (SpO2), ex-vivo antisickling effects in a hypoxic environment, and by imaging related changes in tissue blood flow and oxygen levels.

• Phase: Phase 1

Details

• Lead Sponsor: Baxalta now part of Shire; Baxalta US Inc.
• Collaborators: Cato Research; ClinPharm Consulting, LLC; Infrared Imaging and Thermometry Unit, Biomedical Engineering and Physical Science Shared Resource (NIBIB); National Chung Cheng University; National Heart, Lung, and Blood Institute (NHLBI); QS Pharma; Ricerca Biosciences LLC; SAIC-Frederick, Inc.; Therapeutics for Rare and Neglected Diseases (TRND)