Effects of HQK-1001 in Patients With Sickle Cell Disease

Exclusion Criteria

• •Assigned to a regular transfusion program
• •Use of erythropoiesis stimulating agents within 90 days prior to screening
• •An SCD pain crisis or SCD-related acute complication within 3 weeks prior to randomization
• •More than 5 SCD pain crisis or SCD-related acute complications within 12 months prior to screening
• •Pulmonary hypertension requiring therapy
• •ALT or AST \> 3x ULN
• •Serum creatinine \> 1.5x ULN
• •Serum amylase levels \> 1.5x ULN
• •Serum lipase level \> 1.5x ULN
• •A serious, concurrent illness that would limit ability to complete or comply with the study requirements
• •An acute illness (e.g., febrile, GI, respiratory) within 72 hours prior to screening
• •History of syncope, clinically significant dysrhythmias or resuscitation from sudden death due to SCD-related complication
• •Symptomatic peptic ulcer, hiatus hernia, or gastroesophageal reflux disease (GERD)
• •History of pancreatitis
• •Chronic opiate use, which, in the view of the investigator, could confound evaluation of an investigational drug
• •Current abuse of alcohol or drugs
• •Use of another investigational agent within 4 weeks or 5 half-lives, whichever is longer, prior to screening
• •Currently pregnant or breast feeding a child
• •Known infection with HIV-1
• •Infection with hepatitis B or hepatitis C, such that subjects are currently on anti-viral therapy or will be placed on therapy