Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease

Phase 1/Phase 2 Study of the Safety, Pharmacokinetics, and Microvascular Effect of Titrating Doses of Intravenous GMI-1070, a Pan-Selectin Inhibitor, in Adults With Sickle Cell Disease

This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the outpatient setting.

Inclusion Criteria:

• Age 18 to 45 years
• Established diagnosis of SCD-SS or SCD-SB0-thal
• At medical baseline, with no evidence of worsening of disease over the last 3 months
• Available and agree to return for follow-up visits for the full duration of the study
• Able to cooperate with study procedures
• Documented and observed written informed consent

Exclusion Criteria:

• Vaso-occlusive crisis
• Recent major surgery, hospitalization, infection, significant bleeding, cerebrovascular accident or seizure, or transfusion
• Currently receiving, or has received within the previous 4 weeks, any other investigational agent
• Pregnant or lactating female; or female of childbearing age unable or unwilling to comply with birth control or abstinence during the course of the study