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

GlycoMimetics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Sickle Cell Disease

Treatments: Drug: GMI-1070

Study type: Interventional

Funder types: Industry

Identifiers

NCT00911495

GMI-1070-103

Details and patient eligibility

About

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.

Enrollment: 15 patients

Sex: All

Ages: 18 to 45 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

GMI-1070

Experimental group

Treatment:

Drug: GMI-1070