Brief Summary
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.
Brief Title
Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease
Completion Date
Completion Date Type
Actual
Conditions
Sickle Cell Disease
Eligibility Criteria
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
* 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
Inclusion Criteria
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
* 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
Gender
All
Gender Based
false
Keywords
Sickle Cell Disease
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
45 Years
Minimum Age
18 Years
NCT Id
NCT00911495
Org Class
Industry
Org Full Name
GlycoMimetics Incorporated
Org Study Id
GMI-1070-103
Overall Status
Completed
Phases
Phase 1
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
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
Primary Outcomes
Outcome Measure
Safety as Measured by the Number of Participants With Adverse Events
Outcome Time Frame
28 days
Secondary Outcomes
Outcome Time Frame
48 hours
Outcome Measure
Total Plasma Clearance
Outcome Time Frame
48 hours
Outcome Measure
Volume of the Central Compartment
Outcome Time Frame
48 hours
Outcome Measure
Intercompartmental Clearance
Outcome Time Frame
48 hours
Outcome Measure
Volume of the Peripheral Compartment
Start Date
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Adult
Maximum Age Number (converted to Years and rounded down)
45
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
M. Catherine Driscoll
Investigator Email
cdriscol@montefiore.org
Investigator Phone