Back to resultsStudy of Intravenous GMI-1070 in Adults With Sickle Cell Disease
Primary Purpose
Sickle Cell Disease
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
GMI-1070
Sponsored by
About this trial
This is an interventional treatment trial for Sickle Cell Disease focused on measuring 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
Sites / Locations
- Children's Hospital & Research Center Oakland
- University of California at Davis, CCRC
- Duke Comprehensive Sickle Cell Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
GMI-1070
Arm Description
Outcomes
Primary Outcome Measures
Safety as Measured by the Number of Participants With Adverse Events
Secondary Outcome Measures
Total Plasma Clearance
Volume of the Central Compartment
Intercompartmental Clearance
Volume of the Peripheral Compartment
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00911495
Brief Title
Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease
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
Study Type
Interventional
2. Study Status
Record Verification Date
May 2020
Overall Recruitment Status
Completed
Study Start Date
May 2009 (undefined)
Primary Completion Date
July 2010 (Actual)
Study Completion Date
September 2010 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
GlycoMimetics Incorporated
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
Keywords
Sickle Cell Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)
8. Arms, Groups, and Interventions
Arm Title
GMI-1070
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
GMI-1070
Intervention Description
Intravenous GMI-1070 given as two doses over the course of one day
Primary Outcome Measure Information:
Title
Safety as Measured by the Number of Participants With Adverse Events
Time Frame
28 days
Secondary Outcome Measure Information:
Title
Total Plasma Clearance
Time Frame
48 hours
Title
Volume of the Central Compartment
Time Frame
48 hours
Title
Intercompartmental Clearance
Time Frame
48 hours
Title
Volume of the Peripheral Compartment
Time Frame
48 hours
Other Pre-specified Outcome Measures:
Title
Blood Flow and Biomarkers of Adhesion
Description
As an exploratory outcome mean change in microvascular blood flow from baseline to each time point was measured. Microvascular blood flow was also measured as microFI, perfused vessel density, and RBC velocity.
Time Frame
48 hours
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
45 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Helen Thackray, MD
Organizational Affiliation
GlycoMimetics Incorporated
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital & Research Center Oakland
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
University of California at Davis, CCRC
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Duke Comprehensive Sickle Cell Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
24988449
Citation
Wun T, Styles L, DeCastro L, Telen MJ, Kuypers F, Cheung A, Kramer W, Flanner H, Rhee S, Magnani JL, Thackray H. Phase 1 study of the E-selectin inhibitor GMI 1070 in patients with sickle cell anemia. PLoS One. 2014 Jul 2;9(7):e101301. doi: 10.1371/journal.pone.0101301. eCollection 2014. Erratum In: PLoS One. 2014;9(10):e111690.
Results Reference
derived
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Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease
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