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

Purpose

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.

Condition	Intervention	Phase
Sickle Cell Disease	Drug: GMI-1070	Phase 1 Phase 2


Study Type:	Interventional
Study Design:	Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
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

Resource links provided by NLM:

Genetics Home Reference related topics: sickle cell disease

MedlinePlus related topics: Sickle Cell Anemia

Genetic and Rare Diseases Information Center resources: Sickle Cell Anemia

U.S. FDA Resources

Further study details as provided by Pfizer:

Primary Outcome Measures:

Safety as Measured by the Number of Participants With Adverse Events [ Time Frame: 28 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Total Plasma Clearance [ Time Frame: 48 hours ] [ Designated as safety issue: No ]
Volume of the Central Compartment [ Time Frame: 48 hours ] [ Designated as safety issue: No ]
Intercompartmental Clearance [ Time Frame: 48 hours ] [ Designated as safety issue: No ]
Volume of the Peripheral Compartment [ Time Frame: 48 hours ] [ Designated as safety issue: No ]

Other Outcome Measures:

Blood Flow and Biomarkers of Adhesion [ Time Frame: 48 hours ] [ Designated as safety issue: No ]
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.


Enrollment:	15
Study Start Date:	May 2009
Study Completion Date:	September 2010
Primary Completion Date:	July 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: GMI-1070	Drug: GMI-1070 Intravenous GMI-1070 given as two doses over the course of one day

Eligibility


Ages Eligible for Study:	18 Years to 45 Years (Adult)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00911495

Locations


United States, California
Children's Hospital & Research Center Oakland
Oakland, California, United States, 94609
University of California at Davis, CCRC
Sacramento, California, United States, 95817
United States, North Carolina
Duke Comprehensive Sickle Cell Center
Durham, North Carolina, United States, 27710

Sponsors and Collaborators

Pfizer

Investigators


Study Director:	Helen Thackray, MD	GlycoMimetics Incorporated

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.


Responsible Party:	Pfizer
ClinicalTrials.gov Identifier:	NCT00911495 History of Changes
Other Study ID Numbers:	GMI-1070-103
Study First Received:	May 27, 2009
Results First Received:	September 11, 2012
Last Updated:	August 17, 2016
Health Authority:	United States: Food and Drug Administration

Keywords provided by Pfizer:


Sickle Cell Disease

Additional relevant MeSH terms:


Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia	Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on September 26, 2016