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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00911495
First received: May 27, 2009
Last updated: January 31, 2014
Last verified: May 2013
  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:


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
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

No publications provided by Pfizer

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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: January 31, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
Sickle Cell Disease

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies

ClinicalTrials.gov processed this record on November 27, 2014