- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00834899
A Safety Study of Eptifibatide in Patients With Sickle Cell Disease
A Phase I/II Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety of Eptifibatide as Treatment for Acute Pain Episodes in Sickle Cell Disease
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Sickle cell disease has been referred to both as a condition associated with increased risk of blood clots and increased inflammation. Despite the abundant laboratory evidence of abnormal blood clotting and inflammation, the contribution of these changes to the problems experienced by patients with sickle cell disease remains uncertain. In additional to abnormal blood clotting, platelets (small blood cells that help blood clotting) are more activated in sickle cell disease patients compared to healthy patients without this disease.
In addition, when sickle cell disease patients experience a painful crisis, there is evidence that the platelet activation and abnormal blood clotting increase even further. Activated platelets release a substance called cluster of designation 40 ligand, which can increase how sticky the lining of blood vessels are and can increase the abnormal blood clotting. The level of cluster of designation 40 ligand is much higher in sickle cell disease patients compared to healthy individuals without this disease. In addition, the levels increase even further when sickle cell patients are experiencing a painful crisis.
Painful crisis represent the most common clinical problem in sickle cell disease, and are largely responsible for making the lives of these patients so unpredictable. However, the treatment of these painful crisis remains inadequate, consisting mainly of strong pain medications. In this study, we will evaluate the safety of eptifibatide in sickle cell patients and how well it works during the course of painful crises. At the completion of this trial, we will have an improved understanding of the contribution of platelet activation and inflammation to the problems in sickle cell disease.
The overall hypothesis that we seek to test is that increased platelet activation and the resultant inflammatory responses are important contributors to the problems of sickle cell disease. We believe that by decreasing platelet stickiness, and the release of mediators of inflammation and abnormal blood clotting, eptifibatide will affect the clinical course of complications in this disease.
If the results from our study support the hypothesis that eptifibatide is safe and effective in this population, we plan on carrying out larger studies to more definitively evaluate the safety of eptifibatide and how well it works in the treatment and/or prevention of painful crises in sickle cell disease.
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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North Carolina
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Chapel Hill, North Carolina, United States, 27599-7305
- University of North Carolina
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Age between 18 and 55 years
- Have confirmed diagnosis of sickle cell anemia or sickle beta zero thalassemia
- Have a serum creatinine </= 1.2 mg/dl
- Have serum transaminase values < 3 times upper limits of normal
- Have a platelet count >/= 150 x 10^9/L
- Have normal baseline coagulation profile
- Sudden onset of pain involving one or more sites and typical of usual pain episodes
- Have adequate intravenous access
- Be able to understand the requirements of the study and be willing to give informed consent
- Women of child-bearing age must be practicing (and will continue to practice for the course of the study) an adequate method of contraception (oral contraception, depo-provera, bilateral tubal ligation or barrier method)
Exclusion Criteria:
- Have a baseline hemoglobin < 6.0 gm/dl
- Have a history of major gastrointestinal bleeding or a bleeding diathesis
- Have an ongoing episode of acute chest syndrome
- Have a past history of clinically overt stroke(s)
- Have severe hypertension (systolic blood pressure > 200mmHg and/or diastolic BP >110mmHg) not adequately controlled on hypertensive medication
- Have had major surgery within the six weeks preceding enrollment
- Are pregnant or breastfeeding
- Are on chronic anticoagulation or antiplatelet (including non-steroidal anti-inflammatory drugs) therapy
- Have a history of metastatic cancer
- Are on a chronic transfusion program or have received a blood transfusion in the prior 8 weeks
- Have a positive urine toxicology screen for phencyclidine, cocaine or amphetamines.
- Have a history of alcohol abuse
- Have received any investigational drugs within the past 4 weeks.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: 1
As soon as eligible patients are identified and provide consent to participate in the study, patients randomized to the eptifibatide arm will receive two 180 mcg/kg boluses of eptifibatide 10 minutes apart (i.e., a double bolus), followed by a continuous infusion at 2 mcg/kg/min for 6 hours.
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Patients randomized to eptifibatide will receive two 180 mcg/kg boluses of eptifibatide 10 minutes apart (i.e., a double bolus), followed by a continuous infusion at 2 mcg/kg/min for 6 hours.
Other Names:
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Placebo Comparator: 2
As soon as eligible patients are identified and provide consent to participate in the study, patients randomized to the placebo arm will receive a saline solution delivered at a volume and rate identical to that of the active drug.
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Patients randomized to the placebo arm will receive a saline solution delivered at a volume and rate identical to that of the active drug.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
1) Major Bleeding Episodes
Time Frame: Up to 35 days
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Major bleeding episodes are defined as any episode, such as gastrointestinal bleeding or intracranial bleed that typically leads to hospitalization or other prolonged bleeding requiring a blood transfusion
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Up to 35 days
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Change in Platelet Count
Time Frame: Up to 35 days
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Change in platelet counts occurring anytime from randomization up to day 35 (final follow-up visit).
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Up to 35 days
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Effect of Eptifibatide on Duration of Acute Pain Episodes
Time Frame: Up to 7 days
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The duration of the pain episode will be defined as the time from randomization to termination of the pain episode. The pain episode will be considered terminated when the patient states that the crisis is resolved (defined as being ready to go home on oral analgesics) or all of the following criteria are met:
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Up to 7 days
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Effect of Eptifibatide on Duration of Hospitalization
Time Frame: Up to 7 days
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The duration of hospitalization will be defined as the period from randomization to the time an order for discharge from the hospital is written.
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Up to 7 days
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Collaborators and Investigators
Investigators
- Principal Investigator: Kenneth I Ataga, MD, University of North Carolina, Chapel Hill
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 1R21HL091265-01A1 (U.S. NIH Grant/Contract)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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