A Safety Study of Eptifibatide in Patients With Sickle Cell Disease

NCT00834899 | Terminated Phase 1 Results DMC

• 1 other identifier: 1R21HL091265-01A1
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 1

Brief Summary

This study will evaluate the safety of eptifibatide in sickle cell patients and how well it works during the course of painful crises. 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. Sickle cell disease has been referred to both as a condition associated with increased risk of blood clots and increased inflammation. A painful crisis represents the most common cli nical problem in sickle cell disease, but the treatment of these crises remains inadequate.

Conditions

Sickle Cell Disease

Keywords

Sickle cell disease; Pain crisis; Acute pain episode; Eptifibatide; Antiplatelet therapy; Safety; Treatment

Outcome Measures

Primary Outcomes (2)

• 1) Major Bleeding Episodes

  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

  Up to 35 days

• Change in Platelet Count

  Change in platelet counts occurring anytime from randomization up to day 35 (final follow-up visit).

  Up to 35 days

Secondary Outcomes (2)

• Effect of Eptifibatide on Duration of Acute Pain Episodes

  Up to 7 days

• Effect of Eptifibatide on Duration of Hospitalization

  Up to 7 days

Study Arms (2)

1

EXPERIMENTAL

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.

Drug: Eptifibatide

2

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

Eptifibatide DRUG

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.

Also known as: Integrilin

1

Placebo DRUG

Patients randomized to the placebo arm will receive a saline solution delivered at a volume and rate identical to that of the active drug.

2

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of North Carolina, Chapel Hill: lead

Study Sites (1)

University of North Carolina

Chapel Hill, North Carolina, 27599-7305, United States

Location

Related Publications (1)

• Desai PC, Brittain JE, Jones SK, McDonald A, Wilson DR, Dominik R, Key NS, Parise LV, Ataga KI. A pilot study of eptifibatide for treatment of acute pain episodes in sickle cell disease. Thromb Res. 2013 Sep;132(3):341-5. doi: 10.1016/j.thromres.2013.08.002. Epub 2013 Aug 8.

  PMID: 23973010 DERIVED

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Eptifibatide

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Peptides, Cyclic; Peptides; Amino Acids, Peptides, and Proteins

Limitations and Caveats

The study was terminated early resulting in small numbers of subjects analyzed.

Results Point of Contact

• Title: Kenneth I. Ataga, MD
• Organization: University of North Carolina at Chapel Hill

kataga@med.unc.edu

919-966-0178

Study Officials

• Kenneth I Ataga, MD

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 31, 2009
• First Posted: February 3, 2009
• Study Start: January 1, 2009
• Primary Completion: March 1, 2012
• Study Completion: March 1, 2012
• Last Updated: June 27, 2013
• Results First Posted: June 27, 2013

Record last verified: 2013-02

Locations

US (1)