TRF-1101 Assessment in Sickle Cell Disease
- Registration Number
- NCT00773890
- Lead Sponsor
- TRF Pharma, Inc
- Brief Summary
This study is designed to assess the safety, tolerability, and activity of TRF-1101 on microvascular blood flow, vascular endothelial injury, and vasoocclusive pain associated with sickle cell disease.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 80
Inclusion Criteria
- Be 18 years of age or older at the time of informed consent;
- Have a diagnosis of homozygous sickle cell anemia or sickle cell-beta° thalassemia;
- Have had 2 - 10 documented pain crises in the past year (pain crises are defined as visits to a medical clinic, Emergency Department or hospital, being bedridden and requiring constant analgesia at home for at least three days, or having a three-day interruption of life's activities [i.e., school, work, planned leisure activity] because of pain);
- If female and of child bearing potential, have a negative serum or urine pregnancy test and be using an effective birth-control method with a history of reliability for the individual patient (use of mifepristone is not allowed);
- Be properly informed of the nature and risks of the clinical investigation, be willing and able to comply with all clinical investigation-related procedures and assessments, and sign an Institutional Review Board (IRB) approved Informed Consent Form prior to entering the clinical investigation.
Exclusion Criteria
- Have a history of abnormal bleeding, stroke, moya moya vascular malformations, or any other contraindication to anticoagulation;
- Be currently taking anticoagulant or thrombolytic medication;
- Be currently taking an endothelin receptor antagonist, e.g., bosentan (Tracleer®);
- Have a known sensitivity or allergy to heparin or related drugs;
- Have a history of thrombocytopenia (platelet count < 100 x 103/mm3) induced by heparin or related drugs;
- Have had fewer than 2 documented pain crises in the past year;
- Have had a pain crisis within one month of screening or randomization;
- If currently on or recently discontinued hydroxyurea treatment, have initiated or discontinued treatment or changed regimen within the past 6 months;
- Have had a transfusion within last 120 days or have HbA% > 15% from prior transfusion;
- Creatinine levels > 1.53 mg/dL (135 umol/L);
- ALT levels ≥ 3 times normal;
- Platelet count < 100 x 103/mm3;
- INR > 2.0;
- Be unable to tolerate oral medications;
- Have unreliable venous access;
- Be noncompliant with regular care;
- Have a positive pregnancy test, be currently lactating, or be trying to become pregnant;
- Have participation in an investigational drug or medical device study within previous 30 days;
- Have any other condition or circumstance that in the opinion of the Investigator makes the patient a poor candidate for participation in the study.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description TRF-1101 TRF-1101 Daily treatment with TRF-1101 Placebo Placebo Daily treatment with placebo
- Primary Outcome Measures
Name Time Method endothelial cell injury/inflammation Throughout trial
- Secondary Outcome Measures
Name Time Method Microvascular blood flow and trends in frequency of vasoocclusive pain throughout trial
Trial Locations
- Locations (6)
Boston Medical Center
🇺🇸Boston, Massachusetts, United States
University of North Carolina
🇺🇸Chapel Hill, North Carolina, United States
Medical College of Georgia
🇺🇸Augusta, Georgia, United States
Wayne State University Medical Center
🇺🇸Detroit, Michigan, United States
University of Illinios Medical Center
🇺🇸Chicago, Illinois, United States
Howard University
🇺🇸Washington, District of Columbia, United States