TRF-1101 Assessment in Sickle Cell Disease
TRF-1101 Assessment in Sickle Cell Disease
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.
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.