TRF-1101 Assessment in Sickle Cell Disease

TRF Pharma

Status and phase

Terminated

Phase 2

Phase 1

Conditions

Sickle Cell Disease

Treatments

Drug: TRF-1101

Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT00773890

1101-201

Details and patient eligibility

About

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.

Enrollment

80 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.