Clinical trial NCT00773890: TRF-1101 Assessment in Sickle Cell Disease
Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Activity of TRF-1101 on Microvascular Blood Flow, Vascular Endothelial Injury, and Vasoocclusive Pain in Patients With Sickle Cell Disease
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Sickle Cell Disease
Lead sponsor: TRF Pharma, Inc
Sponsor class: INDUSTRY
First posted: 2008-10-16
Enrollment: 80
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: endothelial cell injury/inflammation
timeFrame: Throughout trial
Secondary outcomes:
measure: Microvascular blood flow and trends in frequency of vasoocclusive pain
timeFrame: throughout trial
Other outcomes:
Locations:
city: Washington
country: United States
facility: Howard University
state: District of Columbia
city: Augusta
country: United States
facility: Medical College of Georgia
state: Georgia
city: Chicago
country: United States
facility: University of Illinios Medical Center
state: Illinois
city: Boston
country: United States
facility: Boston Medical Center
state: Massachusetts
city: Detroit
country: United States
facility: Wayne State University Medical Center
state: Michigan
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
Study officials:
affiliation: TRF Pharma, Inc
name: Stephen H Embury, M.D.
role: STUDY_DIRECTOR
References: