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TRF-1101 Assessment in Sickle Cell Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00773890
Recruitment Status : Terminated (Study was stopped due to perceived futility because the baseline pain score in first 40 patients was too low to be able demonstrate an improvement)
First Posted : October 16, 2008
Last Update Posted : May 11, 2009
Information provided by:
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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: TRF-1101 Drug: Placebo Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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 Start Date : July 2008
Actual Primary Completion Date : May 2009
Actual Study Completion Date : May 2009

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: TRF-1101
Daily treatment with TRF-1101
Drug: TRF-1101
once daily treatment with 300 mg orally for 12 weeks

Placebo Comparator: Placebo
Daily treatment with placebo
Drug: Placebo
Daily treatment with TRF-1101 vehicle for 12 weeks

Primary Outcome Measures :
  1. endothelial cell injury/inflammation [ Time Frame: Throughout trial ]

Secondary Outcome Measures :
  1. Microvascular blood flow and trends in frequency of vasoocclusive pain [ Time Frame: throughout trial ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00773890

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United States, District of Columbia
Howard University
Washington, District of Columbia, United States, 20060
United States, Georgia
Medical College of Georgia
Augusta, Georgia, United States, 30912
United States, Illinois
University of Illinios Medical Center
Chicago, Illinois, United States, 60612
United States, Massachusetts
Boston Medical Center
Boston, Massachusetts, United States, 02118
United States, Michigan
Wayne State University Medical Center
Detroit, Michigan, United States, 48201
United States, North Carolina
University of North Carolina
Chapel Hill, North Carolina, United States, 27599
Sponsors and Collaborators
TRF Pharma, Inc
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Study Director: Stephen H Embury, M.D. TRF Pharma, Inc
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Responsible Party: Edward Monaghan, Vice President, Clinical Operations, TRF Pharma, Inc. Identifier: NCT00773890    
Other Study ID Numbers: 1101-201
First Posted: October 16, 2008    Key Record Dates
Last Update Posted: May 11, 2009
Last Verified: May 2009
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn