TRF-1101 Assessment in Sickle Cell Disease

This study has been 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)
Sponsor:
Information provided by:
TRF Pharma, Inc
ClinicalTrials.gov Identifier:
NCT00773890
First received: October 14, 2008
Last updated: May 7, 2009
Last verified: May 2009
  Purpose

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 Intervention Phase
Sickle Cell Disease
Drug: TRF-1101
Drug: Placebo
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Pharmacokinetics/Dynamics Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:


Further study details as provided by TRF Pharma, Inc:

Primary Outcome Measures:
  • endothelial cell injury/inflammation [ Time Frame: Throughout trial ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Microvascular blood flow and trends in frequency of vasoocclusive pain [ Time Frame: throughout trial ] [ Designated as safety issue: No ]

Estimated Enrollment: 80
Study Start Date: July 2008
Study Completion Date: May 2009
Primary Completion Date: May 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00773890

Locations
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
Investigators
Study Director: Stephen H Embury, M.D. TRF Pharma, Inc
  More Information

No publications provided

Responsible Party: Edward Monaghan, Vice President, Clinical Operations, TRF Pharma, Inc.
ClinicalTrials.gov Identifier: NCT00773890     History of Changes
Other Study ID Numbers: 1101-201
Study First Received: October 14, 2008
Last Updated: May 7, 2009
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies

ClinicalTrials.gov processed this record on October 23, 2014