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TRF-1101 Assessment in Sickle Cell Disease

Sponsor
TRF Pharma, Inc (Industry)
Overall Status
Terminated
CT.gov ID
NCT00773890
Collaborator
(none)
80
Enrollment
6
Locations
2
Arms
10
Duration (Months)
13.3
Patients Per Site
1.3
Patients Per Site Per Month

Study Details

Study Description

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
Phase 1/Phase 2

Study Design

Study Type:
Interventional
Anticipated 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 :
Jul 1, 2008
Actual Primary Completion Date :
May 1, 2009
Actual Study Completion Date :
May 1, 2009

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

  1. endothelial cell injury/inflammation [Throughout trial]

Secondary Outcome Measures

  1. Microvascular blood flow and trends in frequency of vasoocclusive pain [throughout trial]

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Howard University Washington District of Columbia United States 20060
2 Medical College of Georgia Augusta Georgia United States 30912
3 University of Illinios Medical Center Chicago Illinois United States 60612
4 Boston Medical Center Boston Massachusetts United States 02118
5 Wayne State University Medical Center Detroit Michigan United States 48201
6 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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
, ,
ClinicalTrials.gov Identifier:
NCT00773890
Other Study ID Numbers:
  • 1101-201
First Posted:
Oct 16, 2008
Last Update Posted:
May 11, 2009
Last Verified:
May 1, 2009
Additional relevant MeSH terms:
Anemia, Sickle Cell

Study Results

No Results Posted as of May 11, 2009