TRF-1101 Assessment in Sickle Cell Disease

Sickle Cell Disease Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT00773890
• Other study ID #: 1101-201
• Status: Terminated
• Phase: Phase 1/Phase 2
• First received: October 14, 2008
• Last updated: May 7, 2009
• Start date: July 2008
• Est. completion date: May 2009

Study information

• Verified date: May 2009
• Source: TRF Pharma, Inc
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 80
• Est. completion date: May 2009
• Est. primary completion date: May 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Pharmacokinetics/Dynamics Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

Drug:
• TRF-1101
once daily treatment with 300 mg orally for 12 weeks
• Placebo
Daily treatment with TRF-1101 vehicle for 12 weeks

Locations

Country	Name	City	State
United States	Medical College of Georgia	Augusta	Georgia
United States	Boston Medical Center	Boston	Massachusetts
United States	University of North Carolina	Chapel Hill	North Carolina
United States	University of Illinios Medical Center	Chicago	Illinois
United States	Wayne State University Medical Center	Detroit	Michigan
United States	Howard University	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
TRF Pharma, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	endothelial cell injury/inflammation		Throughout trial	No
Secondary	Microvascular blood flow and trends in frequency of vasoocclusive pain		throughout trial	No