TRF-1101 Assessment in Sickle Cell Disease

NCT00773890 | Terminated Phase 1 DMC

• 1 other identifier: 1101-201
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 6

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.

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• endothelial cell injury/inflammation

  Throughout trial

Secondary Outcomes (1)

• Microvascular blood flow and trends in frequency of vasoocclusive pain

  throughout trial

Study Arms (2)

TRF-1101

EXPERIMENTAL

Daily treatment with TRF-1101

Drug: TRF-1101

Placebo

PLACEBO COMPARATOR

Daily treatment with placebo

Drug: Placebo

Interventions

TRF-1101 DRUG

once daily treatment with 300 mg orally for 12 weeks

TRF-1101

Placebo DRUG

Daily treatment with TRF-1101 vehicle for 12 weeks

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• TRF Pharma, Inc: lead

Study Sites (6)

Howard University

Washington D.C., District of Columbia, 20060, United States

Location

Medical College of Georgia

Augusta, Georgia, 30912, United States

Location

University of Illinios Medical Center

Chicago, Illinois, 60612, United States

Location

Boston Medical Center

Boston, Massachusetts, 02118, United States

Location

Wayne State University Medical Center

Detroit, Michigan, 48201, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27599, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Stephen H Embury, M.D.

  TRF Pharma, Inc

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: October 14, 2008
• First Posted: October 16, 2008
• Study Start: July 1, 2008
• Primary Completion: May 1, 2009
• Study Completion: May 1, 2009
• Last Updated: May 11, 2009

Record last verified: 2009-05

Locations

US (6)