TRF-1101 Assessment in Sickle Cell Disease

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

80 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-07-31

Study Completion Date

2009-05-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Sickle Cell Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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TRF-1101

Daily treatment with TRF-1101

Group Type EXPERIMENTAL

TRF-1101

Intervention Type DRUG

once daily treatment with 300 mg orally for 12 weeks

Placebo

Daily treatment with placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Daily treatment with TRF-1101 vehicle for 12 weeks

Interventions

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TRF-1101

once daily treatment with 300 mg orally for 12 weeks

Intervention Type DRUG

Placebo

Daily treatment with TRF-1101 vehicle for 12 weeks

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No