A Randomized Controlled Study to Evaluate Efficacy and Safety of S-303 Treated Red Blood Cells in Subjects with Thalassemia Major Requiring Chronic RBC Transfusio

• Conditions: Subjects with Thalassemia Major Requiring Chronic RBC Transfusion; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2012-002920-33-IT
• Lead Sponsor: CERUS CORPORATIO

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-10-16
• Last Update Posted: 28 December 2015

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

- Age >= 10 years, of either gender - Diagnosed with thalassemia major and currently participating in a chronic transfusion program - At least a one year history of chronic RBC transfusion support with a stable transfusion requirement (per treating physician) - Intervals of at least 14 days between RBC transfusions - All RBC components are given on one day for each transfusion episode - Negative direct antiglobulin tests (DAT) - Stable iron chelation regimen - Available for measurement of hemoglobin level at one hour post transfusion - Signed and dated informed consent form
Are the trial subjects under 18? yes
Number of subjects for this age range: 35
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 35
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Baseline antibody specific to S-303 treated RBC (positive test, as defined in Section 8.4.1) - Evidence of splenic hyper function defined as a transfusion requirement >180 cc/kg/year (at 100% hematocrit) - Splenic enlargement: spleen palpable =4 cm below costal margin - Alloimmunization to minor blood group antigens to the extent that the ready provision of compatible blood may not be feasible for the study (alloimmunization alone is not an automatic exclusion) - Current specialized treatment with washed or frozen RBC Requirement for gamma irradiated RBC components (would present blinding difficulty due to blood component labeling regulations - Treatment with any medication that is known to adversely affect RBC viability - HIV or HCV infection (defined as RNA positive) - Pregnant or breast feeding female, or female of child-bearing potential not using a medically approved form of contraception - Acute or chronic medical disorder other than thalassemia that, in the opinion of the Investigator or medical monitor, may prevent the subject from completing participation in the study - Participation in another clinical study, either concurrently or within the previous 28 days, in which the study drug or device may influence red blood cell viability

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The objectives of this study are to evaluate the efficacy and safety of S 303 treated RBCs in subjects who require chronic transfusion support due to thalassemia major;Secondary Objective: N/A;Primary end point(s): The primary efficacy endpoint is: - Hemoglobin consumption measured as total hemoglobin mass transfused per subject adjusted for average body weight and the number of days during the efficacy evaluation period (adjusted Hgb consumption units are g Hgb/kg body weight/day). The primary safety endpoint is: - Incidence of a treatment-emergent antibody with confirmed specificity to S-303 treated RBC associated with clinically significant hemolysis;Timepoint(s) of evaluation of this end point: Study participation is expected to require approximately 12 months.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Secondary Efficacy Endpoints The secondary efficacy endpoints are: ? Hemoglobin increment one hour post-transfusion ? Proportional decline in post transfusion hemoglobin level per day (%/day) The secondary safety endpoints are: - Adverse events - Transfusion reactions within 24 hours of a study transfusion with the assigned study product - Frequency of allo-immunization to RBC allo-antigens;Timepoint(s) of evaluation of this end point: Study participation is expected to require approximately 12 months.