Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion

NCT ID: NCT01740531

Last Updated: 2018-07-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 86 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-12-31
• Study Completion Date: 2017-12-31

Brief Summary

To evaluate the efficacy and safety of S 303 treated red blood cells (RBCs) in subjects who require chronic transfusion support due to thalassemia major.

Detailed Description

To evaluate the efficacy and safety of S 303 treated red blood cells (RBCs) in subjects who require chronic transfusion support due to thalassemia major.

Conditions

Thalassemia Major

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: OTHER
• Blinding Strategy: DOUBLE

Study Groups

S-303 Treated Red Blood Cells (RBC)

Patients will be randomly assigned to the sequence of administration of Test and Control RBCs; eligible patients are randomly assigned to receive Test RBCs followed by Control RBCs or Control RBCs followed by Test RBCs. Each patient will complete both treatment periods.

Group Type: EXPERIMENTAL

S-303 Treated Red Blood Cells (RBCs)

Intervention Type: BIOLOGICAL

Conventional, untreated Red Blood Cells

Patients will be randomly assigned to the sequence of administration of Test and Control RBCs; eligible patients are randomly assigned to receive Test RBCs followed by Control RBCs or Control RBCs followed by Test RBCs. Each patient will complete both treatment periods.

Group Type: ACTIVE_COMPARATOR

Conventional, untreated Red Blood Cells

Intervention Type: BIOLOGICAL

Interventions

S-303 Treated Red Blood Cells (RBCs)

Intervention Type: BIOLOGICAL

Conventional, untreated Red Blood Cells

Intervention Type: BIOLOGICAL

Eligibility Criteria

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

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 OR ≥18 cm in longitudinal diameter by ultrasound (chosen at the Investigator's discretion according to the data available with ultrasound data being preferable)
• Any subject for whom a transition in the number of RBC units transfused is anticipated within 12 months of study entry due to growth of the subject (e.g. a transition from 1 RBC component per transfusion cycle to 2 OR a transition from 2 to 3 is anticipated based on weight change alone)
• Alloimmunization to high frequency 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 infection (defined as RNA positive)
• HCV (hepatitis C)infection (defined as RNA positive) if treated with concomitant medications known to suppress the bone marrow
• 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

• Minimum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cerus Corporation

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Raffaella Origa, MD

Role: PRINCIPAL_INVESTIGATOR

Ospedale Regionale per le Microcitemie azienda

Antonio Piga, MD

Role: PRINCIPAL_INVESTIGATOR

University of Torino

Yesim Aydinok, MD

Role: PRINCIPAL_INVESTIGATOR

Ege University, Izmir, Turkey

Locations

Ospedale Regionale per le Microcitemie Azienda

Cagliari, , Italy

University of Torino

Torino, , Italy

Ege University

Izmir, , Turkey (Türkiye)

Countries

Italy; Turkey (Türkiye)

Other Identifiers

CLI 00076

Identifier Type: -

Identifier Source: org_study_id