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

Thalassemia Major Clinical Trial

Official title:

A Randomized Controlled Study to Evaluate Efficacy and Safety of S 303 Treated Red Blood Cells (RBC) in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01740531
• Other study ID #: CLI 00076
• Status: Completed
• Phase: Phase 3
• Start date: December 2012
• Est. completion date: December 31, 2017

Study information

• Verified date: July 2018
• Source: Cerus Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 86
• Est. completion date: December 31, 2017
• Est. primary completion date: December 21, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years and older

Eligibility

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

Related Conditions & MeSH terms

• beta-Thalassemia
• Thalassemia
• Thalassemia Major

Intervention

Biological:
• S-303 Treated Red Blood Cells (RBCs)

• Conventional, untreated Red Blood Cells

Locations

Country	Name	City	State
Italy	Ospedale Regionale per le Microcitemie Azienda	Cagliari
Italy	University of Torino	Torino
Turkey	Ege University	Izmir

Sponsors (1)

Lead Sponsor	Collaborator
Cerus Corporation

Countries where clinical trial is conducted

Italy,  Turkey, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Primary Efficacy Endpoint - Hemoglobin consumption	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 hemoglobin (Hgb) consumption units are g Hgb/kg body weight/day).	12 months
Primary	Primary Safety Endpoint-Incidence of a treatment-emergent antibody with confirmed specificity to S 303 treated red blood cells (RBC)	Incidence of a treatment-emergent antibody with confirmed specificity to S 303 treated red blood cells (RBC) associated with clinically significant hemolysis	12 months
Secondary	Secondary Efficacy Endpoint-Hemoglobin increment	Hemoglobin increment one hour post-transfusion	12 months
Secondary	Secondary Efficacy Endpoint-Proportional decline in post transfusion hemoglobin level per day (%/day)	Proportional decline in post transfusion hemoglobin level per day (%/day)	12 months
Secondary	Secondary Safety Endpoint-Adverse Events	Subjects will be actively monitored for adverse events during the transfusion episode and until discharge from the transfusion clinic.	12 months
Secondary	Secondary Safety Endpoint-Transfusion reactions within 24 hours	Transfusion reactions within 24 hours of a study transfusion with the assigned study product.	12 Months
Secondary	Secondary Safety Endpoint-Frequency of allo immunization to red blood cell (RBC) allo-antigens	Frequency of allo immunization to red blood cell (RBC) allo-antigens	12 months