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

Primary Purpose

Thalassemia Major

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
S-303 Treated Red Blood Cells (RBCs)
Conventional, untreated Red Blood Cells
Sponsored by
Cerus Corporation
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Thalassemia Major focused on measuring S303 treated RBCs

Eligibility Criteria

10 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Ospedale Regionale per le Microcitemie Azienda
  • University of Torino
  • Ege University

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

S-303 Treated Red Blood Cells (RBC)

Conventional, untreated Red Blood Cells

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

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).
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

Secondary Outcome Measures

Secondary Efficacy Endpoint-Hemoglobin increment
Hemoglobin increment one hour post-transfusion
Secondary Efficacy Endpoint-Proportional decline in post transfusion hemoglobin level per day (%/day)
Proportional decline in post transfusion hemoglobin level per day (%/day)
Secondary Safety Endpoint-Adverse Events
Subjects will be actively monitored for adverse events during the transfusion episode and until discharge from the transfusion clinic.
Secondary Safety Endpoint-Transfusion reactions within 24 hours
Transfusion reactions within 24 hours of a study transfusion with the assigned study product.
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

Full Information

First Posted
November 21, 2012
Last Updated
July 16, 2018
Sponsor
Cerus Corporation
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1. Study Identification

Unique Protocol Identification Number
NCT01740531
Brief Title
Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2018
Overall Recruitment Status
Completed
Study Start Date
December 2012 (Actual)
Primary Completion Date
December 21, 2017 (Actual)
Study Completion Date
December 31, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cerus Corporation

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thalassemia Major
Keywords
S303 treated RBCs

7. Study Design

Primary Purpose
Other
Study Phase
Phase 3
Interventional Study Model
Crossover Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
86 (Actual)

8. Arms, Groups, and Interventions

Arm Title
S-303 Treated Red Blood Cells (RBC)
Arm Type
Experimental
Arm Description
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.
Arm Title
Conventional, untreated Red Blood Cells
Arm Type
Active Comparator
Arm Description
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.
Intervention Type
Biological
Intervention Name(s)
S-303 Treated Red Blood Cells (RBCs)
Intervention Type
Biological
Intervention Name(s)
Conventional, untreated Red Blood Cells
Primary Outcome Measure Information:
Title
Primary Efficacy Endpoint - Hemoglobin consumption
Description
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).
Time Frame
12 months
Title
Primary Safety Endpoint-Incidence of a treatment-emergent antibody with confirmed specificity to S 303 treated red blood cells (RBC)
Description
Incidence of a treatment-emergent antibody with confirmed specificity to S 303 treated red blood cells (RBC) associated with clinically significant hemolysis
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Secondary Efficacy Endpoint-Hemoglobin increment
Description
Hemoglobin increment one hour post-transfusion
Time Frame
12 months
Title
Secondary Efficacy Endpoint-Proportional decline in post transfusion hemoglobin level per day (%/day)
Description
Proportional decline in post transfusion hemoglobin level per day (%/day)
Time Frame
12 months
Title
Secondary Safety Endpoint-Adverse Events
Description
Subjects will be actively monitored for adverse events during the transfusion episode and until discharge from the transfusion clinic.
Time Frame
12 months
Title
Secondary Safety Endpoint-Transfusion reactions within 24 hours
Description
Transfusion reactions within 24 hours of a study transfusion with the assigned study product.
Time Frame
12 Months
Title
Secondary Safety Endpoint-Frequency of allo immunization to red blood cell (RBC) allo-antigens
Description
Frequency of allo immunization to red blood cell (RBC) allo-antigens
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Raffaella Origa, MD
Organizational Affiliation
Ospedale Regionale per le Microcitemie azienda
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Antonio Piga, MD
Organizational Affiliation
University of Torino
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Yesim Aydinok, MD
Organizational Affiliation
Ege University, Izmir, Turkey
Official's Role
Principal Investigator
Facility Information:
Facility Name
Ospedale Regionale per le Microcitemie Azienda
City
Cagliari
Country
Italy
Facility Name
University of Torino
City
Torino
Country
Italy
Facility Name
Ege University
City
Izmir
Country
Turkey

12. IPD Sharing Statement

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

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