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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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ClinicalTrials.gov Identifier: NCT01740531
Recruitment Status : Completed
First Posted : December 4, 2012
Last Update Posted : July 18, 2018
Sponsor:
Information provided by (Responsible Party):
Cerus Corporation

Study Description
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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.

Condition or disease Intervention/treatment Phase
Thalassemia Major Biological: S-303 Treated Red Blood Cells (RBCs) Biological: Conventional, untreated Red Blood Cells Phase 3

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 86 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Other
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
Actual Study Start Date : December 2012
Actual Primary Completion Date : December 21, 2017
Actual Study Completion Date : December 31, 2017

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 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.
 Biological: S-303 Treated Red Blood Cells (RBCs)
Active Comparator: 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.
 Biological: Conventional, untreated Red Blood Cells


Outcome Measures
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Primary Outcome Measures :
  1. Primary Efficacy Endpoint - Hemoglobin consumption [ Time Frame: 12 months ]
    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).

  2. Primary Safety Endpoint-Incidence of a treatment-emergent antibody with confirmed specificity to S 303 treated red blood cells (RBC) [ Time Frame: 12 months ]
    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 :
  1. Secondary Efficacy Endpoint-Hemoglobin increment [ Time Frame: 12 months ]
    Hemoglobin increment one hour post-transfusion

  2. Secondary Efficacy Endpoint-Proportional decline in post transfusion hemoglobin level per day (%/day) [ Time Frame: 12 months ]
    Proportional decline in post transfusion hemoglobin level per day (%/day)

  3. Secondary Safety Endpoint-Adverse Events [ Time Frame: 12 months ]
    Subjects will be actively monitored for adverse events during the transfusion episode and until discharge from the transfusion clinic.

  4. Secondary Safety Endpoint-Transfusion reactions within 24 hours [ Time Frame: 12 Months ]
    Transfusion reactions within 24 hours of a study transfusion with the assigned study product.

  5. Secondary Safety Endpoint-Frequency of allo immunization to red blood cell (RBC) allo-antigens [ Time Frame: 12 months ]
    Frequency of allo immunization to red blood cell (RBC) allo-antigens


Eligibility Criteria
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Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01740531


Locations
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Italy
Ospedale Regionale per le Microcitemie Azienda
Cagliari, Italy
University of Torino
Torino, Italy
Turkey
Ege University
Izmir, Turkey
Sponsors and Collaborators
Cerus Corporation
Investigators
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Principal Investigator: Raffaella Origa, MD Ospedale Regionale per le Microcitemie azienda
Principal Investigator: Antonio Piga, MD University of Torino
Principal Investigator: Yesim Aydinok, MD Ege University, Izmir, Turkey
More Information
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Responsible Party: Cerus Corporation
ClinicalTrials.gov Identifier: NCT01740531    
Other Study ID Numbers: CLI 00076
First Posted: December 4, 2012    Key Record Dates
Last Update Posted: July 18, 2018
Last Verified: July 2018
Keywords provided by Cerus Corporation:
S303 treated RBCs
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
 Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn