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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

Tracking Information
First Submitted Date  ICMJE November 21, 2012
First Posted Date  ICMJE December 4, 2012
Last Update Posted Date July 18, 2018
Actual Study Start Date  ICMJE December 2012
Actual Primary Completion Date December 21, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 30, 2012)
  • 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).
  • 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
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 30, 2012)
  • Secondary Efficacy Endpoint-Hemoglobin increment [ Time Frame: 12 months ]
    Hemoglobin increment one hour post-transfusion
  • 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)
  • 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.
  • 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.
  • 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
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion
Official Title  ICMJE 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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Other
Condition  ICMJE Thalassemia Major
Intervention  ICMJE
  • Biological: S-303 Treated Red Blood Cells (RBCs)
  • Biological: Conventional, untreated Red Blood Cells
Study Arms  ICMJE
  • 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.
    Intervention: 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.
    Intervention: Biological: Conventional, untreated Red Blood Cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 1, 2016)
86
Original Estimated Enrollment  ICMJE
 (submitted: November 30, 2012)
70
Actual Study Completion Date  ICMJE December 31, 2017
Actual Primary Completion Date December 21, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 10 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Italy,   Turkey
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01740531
Other Study ID Numbers  ICMJE CLI 00076
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Cerus Corporation
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Cerus Corporation
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
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
PRS Account Cerus Corporation
Verification Date July 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP