ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01740531
Recruitment Status : Active, not recruiting
First Posted : December 4, 2012
Last Update Posted : December 27, 2017
Sponsor:
Information provided by (Responsible Party):
Cerus Corporation

November 21, 2012
December 4, 2012
December 27, 2017
December 2012
December 21, 2017   (Final data collection date for primary outcome measure)
  • 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
Same as current
Complete list of historical versions of study NCT01740531 on ClinicalTrials.gov Archive Site
  • 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
Same as current
Not Provided
Not Provided
 
Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion
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
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.
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.
Interventional
Phase 3
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Other
Thalassemia Major
  • Biological: S-303 Treated Red Blood Cells (RBCs)
  • Biological: Conventional, untreated Red Blood Cells
  • 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
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
86
70
March 2018
December 21, 2017   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
10 Years and older   (Child, Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Italy,   Turkey
 
 
NCT01740531
CLI 00076
No
Not Provided
Not Provided
Cerus Corporation
Cerus Corporation
Not Provided
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
Cerus Corporation
December 2017

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