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

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2013 by Cerus Corporation
Sponsor:
Information provided by (Responsible Party):
Cerus Corporation
ClinicalTrials.gov Identifier:
NCT01740531
First received: November 21, 2012
Last updated: June 5, 2013
Last verified: June 2013
  Purpose

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

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Investigator)
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

Resource links provided by NLM:


Further study details as provided by Cerus Corporation:

Primary Outcome Measures:
  • Primary Efficacy Endpoint - Hemoglobin consumption [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    Hemoglobin increment one hour post-transfusion

  • Secondary Efficacy Endpoint-Proportional decline in post transfusion hemoglobin level per day (%/day) [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    Proportional decline in post transfusion hemoglobin level per day (%/day)

  • Secondary Safety Endpoint-Adverse Events [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: Yes ]
    Frequency of allo immunization to red blood cell (RBC) allo-antigens


Estimated Enrollment: 70
Study Start Date: December 2012
Estimated Study Completion Date: June 2014
Estimated Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01740531

Contacts
Contact: Raffaella Origa, MD 39 340 2453099 secondacasella@hotmail.com
Contact: Antonio Piga, MD 39 011 9026854 antonio.piga@unito.it

Locations
Italy
Ospedale Regionale per le Microcitemie Azienda Not yet recruiting
Cagliari, Italy
Contact: Raffaella Origa, MD    0039 340 2453099    secondacasella@hotmail.com   
Principal Investigator: Raffaella Origa, MD         
University of Torino Recruiting
Torino, Italy
Contact: Antonio Piga, MD    39 011 9026854    antonio.piga@unito.it   
Principal Investigator: Antonio Piga, MD         
Sponsors and Collaborators
Cerus Corporation
Investigators
Principal Investigator: Raffaella Origa, MD Ospedale Regionale per le Microcitemie azienda
Principal Investigator: Antonio Piga, MD University of Torino
  More Information

No publications provided

Responsible Party: Cerus Corporation
ClinicalTrials.gov Identifier: NCT01740531     History of Changes
Other Study ID Numbers: CLI 00076
Study First Received: November 21, 2012
Last Updated: June 5, 2013
Health Authority: Italy: Ethics Committee

Keywords provided by Cerus Corporation:
S303 treated RBCs

Additional relevant MeSH terms:
Beta-Thalassemia
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 20, 2014