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

NCT01740531 | Completed Phase 3

• 1 other identifier: CLI 00076
• Study Type: interventional
• Target: 86
• Locations: 2 countries
• Sites: 3

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.

Conditions

Thalassemia Major

Keywords

S303 treated RBCs

Outcome Measures

Primary Outcomes (2)

• 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).

  12 months

• 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

  12 months

Secondary Outcomes (5)

• Secondary Efficacy Endpoint-Hemoglobin increment

  12 months

• Secondary Efficacy Endpoint-Proportional decline in post transfusion hemoglobin level per day (%/day)

  12 months

• Secondary Safety Endpoint-Adverse Events

  12 months

• Secondary Safety Endpoint-Transfusion reactions within 24 hours

  12 Months

• Secondary Safety Endpoint-Frequency of allo immunization to red blood cell (RBC) allo-antigens

  12 months

Study Arms (2)

S-303 Treated Red Blood Cells (RBC)

EXPERIMENTAL

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)

Conventional, untreated Red Blood Cells

ACTIVE COMPARATOR

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

Interventions

S-303 Treated Red Blood Cells (RBCs) BIOLOGICAL

S-303 Treated Red Blood Cells (RBC)

Conventional, untreated Red Blood Cells BIOLOGICAL

Conventional, untreated Red Blood Cells

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)
• 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

Sponsors & Collaborators

• Cerus Corporation: lead

Study Sites (3)

Ospedale Regionale per le Microcitemie Azienda

Cagliari, Italy

Location

University of Torino

Torino, Italy

Location

Ege University

Izmir, Turkey (Türkiye)

Location

MeSH Terms

Conditions

beta-Thalassemia

Interventions

Congresses as Topic

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Organizations; Health Care Economics and Organizations

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: OTHER
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 21, 2012
• First Posted: December 4, 2012
• Study Start: December 1, 2012
• Primary Completion: December 21, 2017
• Study Completion: December 31, 2017
• Last Updated: July 18, 2018

Record last verified: 2018-07

Locations

TU (1); IT (2)