Thalassemia MajorPhase 3NCT01740531
Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion
A Phase 3 study testing S-303 Treated Red Blood Cells (RBCs) and Conventional, untreated Red Blood Cells in people with Thalassemia Major, run by Cerus Corporation at 3 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Thalassemia Major
- What is being tested?
- S-303 Treated Red Blood Cells (RBCs) and Conventional, untreated Red Blood Cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 86 participants
- Where
- 3 sitesin 2 countries
- Study dates
- Dec 2012to 31 Dec 2017
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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)
Show all 9Show fewer
- Stable iron chelation regimen
- Available for measurement of hemoglobin level at one hour post transfusion
- Signed and dated informed consent form
Who cannot join
- 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
Show all 13Show fewer
- 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
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
Italy2 sites
Ospedale Regionale per le Microcitemie Azienda
Cagliari
University of Torino
Torino
About this study
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
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.
Read the 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.
- Conditions
- Thalassemia Major
- Being tested
- S-303 Treated Red Blood Cells (RBCs), Conventional, untreated Red Blood Cells
- Keywords
- S303 treated RBCs
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
S-303 Treated Red Blood Cells (RBCs)Biological
Conventional, untreated Red Blood CellsBiological
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Biological: Conventional, untreated Red Blood Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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
Measured: 12 months
Secondary outcomes (5)
Secondary Efficacy Endpoint-Hemoglobin increment
Hemoglobin increment one hour post-transfusion
Measured: 12 months
Secondary Efficacy Endpoint-Proportional decline in post transfusion hemoglobin level per day (%/day)
Proportional decline in post transfusion hemoglobin level per day (%/day)
Measured: 12 months
Secondary Safety Endpoint-Adverse Events
Subjects will be actively monitored for adverse events during the transfusion episode and until discharge from the transfusion clinic.
Measured: 12 months
Secondary Safety Endpoint-Transfusion reactions within 24 hours
Transfusion reactions within 24 hours of a study transfusion with the assigned study product.
Measured: 12 Months
Secondary Safety Endpoint-Frequency of allo immunization to red blood cell (RBC) allo-antigens
Frequency of allo immunization to red blood cell (RBC) allo-antigens
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2012 (estimated)
- Study startDec 2012
- Primary completion21 Dec 2017
- Study completion31 Dec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia MajorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 86 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cerus Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Raffaella Origa, MD, Principal investigator, Ospedale Regionale per le Microcitemie aziendaAntonio Piga, MD, Principal investigator, University of TorinoYesim Aydinok, MD, Principal investigator, Ege University, Izmir, Turkey
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01740531
- Sponsor's protocol ID
- CLI 00076
- Status last verified
- Jul 2018
- First submitted
- 21 Nov 2012
- Last updated
- 18 Jul 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01740531
- Acronym / sponsor ID
- CLI 00076
- Conditions
- Thalassemia Major
- Keywords
- S303 treated RBCs
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 86 participants (actual)
- Lead sponsor
- Cerus CorporationIndustry (pharma, biotech, devices)
- Study start
- Dec 2012 (actual)
- Primary completion
- 21 Dec 2017 (actual)
- Study completion
- 31 Dec 2017 (actual)
- First posted
- 4 Dec 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| S-303 Treated Red Blood Cells (RBC) | Experimental | Biological: S-303 Treated Red Blood Cells (RBCs)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. |
| Conventional, untreated Red Blood Cells | Active comparator | Biological: Conventional, untreated Red Blood CellsPatients 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. |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (13 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 |
Primary outcome
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
5 secondary outcomes
Secondary Efficacy Endpoint-Hemoglobin increment
Time frame: 12 months
Secondary Efficacy Endpoint-Proportional decline in post transfusion hemoglobin level per day (%/day)
Time frame: 12 months
Secondary Safety Endpoint-Adverse Events
Time frame: 12 months
Secondary Safety Endpoint-Transfusion reactions within 24 hours
Time frame: 12 Months
Secondary Safety Endpoint-Frequency of allo immunization to red blood cell (RBC) allo-antigens
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2018.
Related trials for Thalassemia Major
Active, not recruiting
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
Transfusion-dependent Beta-Thalassemia
RecruitingObservational study
Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
Transfusion-dependent Beta-Thalassemia, Gene Therapy
Not yet recruiting
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
Beta-Thalassemia
RecruitingPhase 1/2
β-Thalassemia Treatment With KL003 Cell Injection
Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Raffaella Origa, MD principal investigator
Ospedale Regionale per le Microcitemie azienda
Antonio Piga, MD principal investigator
University of Torino
Yesim Aydinok, MD principal investigator
Ege University, Izmir, Turkey
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing S-303 Treated Red Blood Cells (RBCs) and Conventional, untreated Red Blood Cells in people with Thalassemia Major, run by Cerus Corporation at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Jul 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Italy, Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Efficacy Endpoint - Hemoglobin consumption (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jul 2018.