Thalassemia MajorPhase 4NCT03992001
Impact of the Preparation Method of Red Cell Concentrates on Transfusion Indices in Thalassemic Patients
A Phase 4 study testing Blood component A and Blood component B in people with Thalassemia Major, run by Università degli Studi di Ferrara at 1 site in Italy.
Trial summary
This study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients. The two blood components types derive from the whole blood. In one case, the whole blood is leukoreduced with subsequent plasma removal. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thalassemia Major
- What is being tested?
- Blood component A and Blood component B
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 55 participants
- Where
- 1 siteItaly
- Study dates
- 14 May 2018to 31 Jul 2019
- Phase
- Phase 4
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient with beta(ß)-Thalassaemia transfusion-dependent (ß-Thalassemia Major or ß-Thalassemia Intermedia transfusion-dependent, regularly transfused since at least 5 years
Who cannot join
- Patient not exclusively transfused at Day Hospital Thalassaemia and Haemoglobinopathies of Ferrara
- Patient with haemolytic auto-antibodies
- Patient transfused with washed Packet RBCs units
- Severe splenomegaly (>18 cm on echography)
- Elevated blood consumption (>200 mL/kg of pure RBCs in the last year)
- Patient receiving haemoglobin inducers in the last 6 months
Show all 9Show fewer
- Any significant clinical pulmonary, cardiovascular, endocrine, hepatic, gastrointestinal, renal, infectious, immunological including significant allo- or auto-immunisation disease, considered not adequately controlled prior to the study
- Patient treated with erythrocyte exchange
- Pregnant females
Trial locations and contacts
1 site in 1 country.
Italy1 site
Day Hospital Thalassaemia and Haemoglobinopathies (DHTE)
Ferrara, 44134
About this study
Official title: Prospective Crossover Study on Beta(ß)-Thalassaemia Transfusion-dependent to Evaluate the Impact on Transfusion Regimen of Two Pre-storage Leukoreduced PRBCs(In-line Filtration + B-C Separation; Whole Blood Filtration + B-C Conservation)
This study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients. The two blood components types derive from the whole blood. In one case, the whole blood is leukoreduced with subsequent plasma removal. In the other case, plasma, buffy coat, and red blood cells (RBCs) are first separated and subsequently, the RBCs leukoreduced. Each type of blood components will be subsequently given to one-half of the patients for a 6-month period and to the other half for other 6-month at the randomization phase, for a total of 12 months of crossed-treatment per patient.
Read the detailed description
At Day Hospital Talassemia ed Emoglobinopatie of Ferrara, two different PRBCs are available. The two types of blood components are obtained from whole blood, pre-storage leukoreduced and suspended in saline-adenine-glucose-mannitol (SAGM). One method of preparation consists of the whole blood leukoreduction with subsequent plasma removal. The other method first separates plasma, buffy coat, and RBCs, and then the RBCs are leukoreduced. The two methods mainly differ in the final haemoglobin (Hb) content: the Hb level is lower (-13%, approximately) in the second method that also shows the advantage to produce platelets from the buffy coat. A PRBCs unit is not as strictly defined as a therapeutic medication dose (pill or vial): individual PRBCs units may substantially differ in their Hb content, much more than the average difference between the two types of preparations. The aim of this study is to document the extent of the average difference between the two types of preparations, and its impacts on the transfusion indices of ß-Thalassaemia transfusion-dependent patients. All patients will receive each blood component for a period of 6 months (crossover design), for a total of 12 months of transfusion treatment per patient.
- Conditions
- Thalassemia Major
- Being tested
- Blood component A, Blood component B
- Keywords
- Packed RBCs, leukodepletion, buffy-coat, transfusion
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Blood component ABiological
PRBCs obtained from whole blood after separation of plasma, buffy coat, and RBCs and successive leukoreduction of RBCs
Blood component BBiological
PRBCs obtained by leukoreduction of whole blood, and successive separation of plasma and RBCs
| Study group | Role | What they receive |
|---|---|---|
| Sequence A-B | Experimental | Patients in this arm will receive blood component A for 6 months and blood component B for the next 6 months Receives: Biological: Blood component A; Biological: Blood component B |
| Sequence B-A | Experimental | Patients in this arm will receive blood component B for 6 months and blood component A for the next 6 months Receives: Biological: Blood component A; Biological: Blood component B |
What is being measured
The primary outcome is the main question the study is designed to answer.
Transfusion Power Index
(Average pre-transfusion Hb concentration)/(Unit Index) [for the definition of Unit Index, see the secondary outcomes]
Measured: For each of the two blood components studied, at the end of 6-month period of study
Secondary outcomes (5)
Average pre-transfusion Hb concentration
Mean pre-transfusion Hb levels, calculated starting from the second transfusion of the period to the first transfusion of the following period
Measured: For each of the two blood components studied, at the end of 6-month period of study
Unit Index
(Total number of PRBCs (A or B) transfused in the period)/(Number of days between the first transfusion of the period and the first transfusion of the following period)
Measured: For each of the two blood components studied, at the end of 6-month period of study
Average Transfusion Interval
(Number of days between the first transfusion of the period and the first transfusion of the following period)/(Number of transfusions in the period)
Measured: For each of the two blood components studied, at the end of 6-month period of study
Number of Transfusion Reactions
Number of transfusion reactions to the two blood components that may occur in the study periods (2 periods of 6 months each)
Measured: Study periods (2 periods of 6 months each)
Transfusion Reaction Rate
(Number of transfusion reactions to the two blood components occurring in the study periods)/(Total number of PRBCs (A or B) transfused in the period)
Measured: Study periods (2 periods of 6 months each)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jun 2019
- Study start14 May 2018
- Primary completion31 Jul 2019
- Study completion31 Jul 2019
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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Patients will be divided into two groups of approximately equal number. The first group will receive the blood component A for a period of 6 months and then the blood component B for the next 6 months. The second group will receive the blood components in inverted order
- Masking
- None (open label)
- Enrolment
- 55 participants (actual)
Sponsor and investigators
- Lead sponsor
- Università degli Studi di Ferrara
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Maria Rita Gamberini, Università degli Studi di Ferrara
- Investigators
- Maria Rita Gamberini, MD, Study chair, D.H. Thalassaemia-Haemoglobinopathies (DHTE) - A.O.U. S. Anna of Ferrara
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03992001
- Sponsor's protocol ID
- CrossoverFE2018
- Status last verified
- Nov 2021
- First submitted
- 15 Nov 2018
- Last updated
- 12 Nov 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: There is a plan to make IPD (deidentified) available to other researchers and support already published results
Study at a glance
From the registry record, last updated by the sponsor on 12 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Crossover Study on Beta(ß)-Thalassaemia Transfusion-dependent to Evaluate the Impact on Transfusion Regimen of Two Pre-storage Leukoreduced PRBCs(In-line Filtration + B-C Separation; Whole Blood Filtration + B-C Conservation)
- ClinicalTrials.gov ID
- NCT03992001
- Acronym / sponsor ID
- CrossoverFE2018
- Conditions
- Thalassemia Major
- Keywords
- Packed RBCs, leukodepletion, buffy-coat, transfusion
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 55 participants (actual)
- Lead sponsor
- Università degli Studi di FerraraUniversity, hospital or other
- Study start
- 14 May 2018 (actual)
- Primary completion
- 31 Jul 2019 (actual)
- Study completion
- 31 Jul 2019 (actual)
- First posted
- 19 Jun 2019 (actual)
- 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.
This study compares the effects of Packed Red Blood Cells (PRBCs) prepared in two different ways on the transfusion indices in beta(ß)-Thalassemia transfusion-dependent patients. The two blood components types derive from the whole blood. In one case, the whole blood is leukoreduced with subsequent plasma removal. In the other case, plasma, buffy coat, and red blood cells (RBCs) are first separated and subsequently, the RBCs leukoreduced. Each type of blood components will be subsequently given to one-half of the patients for a 6-month period and to the other half for other 6-month at the randomization phase, for a total of 12 months of crossed-treatment per patient.
Detailed description
At Day Hospital Talassemia ed Emoglobinopatie of Ferrara, two different PRBCs are available. The two types of blood components are obtained from whole blood, pre-storage leukoreduced and suspended in saline-adenine-glucose-mannitol (SAGM). One method of preparation consists of the whole blood leukoreduction with subsequent plasma removal. The other method first separates plasma, buffy coat, and RBCs, and then the RBCs are leukoreduced. The two methods mainly differ in the final haemoglobin (Hb) content: the Hb level is lower (-13%, approximately) in the second method that also shows the advantage to produce platelets from the buffy coat. A PRBCs unit is not as strictly defined as a therapeutic medication dose (pill or vial): individual PRBCs units may substantially differ in their Hb content, much more than the average difference between the two types of preparations. The aim of this study is to document the extent of the average difference between the two types of preparations, and its impacts on the transfusion indices of ß-Thalassaemia transfusion-dependent patients. All patients will receive each blood component for a period of 6 months (crossover design), for a total of 12 months of transfusion treatment per patient.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Patients will be divided into two groups of approximately equal number. The first group will receive the blood component A for a period of 6 months and then the blood component B for the next 6 months. The second group will receive the blood components in inverted order
| Arm | Type | Receives |
|---|---|---|
| Sequence A-B | Experimental | Biological: Blood component A, Biological: Blood component BPatients in this arm will receive blood component A for 6 months and blood component B for the next 6 months |
| Sequence B-A | Experimental | Biological: Blood component A, Biological: Blood component BPatients in this arm will receive blood component B for 6 months and blood component A for the next 6 months |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Patient with beta(ß)-Thalassaemia transfusion-dependent (ß-Thalassemia Major or ß-Thalassemia Intermedia transfusion-dependent, regularly transfused since at least 5 years
Key exclusion criteria (9 listed)
- Patient not exclusively transfused at Day Hospital Thalassaemia and Haemoglobinopathies of Ferrara
- Patient with haemolytic auto-antibodies
- Patient transfused with washed Packet RBCs units
- Severe splenomegaly (>18 cm on echography)
- Elevated blood consumption (>200 mL/kg of pure RBCs in the last year)
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 |
|---|---|---|
| Blood component APRBCs obtained from whole blood after separation of plasma, buffy coat, and RBCs and successive leukoreduction of RBCs | Biological | Sequence A-B, Sequence B-A |
| Blood component BPRBCs obtained by leukoreduction of whole blood, and successive separation of plasma and RBCs | Biological | Sequence A-B, Sequence B-A |
Primary outcome
Transfusion Power Index
Time frame: For each of the two blood components studied, at the end of 6-month period of study
(Average pre-transfusion Hb concentration)/(Unit Index) [for the definition of Unit Index, see the secondary outcomes]
5 secondary outcomes
Average pre-transfusion Hb concentration
Time frame: For each of the two blood components studied, at the end of 6-month period of study
Unit Index
Time frame: For each of the two blood components studied, at the end of 6-month period of study
Average Transfusion Interval
Time frame: For each of the two blood components studied, at the end of 6-month period of study
Number of Transfusion Reactions
Time frame: Study periods (2 periods of 6 months each)
Transfusion Reaction Rate
Time frame: Study periods (2 periods of 6 months each)
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 12 Nov 2021.
- Individual participant dataSharing planned: Yes. There is a plan to make IPD (deidentified) available to other researchers and support already published results
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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.
Maria Rita Gamberini, MD study chair
D.H. Thalassaemia-Haemoglobinopathies (DHTE) - A.O.U. S. Anna of Ferrara
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Blood component A and Blood component B in people with Thalassemia Major, run by Università degli Studi di Ferrara at 1 site in Italy.
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 1 inclusion and 9 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 12 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Transfusion Power Index (time frame: For each of the two blood components studied, at the end of 6-month period of study). 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 12 Nov 2021.