Sickle Cell DiseaseNCT03903289
The Implementation of the Automated Erythrocytapheresis in Egyptian Sickle Cell Disease Center
A study testing Automated red cell exchange, Manual red cell exchange and Simple red cell transfusion in people with Sickle Cell Disease, run by Ain Shams University at 1 site in Egypt.
Trial summary
Improvements of health infrastructure, preventive care and clinical treatment have reduced the morbidity and mortality of sickle cell disease (SCD). However, SCD is still an increasing national health problem, with increase longevity the chronic effect of sustained hemolysis and episodic vaso-occlusive events and the… Read more about this study
- Who is it for?
- Ages 2 to 30 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Automated red cell exchange, Manual red cell exchange and Simple red cell transfusion
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteEgypt
- Study dates
- 16 Aug 2017to Jan 2026
- Phase
- Interventional
- 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 2 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patient with sickle cell disease (HbS with other variant hemoglobin)
- Age from 2-30y old
Who cannot join
- Patients with other hemoglobinopathy Patients younger than two years
Trial locations and contacts
1 site in 1 country.
Egypt1 site, 1 recruiting
- Recruiting
Faculty of Medicine, Ain Shams University
Cairo, Non-US, 11556
Fatma SE Ebeid, MD, 01095569596, dr.fatma_ebeid@yahoo.com
Fatma SE Ebeid, MD
About this study
Official title: Assessment of Implementation of the Automated Erythrocytapheresis in the Outcome of Egyptian Sickle Cell Disease Patients: Single Center Experience
Improvements of health infrastructure, preventive care and clinical treatment have reduced the morbidity and mortality of sickle cell disease (SCD). However, SCD is still an increasing national health problem, with increase longevity the chronic effect of sustained hemolysis and episodic vaso-occlusive events and the recurrent episodes of ischemic reperfusion injury drive the development of progressive end organ complications and cardiovascular, pulmonary, neurological and renal systems are most commonly affected. Today there is hope for a cure using hematopoietic stem cell transplantation (HSCT). However, at present; the procedure is infrequently performed and very expensive. In this research we will assess the effect of implementation of the automated erythrocytapheresis in the outcome of sickle cell disease in single Egyptian center.
Read the detailed description
There is a wide scoop for treatment of SCD ranging from medical treatment with hydroxyurea, simple transfusion with chelation therapy, exchange transfusion either manual or automated and stem cell transplantation which need highly equipped institutes.
The goals of treatment of SCD are symptom control with early detection and management of disease complications. Although several pharmacological agents have been studied for the treatment of SCD, the only drug currently approved by the US Food and Drug Administration (FDA) for the treatment of SCD is hydroxyurea. Allogeneic bone marrow transplantation (BMT) can cure SCD, but it is difficult to decide which patients should be offered BMT. Many risks are associated with BMT, and the risk-to-benefit ratio must be assessed carefully. The lack of availability of a matched donor may limit the utility of BMT. Transfusions are not needed for the usual anemia or episodes of pain associated with SCD. Urgent replacement of blood is often required for sudden severe anemia and regular blood transfusions are used for primary and secondary stroke prevention in children with SCD. With continued transfusion, iron overload inevitably develops and can result in heart and liver failure, and multiple other complications necessitate the use of chelation therapy.
Erythrocytapheresis is an automated red cell exchange procedure that removes blood that contains HbS from the patient while simultaneously replacing that same volume with packed red cells free of HbS. Transfusion usually consists of sickle-negative, leuco-reduced, and phenotypically matched blood for red cell antigens. Erythrocytapheresis thus has the advantage of controlling iron accumulation in patients with SCD who undergo long-term transfusion, as well as the ability to allow rapid reduction of HbS concentrations to less than 30% without significantly increasing total hemoglobin concentration post transfusion. This precision is achieved because, before the start of the transfusion, the computer in the pheresis machine calculates the expected amount of packed RBCs required to obtain a specific post-transfusion hemoglobin level, using various physiologic parameters (eg, height, weight, Hb level). Further, erythrocytapheresis requires less time than simple transfusion of similar blood volumes. Although erythrocytapheresis is more expensive than simple transfusion, the additional costs associated with simple transfusions (ie, those of chelation and organ damage due to iron overload) make erythrocytapheresis more cost-effective than simple transfusion programs. Central venous access devices can safely be used for long-term erythrocytapheresis in patients with SCD with a low rate of complications.
- Conditions
- Sickle Cell Disease
- Being tested
- Automated red cell exchange, Manual red cell exchange, Simple red cell transfusion
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Automated red cell exchangeProcedure or surgery
Also called Erythrocytapheresis
Erythrocytapheresis is an automated red cell exchange procedure that removes blood that contains HbS from the patient while simultaneously replacing that same volume with packed red cells free of HbS.
Manual red cell exchangeProcedure or surgery
Active Comparator: Manual red cell exchange
Simple red cell transfusionProcedure or surgery
Active Comparator: Simple red cell transfusion
| Study group | Role | What they receive |
|---|---|---|
| Automated red cell exchange | Experimental | Automated red cell exchange Receives: Procedure: Automated red cell exchange |
| Manual red cell exchange | Active comparator | Manual red cell exchange Receives: Procedure: Manual red cell exchange |
| Simple red cell transfusion | Sham comparator | Simple red cell transfusion Receives: Procedure: Simple red cell transfusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
The change of health related quality of life (HRQoL) is being assessed
The medical Outcome Study (MOS) short form (SF-36) is a 36- item tool for measuring health status and outcome from the patients' perceptions
Measured: assessed at enrollment and after one year of intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Apr 2019
- Study start16 Aug 2017
- Primary completionDec 2025 (estimated)
- Study completionJan 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Ain Shams UniversityThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ain Shams University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fatma Soliman Elsayed Ebeid, MD, Ain Shams University
- Investigators
- Fatma Solima SE Ebeid, MD, Principal investigator, Faculty of Medicine, Ain Shams university
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03903289
- Sponsor's protocol ID
- FMASU M D 94/ 2018
- Status last verified
- Jan 2024
- First submitted
- 14 Mar 2019
- Last updated
- 11 Jan 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Implementation of the Automated Erythrocytapheresis in the Outcome of Egyptian Sickle Cell Disease Patients: Single Center Experience
- ClinicalTrials.gov ID
- NCT03903289
- Acronym / sponsor ID
- FMASU M D 94/ 2018
- Conditions
- Sickle Cell Disease
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Ain Shams UniversityUniversity, hospital or other
- Study start
- 16 Aug 2017 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Jan 2026 (estimated)
- First posted
- 4 Apr 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.
Improvements of health infrastructure, preventive care and clinical treatment have reduced the morbidity and mortality of sickle cell disease (SCD). However, SCD is still an increasing national health problem, with increase longevity the chronic effect of sustained hemolysis and episodic vaso-occlusive events and the recurrent episodes of ischemic reperfusion injury drive the development of progressive end organ complications and cardiovascular, pulmonary, neurological and renal systems are most commonly affected. Today there is hope for a cure using hematopoietic stem cell transplantation (HSCT). However, at present; the procedure is infrequently performed and very expensive. In this research we will assess the effect of implementation of the automated erythrocytapheresis in the outcome of sickle cell disease in single Egyptian center.
Detailed description
There is a wide scoop for treatment of SCD ranging from medical treatment with hydroxyurea, simple transfusion with chelation therapy, exchange transfusion either manual or automated and stem cell transplantation which need highly equipped institutes.
The goals of treatment of SCD are symptom control with early detection and management of disease complications. Although several pharmacological agents have been studied for the treatment of SCD, the only drug currently approved by the US Food and Drug Administration (FDA) for the treatment of SCD is hydroxyurea. Allogeneic bone marrow transplantation (BMT) can cure SCD, but it is difficult to decide which patients should be offered BMT. Many risks are associated with BMT, and the risk-to-benefit ratio must be assessed carefully. The lack of availability of a matched donor may limit the utility of BMT. Transfusions are not needed for the usual anemia or episodes of pain associated with SCD. Urgent replacement of blood is often required for sudden severe anemia and regular blood transfusions are used for primary and secondary stroke prevention in children with SCD. With continued transfusion, iron overload inevitably develops and can result in heart and liver failure, and multiple other complications necessitate the use of chelation therapy.
Erythrocytapheresis is an automated red cell exchange procedure that removes blood that contains HbS from the patient while simultaneously replacing that same volume with packed red cells free of HbS. Transfusion usually consists of sickle-negative, leuco-reduced, and phenotypically matched blood for red cell antigens. Erythrocytapheresis thus has the advantage of controlling iron accumulation in patients with SCD who undergo long-term transfusion, as well as the ability to allow rapid reduction of HbS concentrations to less than 30% without significantly increasing total hemoglobin concentration post transfusion. This precision is achieved because, before the start of the transfusion, the computer in the pheresis machine calculates the expected amount of packed RBCs required to obtain a specific post-transfusion hemoglobin level, using various physiologic parameters (eg, height, weight, Hb level). Further, erythrocytapheresis requires less time than simple transfusion of similar blood volumes. Although erythrocytapheresis is more expensive than simple transfusion, the additional costs associated with simple transfusions (ie, those of chelation and organ damage due to iron overload) make erythrocytapheresis more cost-effective than simple transfusion programs. Central venous access devices can safely be used for long-term erythrocytapheresis in patients with SCD with a low rate of complications.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Automated red cell exchange | Experimental | Procedure: Automated red cell exchangeAutomated red cell exchange |
| Manual red cell exchange | Active comparator | Procedure: Manual red cell exchangeManual red cell exchange |
| Simple red cell transfusion | Sham comparator | Procedure: Simple red cell transfusionSimple red cell transfusion |
Who may qualify
- Ages
- 2 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Patient with sickle cell disease (HbS with other variant hemoglobin)
- Age from 2-30y old
Key exclusion criteria (1 listed)
- Patients with other hemoglobinopathy Patients younger than two years
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 |
|---|---|---|
| Automated red cell exchangeAlso: ErythrocytapheresisErythrocytapheresis is an automated red cell exchange procedure that removes blood that contains HbS from the patient while simultaneously replacing that same volume with packed red cells free of HbS. | Procedure or surgery | Automated red cell exchange |
| Manual red cell exchangeActive Comparator: Manual red cell exchange | Procedure or surgery | Manual red cell exchange |
| Simple red cell transfusionActive Comparator: Simple red cell transfusion | Procedure or surgery | Simple red cell transfusion |
Primary outcome
The change of health related quality of life (HRQoL) is being assessed
Time frame: assessed at enrollment and after one year of intervention
The medical Outcome Study (MOS) short form (SF-36) is a 36- item tool for measuring health status and outcome from the patients' perceptions
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jan 2024.
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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.
Fatma Solima SE Ebeid, MD principal investigator
Faculty of Medicine, Ain Shams university
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Automated red cell exchange, Manual red cell exchange and Simple red cell transfusion in people with Sickle Cell Disease, run by Ain Shams University at 1 site in Egypt.
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 2 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 11 Jan 2024. 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 Egypt. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fatma SE Ebeid, MD (01095569596, dr.fatma_ebeid@yahoo.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The change of health related quality of life (HRQoL) is being assessed (time frame: assessed at enrollment and after one year of intervention). 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 11 Jan 2024.