Sickle Cell DiseaseRBC-IMPACTNCT05255445
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients
An observational study of Sickle Cell Disease and Thalassemia, run by Westat at 14 sites in 2 countries.
Trial summary
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and episodically transfused patients. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Sickle Cell Disease, Thalassemia, Pediatric Cancer
- What is being tested?
- Red Blood Cell (RBC) Transfusion
- How many people
- 157 participants
- Where
- 14 sitesin 2 countries
- Study dates
- 16 Mar 2022to 31 Mar 2024
- Phase
- Observational
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: All transfused patients with sickle cell disease (in the U.S or Brazil) and thalassemia on simple chronic transfusion (or partial manual exchange transfusion) from hospitals affiliated with REDS-IV-P Domestic hubs or Brazil hemocenters. (Aim #1) Chronically transfused patients with sickle cell disease or thalassemia, and patients with pediatric oncology diagnoses with hypo-proliferative bone marrow receiving care at hospitals affiliated with REDS-IV-P Domestic hubs or Brazil hemocenters (for SCD only). (Aim #2).
Who may be able to join
- Well-characterized transfusion-dependent form of SCD or thalassemia (including Hemoglobin E-thalassemia and sickle-beta thalassemia) on chronic simple transfusion therapy or partial manual exchange
- On a regular simple RBC transfusion schedule, including partial manual exchange (i.e., 1-3 units scheduled every 2-6 weeks and on a minimum 6-month chronic transfusion trial; for partial manual exchange, the phlebotomy must be completed before the transfusion is started without a back and forth between rounds of phlebotomy and transfusion)
- Seen at any participating domestic hub hospital (i.e., Columbia University Irving Medical Center/Morgan Stanley Children's Hospital of New York, Weill Cornell Medical Center/Komansky Children's Hospital, Boston Children's Hospital, Froedtert \& Medical College of Wisconsin/Children's Wisconsin, University of California San Francisco, Benioff Children's Hospital Oakland) or enrolled in the Brazil REDS-IV-P sickle cell disease cohort and seen at any participating Brazil hemocenter (i.e., Childrens Institute and Adult Clinics at Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo - HCFMUSP, HEMOAM - Amazonas, HEMOMINAS - Minas Gerais, HEMOPE -Pernambuco, and HEMORIO - Rio de Janeiro)
- Either included in Aim #1 (consented patient with SCD or thalassemia) or patient with pediatric oncologic diagnosis under care in a pediatric hematology/oncology service with anemia due to chemotherapy or primary/secondary hypo-proliferative bone marrow requiring a RBC transfusion (including HSCT)
- [In domestic study only] Age ≤21 years old (many pediatric services include care of patients up to age 21, therefore the protocol will not limit by age but instead on whether they are seen in a pediatric service)
- Planned transfusion of RBC from an aliquot or unit from a single donor
- Seen at any participating domestic hub hospital (i.e., Columbia University Irving Medical Center/Morgan Stanley Children's Hospital of New York, Weill Cornell Medical Center/Komansky Children's Hospital, Boston Children's Hospital, Froedtert \& Medical College of Wisconsin/Children's Wisconsin, University of California San Francisco, Benioff Children's Hospital Oakland) or at any REDS-IV-P participating Brazil hemocenter (i.e., Childrens Institute and Adult Clinics at Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo - HCFMUSP, HEMOAM - Amazonas, HEMOMINAS - Minas Gerais, HEMOPE -Pernambuco, and HEMORIO - Rio de Janeiro).
Who cannot join
- Institutionalization or imprisonment
- Foster care
- Weight <11 kg
- Institutionalization or imprisonment
- Foster care
- Current active auto-immune hemolytic anemia based on positive direct antiglobulin test (DAT) with laboratory evidence of hemolysis and increased transfusion requirement
- [In domestic study only] Microangiopathic hemolytic anemia
- Weight <18 kg
Trial locations and contacts
14 sites in 2 countries.
14 sites in 2 countries
United States9 sites
Boston Children's Hospital
Boston, Massachusetts, 02115
Children's Wisconsin
Milwaukee, Wisconsin, 53226
Froedtert Hospital
Milwaukee, Wisconsin, 53226
Versiti Wisconsin, Inc.
Milwaukee, Wisconsin, 53233
Weill Cornell Medical Collection (WCMC)/New York Presbyterian Hospital (NYPH)
New York, New York, 10021
Columbia University Irving Medical Center/New York Presbyterian Hospital (NYPH)
New York, New York, 10032
New York Blood Center (NYBC)
New York, New York, 10065
UCSF Benioff Children's Hospital
Oakland, California, 94609
Vitalant Research Institute
San Francisco, California, 94118
Brazil5 sites
HEMOMINAS - Minas Gerais
Belo Horizonte, Minas Gerais, 30622-020
HEMOAM - Amazonas
Manaus, Amazonas, 69050-001
HEMOPE - Pernambuco
Recife, Pernambuco, 52011-000
HEMORIO - Rio De Janeiro
Rio de Janeiro, 20211-030
Childrens Institute and Adult Clinics at Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo, 05403-000
About this study
Official title: Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients (RBC-IMPACT)
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and episodically transfused patients. The objective of the study is to determine how specific genetic and non-genetic factors in donors and recipients may impact RBC survival after transfusion - in short, what factors on both the donor and recipient side may improve the efficacy of the transfusion.
Read the detailed description
Sickle cell disease (SCD) and thalassemia are genetic disorders inducing anemia of differing pathophysiology. A primary therapy for preventing certain SCD complications (e.g., stroke) and for thalassemia major is regular red blood cell (RBC) transfusion, coupled with iron chelation to prevent the complications of transfusion-induced iron overload. For patients with pediatric hematology-oncology diagnoses with chemotherapy-induced aplasia, RBC transfusion is also common, but the degree of transfusion-induced iron overload and its implications for these patients is incompletely understood. Because iron-related tissue toxicity is a major cause of morbidity and mortality in regularly transfused patients, developing strategies to minimize iron loading and iron toxicity is a key objective of this proposal (study Aim #2), stemming from the objective to optimize RBC unit characteristics that patients with SCD and thalassemia receive beyond RBC phenotype matching for Rh C, E and K antigens (study Aim #1). The study will enroll patients with SCD, thalassemia or pediatric oncologic diagnoses receiving eligible transfusion at 6 hospital sites in the United States, as well as patients with SCD at 5 hemocenters in Brazil.
- Conditions
- Sickle Cell Disease, Thalassemia, Pediatric Cancer
- Being tested
- Red Blood Cell (RBC) Transfusion
- Keywords
- Transfusion, Red Blood Cell, Sickle Cell Disease, Thalassemia, Pediatric, Oncology, RBC survival, Genetic, Non-genetic, Observational, Blood donor
- Medical subject headings
- Anemia, Sickle Cell, Thalassemia, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Red Blood Cell (RBC) TransfusionBiological
Simple RBC transfusion or partial manual exchange
| Study group | Role | What they receive |
|---|---|---|
| Sickle cell disease (SCD) | Patients with SCD who are chronically transfused (in the U.S. and Brazil) Receives: Biological: Red Blood Cell (RBC) Transfusion | |
| Thalassemia | Patients with thalassemia who are chronically transfused in the U.S. Receives: Biological: Red Blood Cell (RBC) Transfusion | |
| Pediatric Hematology-Oncology | Patients in U.S. with pediatric oncologic diagnoses with hypo-proliferative bone marrow requiring single unit red blood cell transfusion Receives: Biological: Red Blood Cell (RBC) Transfusion | |
| Blood Donors | Allogenic blood donors in the US (estimated: 10,200) and allogenic blood donors in Brazil (estimated: 2,100) with extended donation genotyping using an investigational hematology array. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Hemoglobin A or Hemoglobin Level per day (RBC Survival)
Change in hemoglobin A or hemoglobin level per day in between subsequent transfusion episodes, for sickle cell disease and thalassemia cohorts, respectively
Measured: Baseline (immediately pre-) to post-transfusion over 2 years
Change in Serum Iron Level
For all groups participating, change in serum iron measured from immediately prior to 2 hours post-transfusion
Measured: Baseline (immediately before) and 2-hours after transfusion
Secondary outcomes (5)
Hemoglobin Increment
Hemoglobin increment [defined as Hb/HbA(post-transfusion)visit(i) - Hb/HbA(pre-transfusion)visit(i)] is associated with "RBC survival"
Measured: Baseline (immediately pre-) to post-transfusion, over 2 years
Hemolysis Parameter Increment
Includes serum iron, indirect bilirubin, or plasma free hemoglobin
Measured: Baseline (immediately pre-) to post-transfusion or 2-hours post-transfusion, over 2 years
Hepcidin Level
Hepcidin level at time of transfusion is a predictor of change in iron parameters (i.e., transferrin saturation, serum iron) following transfusion
Measured: Baseline (immediately before) to 2 hours after transfusion
Non-Transferrin-Bound Iron (NTBI) Level
NTBI levels in patients with pediatric oncologic diagnoses with aplasia are elevated at baseline and increase following transfusion
Measured: Baseline (immediately before) to 2 hours after transfusion
Number of Clinical Complications
Increased NTBI, serum iron, or transferrin saturation following transfusion is associated with increased risk of clinical adverse effects (i.e., new infections, SCD complications)
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2022
- Study start16 Mar 2022
- Primary completion31 Mar 2024
- Study completion31 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 157 participants (actual)
Sponsor and investigators
- Lead sponsor
- Westat
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Responsible party
- Sponsor
- Investigators
- Eldad A Hod, MD, Principal investigator, Columbia UniversityBrian Custer, PhD, MPH, Principal investigator, Vitalant Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05255445
- Sponsor's protocol ID
- 75N92019D00032
- Other IDs
- 75N92019D00033, 75N92019D00034, 75N92019D00035, 75N92019D00036, 75N92019D00037, 75N92019D00038
- Acronym
- RBC-IMPACT
- Status last verified
- May 2024
- First submitted
- 10 Feb 2022
- Last updated
- 17 May 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: De-identified public use datasets will be created and delivered to NHLBI at the end of the study (and end of the REDS-IV-P program) and will be made available indefinitely for future analysis
Publications
Karafin MS, Kelly S, Chapman KM, Kreuziger LB, Manis JP, Dinardo C, Josephson CD, Stone M, Roubinian NH, Branchford B, Sachais BS, Hailu B, Sabino EC, Hod EA, Custer B; National Heart, Lung, and Blood Institute (NHLBI) Recipient Epidemiology and Donor Evaluation Study-IV-Pediatric (REDS-IV-P). The Red Blood Cell-Improving Transfusions for Chronically Transfused Recipients (RBC-IMPACT) study: protocol description of an international multi-site observational clinical study. Blood Transfus. 2025 Sep-Oct;23(5):418-432. doi: 10.2450/BloodTransfus.1026. Epub 2025 May 9.
PubMed 40423585
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients (RBC-IMPACT)
- ClinicalTrials.gov ID
- NCT05255445
- Acronym / sponsor ID
- RBC-IMPACT / 75N92019D00032
- Conditions
- Sickle Cell Disease, Thalassemia, Pediatric Cancer
- Keywords
- Transfusion, Red Blood Cell, Sickle Cell Disease, Thalassemia, Pediatric, Oncology, RBC survival, Genetic
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 157 participants (actual)
- Lead sponsor
- WestatUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Study start
- 16 Mar 2022 (actual)
- Primary completion
- 31 Mar 2024 (actual)
- Study completion
- 31 Mar 2024 (actual)
- First posted
- 24 Feb 2022 (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.
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and episodically transfused patients. The objective of the study is to determine how specific genetic and non-genetic factors in donors and recipients may impact RBC survival after transfusion - in short, what factors on both the donor and recipient side may improve the efficacy of the transfusion.
Detailed description
Sickle cell disease (SCD) and thalassemia are genetic disorders inducing anemia of differing pathophysiology. A primary therapy for preventing certain SCD complications (e.g., stroke) and for thalassemia major is regular red blood cell (RBC) transfusion, coupled with iron chelation to prevent the complications of transfusion-induced iron overload. For patients with pediatric hematology-oncology diagnoses with chemotherapy-induced aplasia, RBC transfusion is also common, but the degree of transfusion-induced iron overload and its implications for these patients is incompletely understood. Because iron-related tissue toxicity is a major cause of morbidity and mortality in regularly transfused patients, developing strategies to minimize iron loading and iron toxicity is a key objective of this proposal (study Aim #2), stemming from the objective to optimize RBC unit characteristics that patients with SCD and thalassemia receive beyond RBC phenotype matching for Rh C, E and K antigens (study Aim #1). The study will enroll patients with SCD, thalassemia or pediatric oncologic diagnoses receiving eligible transfusion at 6 hospital sites in the United States, as well as patients with SCD at 5 hemocenters in Brazil.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Sickle cell disease (SCD) | – | Biological: Red Blood Cell (RBC) TransfusionPatients with SCD who are chronically transfused (in the U.S. and Brazil) |
| Thalassemia | – | Biological: Red Blood Cell (RBC) TransfusionPatients with thalassemia who are chronically transfused in the U.S. |
| Pediatric Hematology-Oncology | – | Biological: Red Blood Cell (RBC) TransfusionPatients in U.S. with pediatric oncologic diagnoses with hypo-proliferative bone marrow requiring single unit red blood cell transfusion |
| Blood Donors | – | –Allogenic blood donors in the US (estimated: 10,200) and allogenic blood donors in Brazil (estimated: 2,100) with extended donation genotyping using an investigational hematology array. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All transfused patients with sickle cell disease (in the U.S or Brazil) and thalassemia on simple chronic transfusion (or partial manual exchange transfusion) from hospitals affiliated with REDS-IV-P Domestic hubs or Brazil hemocenters. (Aim #1) Chronically transfused patients with sickle cell disease or thalassemia, and patients with pediatric oncology diagnoses with hypo-proliferative bone marrow receiving care at hospitals affiliated with REDS-IV-P Domestic hubs or Brazil hemocenters (for SCD only). (Aim #2).
Key inclusion criteria (7 listed)
- Well-characterized transfusion-dependent form of SCD or thalassemia (including Hemoglobin E-thalassemia and sickle-beta thalassemia) on chronic simple transfusion therapy or partial manual exchange
- On a regular simple RBC transfusion schedule, including partial manual exchange (i.e., 1-3 units scheduled every 2-6 weeks and on a minimum 6-month chronic transfusion trial; for partial manual exchange, the phlebotomy must be completed before the transfusion is started without a back and forth between rounds of phlebotomy and transfusion)
- Seen at any participating domestic hub hospital (i.e., Columbia University Irving Medical Center/Morgan Stanley Children's Hospital of New York, Weill Cornell Medical Center/Komansky Children's Hospital, Boston Children's Hospital, Froedtert \& Medical College of Wisconsin/Children's Wisconsin, University of California San Francisco, Benioff Children's Hospital Oakland) or enrolled in the Brazil REDS-IV-P sickle cell disease cohort and seen at any participating Brazil hemocenter (i.e., Childrens Institute and Adult Clinics at Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo - HCFMUSP, HEMOAM - Amazonas, HEMOMINAS - Minas Gerais, HEMOPE -Pernambuco, and HEMORIO - Rio de Janeiro)
- Either included in Aim #1 (consented patient with SCD or thalassemia) or patient with pediatric oncologic diagnosis under care in a pediatric hematology/oncology service with anemia due to chemotherapy or primary/secondary hypo-proliferative bone marrow requiring a RBC transfusion (including HSCT)
- [In domestic study only] Age ≤21 years old (many pediatric services include care of patients up to age 21, therefore the protocol will not limit by age but instead on whether they are seen in a pediatric service)
Key exclusion criteria (8 listed)
- Institutionalization or imprisonment
- Foster care
- Weight <11 kg
- Institutionalization or imprisonment
- Foster care
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 |
|---|---|---|
| Red Blood Cell (RBC) TransfusionSimple RBC transfusion or partial manual exchange | Biological | Pediatric Hematology-Oncology, Sickle cell disease (SCD), Thalassemia |
Primary outcome
Change in Hemoglobin A or Hemoglobin Level per day (RBC Survival)
Time frame: Baseline (immediately pre-) to post-transfusion over 2 years
Change in hemoglobin A or hemoglobin level per day in between subsequent transfusion episodes, for sickle cell disease and thalassemia cohorts, respectively
Change in Serum Iron Level
Time frame: Baseline (immediately before) and 2-hours after transfusion
For all groups participating, change in serum iron measured from immediately prior to 2 hours post-transfusion
5 secondary outcomes
Hemoglobin Increment
Time frame: Baseline (immediately pre-) to post-transfusion, over 2 years
Hemolysis Parameter Increment
Time frame: Baseline (immediately pre-) to post-transfusion or 2-hours post-transfusion, over 2 years
Hepcidin Level
Time frame: Baseline (immediately before) to 2 hours after transfusion
Non-Transferrin-Bound Iron (NTBI) Level
Time frame: Baseline (immediately before) to 2 hours after transfusion
Number of Clinical Complications
Time frame: 2 years
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 17 May 2024.
- Derived from this study: PubMed 40423585Karafin MS, Kelly S, Chapman KM, Kreuziger LB, Manis JP, Dinardo C, Josephson CD, Stone M, Roubinian NH, Branchford B, Sachais BS, Hailu B, Sabino EC, Hod EA, Custer B; National Heart, Lung, and Blood Institute (NHLBI) Recipient Epidemiology and Donor Evaluation Study-IV-Pediatric (REDS-IV-P). The Red Blood Cell-Improving Transfusions for Chronically Transfused Recipients (RBC-IMPACT) study: protocol description of an international multi-site observational clinical study. Blood Transfus. 2025 Sep-Oct;23(5):418-432. doi: 10.2450/BloodTransfus.1026. Epub 2025 May 9.
- REDS-IV-P public websiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. De-identified public use datasets will be created and delivered to NHLBI at the end of the study (and end of the REDS-IV-P program) and will be made available indefinitely for future analysis
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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.
Eldad A Hod, MD principal investigator
Columbia University
Brian Custer, PhD, MPH principal investigator
Vitalant Research Institute
14 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease and Thalassemia, run by Westat at 14 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 7 inclusion and 8 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 17 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Brazil. 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.
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?
Change in Hemoglobin A or Hemoglobin Level per day (RBC Survival) (time frame: Baseline (immediately pre-) to post-transfusion over 2 years). 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?
On the ClinicalTrials.gov record for NCT05255445, which also lists 1 linked publication.
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 17 May 2024.