ClinicalTrials.gg
CompletedNCT05255445RBC-IMPACTUpdated May 17, 2024

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

An observational study in Sickle Cell Disease, Thalassemia and Pediatric Cancer, sponsored by Westat. Completed at 14 sites in 2 countries. Per ClinicalTrials.gov, last updated 2024-05-17.

Sponsored by Westat · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
157
Sex
All
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Study 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. 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.

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Conditions studied

  • Sickle Cell Disease
  • Thalassemia
  • Pediatric Cancer

Keywords

  • Transfusion
  • Red Blood Cell
  • Sickle Cell Disease
  • Thalassemia
  • Pediatric
  • Oncology
  • RBC survival
  • Genetic
  • Non-genetic
  • Observational
  • Blood donor
03

In context

Anemia, Sickle Cell

1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.

This study's enrollment of 157 is above the median of 100 across 296 observational studies indexed under Anemia, Sickle Cell.

Browse Anemia, Sickle Cell studies →

Lead sponsor

Westat is the lead sponsor of 23 studies on the registry; 6 are open to participants now.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

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).

Eligibility criteria

Inclusion Criteria (Aim #1):

  • Well-characterized transfusion-dependent form of SCD or thalassemia (including 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)

Exclusion Criteria (Aim #1):

  • Institutionalization or imprisonment
  • Foster care
  • Weight \<11 kg

Inclusion criteria (Aim #2):

  • 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 )
  • [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).

Exclusion criteria (Aim #2):

  • 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
Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Allogeneic and autologous transplant

Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.

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Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
157 participants (actual)
Patient registry
No
Biospecimen retention
Samples without dna

Groups and cohorts

  • Sickle cell disease (SCD)

    Patients with SCD who are chronically transfused (in the U.S. and Brazil)

    Biological: Red Blood Cell (RBC) Transfusion

  • Thalassemia

    Patients with thalassemia who are chronically transfused in the U.S.

    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

    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.

Interventions

  • BiologicalRed Blood Cell (RBC) Transfusion

    Simple RBC transfusion or partial manual exchange

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: Baseline (immediately pre-) to post-transfusion over 2 years

  2. Change in Serum Iron Level

    For all groups participating, change in serum iron measured from immediately prior to 2 hours post-transfusion

    Time frame: Baseline (immediately before) and 2-hours after transfusion

Secondary outcomes

  1. Hemoglobin Increment

    Hemoglobin increment \[defined as Hb/HbA(post-transfusion)visit(i) - Hb/HbA(pre-transfusion)visit(i)\] is associated with "RBC survival"

    Time frame: Baseline (immediately pre-) to post-transfusion, over 2 years

  2. Hemolysis Parameter Increment

    Includes serum iron, indirect bilirubin, or plasma free hemoglobin

    Time frame: Baseline (immediately pre-) to post-transfusion or 2-hours post-transfusion, over 2 years

  3. Hepcidin Level

    Hepcidin level at time of transfusion is a predictor of change in iron parameters (i.e., transferrin saturation, serum iron) following transfusion

    Time frame: Baseline (immediately before) to 2 hours after transfusion

  4. Non-Transferrin-Bound Iron (NTBI) Level

    NTBI levels in patients with pediatric oncologic diagnoses with aplasia are elevated at baseline and increase following transfusion

    Time frame: Baseline (immediately before) to 2 hours after transfusion

  5. 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)

    Time frame: 2 years

Other outcomes

  1. Rate of Alloimmunization

    Rate of new alloantibody formation

    Time frame: 2 years

  2. 4-hydroxynonenal [4-HNE]

    Recipient oxidative stress pre-transfusion is associated with "RBC survival"

    Time frame: 2 years

  3. Type I interferon (i.e., MxA protein assay) and other cytokines (i.e., IL-6, MCP-1, IFNgamma)

    Recipient inflammation pre-transfusion is associated with "RBC survival"

    Time frame: 2 years

  4. Number of Transfusion Reactions

    Transfusion reactions are associated with "RBC survival"

    Time frame: 2 years

07

Study locations

14 sites
  • UCSF Benioff Children's Hospital
    Oakland, California 94609, United States
  • Vitalant Research Institute
    San Francisco, California 94118, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02115, United States
  • Weill Cornell Medical Collection (WCMC)/New York Presbyterian Hospital (NYPH)
    New York, New York 10021, United States
  • Columbia University Irving Medical Center/New York Presbyterian Hospital (NYPH)
    New York, New York 10032, United States
  • New York Blood Center (NYBC)
    New York, New York 10065, United States
  • Children's Wisconsin
    Milwaukee, Wisconsin 53226, United States
  • Froedtert Hospital
    Milwaukee, Wisconsin 53226, United States
  • Versiti Wisconsin, Inc.
    Milwaukee, Wisconsin 53233, United States
  • HEMOAM - Amazonas
    Manaus, Amazonas 69050-001, Brazil
  • HEMOMINAS - Minas Gerais
    Belo Horizonte, Minas Gerais 30622-020, Brazil
  • HEMOPE - Pernambuco
    Recife, Pernambuco 52011-000, Brazil
  • HEMORIO - Rio De Janeiro
    Rio De Janeiro, 20211-030, Brazil
  • Childrens Institute and Adult Clinics at Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
    São Paulo, 05403-000, Brazil
08

References and documents

Individual participant data

Plan to share: 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

Supporting information: Study protocol, Clinical study report

Time frame: At the end of the REDS-IV-P program, estimated in March 2026, the public use data sets will be available and will be available indefinitely for future analytic use.

Access criteria: Public use datasets will be posted to NIH/NHLBI data repository systems.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Mar 2022 to primary completion in Mar 2024, 24 months.

Of 130 non-industry observational studies under Anemia, Sickle Cell, half reached primary completion within 48 months of starting (the median), counting those still running; a quarter within 23 and three quarters within 109. This study's 24 months is below that.

Counting every sponsor, half of 139 studies reached it within 49 months; a quarter within 24 and three quarters within 109.

How these figures are worked out, and which studies are left out of them.

Registered
Feb 10, 2022
First posted
Feb 24, 2022
StartActual
Mar 16, 2022
Primary completionActual
Mar 31, 2024
CompletionActual
Mar 31, 2024
Last update
May 17, 2024
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on May 17, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT05255445
Lead sponsor
Westat
Responsible party
Sponsor
First posted
Feb 24, 2022
Start date
Mar 16, 2022
Primary completion
Mar 31, 2024
Completion
Mar 31, 2024
Last update
May 17, 2024

Study contacts

Eldad A Hod, MD
principal investigator · Columbia University
Brian Custer, PhD, MPH
principal investigator · Vitalant Research Institute

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in May 2024. You cannot join it, but the record below documents what was studied.

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