AnemiaNCT03086252
Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study
A study testing Red Blood Cell Transfusions (RBCT) in people with Anemia and Aplastic Anemia, run by Emory University at 1 site in United States.
Trial summary
This pilot study evaluates safety of administration of red blood cell transfusions requested by patients based on their symptoms instead of levels of hemoglobin for the treatment of chronic anemia in patients with blood disorders. Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Anemia, Aplastic Anemia, Bone Marrow Failure
- What is being tested?
- Red Blood Cell Transfusions (RBCT)
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Jul 2017to 31 May 2018
- 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 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosed with hematological malignancy or marrow failure syndrome such as but not limited to: aplastic anemia, myelodysplastic syndrome or leukemia
- Chronic transfusion-dependent anemia with exposure to at least 5 RBCT
- Interested in reducing transfusion exposure
- Willing to sign informed consent
Who cannot join
- Recent acute bleeding requiring intervention in less than 24 hours
- Hemoglobin levels < 6 g/dL
- Acute leukemia receiving induction chemotherapy
- Any patient with known ischemic heart disease, history of congestive heart failure, history of stroke, or cardiac arrhythmia for which the patient requires medication or a medical device
- Oxygen dependent
- Oxygen saturation below 92% on room air
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- Receiving erythropoietin stimulating agent
- Thalassemia major or sickle cell disease requiring blood transfusion
- Undergoing major surgery
- Hemolytic anemia
- Coagulopathies including disseminated intravascular coagulation (DIC), receiving anticoagulant or antiplatelet agents
- Diagnosed with chronic obstructive pulmonary disease (COPD) oxygen dependent
- Pregnancy
- Participation in a therapeutic clinical trial
Trial locations and contacts
1 site in 1 country.
United States1 site
Emory University/Winship Cancer Institute
Atlanta, Georgia, 30322
About this study
This pilot study evaluates safety of administration of red blood cell transfusions requested by patients based on their symptoms instead of levels of hemoglobin for the treatment of chronic anemia in patients with blood disorders.
Read the detailed description
PRIMARY OBJECTIVE:
- To assess feasibility and safety of red blood cell transfusions at the time subjects requested based on symptomatic anemia.
SECONDARY OBJECTIVES:
- To define a patient-driven red blood cell transfusion (RBCT) threshold and to compare the median hemoglobin (hgb) at the time subjects request RBCT to the standard threshold of hgb < 8 g/dL.
II. To compare the number of red blood cell (RBC) units transfused and the frequency of transfusions at the time subjects request RBCT during the study period to the number of RBC units received and frequency using the standard threshold hemoglobin < 8 g/dL during a comparable period prior to enrollment.
III. To compare the quality of life (QoL) scores when subjects request RBCT to the scores obtained at the time hgb is 8 +/- 0.5 g/dL.
OUTLINE:
Patients undergo RBCT based on their perception and/or the presence of anemia symptoms for up to 6 months.
After completion of study treatment, patients are followed up for 30 days.
- Conditions
- Anemia, Aplastic Anemia, Bone Marrow Failure, Hematopoietic and Lymphoid Cell Neoplasm, Leukemia, Myelodysplastic Syndrome
- Being tested
- Red Blood Cell Transfusions (RBCT)
- Medical subject headings
- Anemia, Anemia, Aplastic, Bone Marrow Failure Disorders, Hematologic Neoplasms, Leukemia, Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Red Blood Cell Transfusions (RBCT)Procedure or surgery
Also called Blood Component Transfusion, Blood Transfusion, Transfusion, Transfusion of Blood Products
Undergo patient-driven RBCT
| Study group | Role | What they receive |
|---|---|---|
| Supportive care (patient-driven RBCT) | Experimental | Patients undergo red blood cell transfusions (RBCT) based on their perception and/or the presence of anemia symptoms for up to 6 months. Receives: Procedure: Red Blood Cell Transfusions (RBCT) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of laboratory and clinical adverse events (AEs) assessed by the Common Terminology Criteria for Adverse Events version 4.03.
Frequency, duration, and severity of AEs and severe adverse events will be determined. The proportion of subjects with serious side effects will be calculated with 95% confidence interval. Descriptive statistics will be used to characterize subjects enrolled on the trial: age, sex, race and diagnosis.
Measured: Up to 6 months of study participation
Secondary outcomes (5)
Frequency of RBC units transfused
The frequency of RBC units transfused will be compared between study period and prior to enrollment for the same patient (e.g. each patient will serve his/her own control) by Wilcoxon's sign rank test.
Measured: Up to 6 months of study participation
Hemoglobin concentration at the time subject requests RBCT
Hemoglobin concentration (Hgb) will be measured in g/dL.
Measured: Up to 6 months of study participation
Number of RBC units transfused
The number of RBC units transfused will be compared between study period and prior to enrollment for the same patient (e.g. each patient will serve his/her own control) by Wilcoxon's sign rank test.
Measured: Up to 6 months of study participation
QoL scores from the Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaire at the time hemoglobin is 8 +/- 0.5 g/dL
Will be compared to QoL scores at the time subjects request RBCT by paired t-test and/or Wilcoxon sign rank test.
Measured: Up to 6 months of study participation
QoL scores from the Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaire at the time subjects request RBCT
Will be compared to QoL scores at the time hgb is 8 +/- 0.5 g/dL by paired t-test and/or Wilcoxon sign rank test.
Measured: Up to 6 months of study participation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2017
- Study start24 Jul 2017
- Primary completion31 May 2018
- Study completion31 May 2018
Why it stopped: Slow accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AnemiaStudies of the same condition, open and under way.
- More trials run by Emory 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
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Ana G. Antun, MD, Emory University
- Investigators
- Ana Antun, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03086252
- Sponsor's protocol ID
- IRB00090306
- Other IDs
- NCI-2016-01059, Winship3145-16
- Status last verified
- Jul 2018
- First submitted
- 17 Mar 2017
- Last updated
- 26 Jul 2018
- 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 26 Jul 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study
- ClinicalTrials.gov ID
- NCT03086252
- Acronym / sponsor ID
- IRB00090306
- Conditions
- Anemia, Aplastic Anemia, Bone Marrow Failure, Hematopoietic and Lymphoid Cell Neoplasm, Leukemia, Myelodysplastic Syndrome
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Slow accrual
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Study start
- 24 Jul 2017 (actual)
- Primary completion
- 31 May 2018 (actual)
- Study completion
- 31 May 2018 (actual)
- First posted
- 22 Mar 2017 (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 pilot study evaluates safety of administration of red blood cell transfusions requested by patients based on their symptoms instead of levels of hemoglobin for the treatment of chronic anemia in patients with blood disorders.
Detailed description
PRIMARY OBJECTIVE:
- To assess feasibility and safety of red blood cell transfusions at the time subjects requested based on symptomatic anemia.
SECONDARY OBJECTIVES:
- To define a patient-driven red blood cell transfusion (RBCT) threshold and to compare the median hemoglobin (hgb) at the time subjects request RBCT to the standard threshold of hgb < 8 g/dL.
II. To compare the number of red blood cell (RBC) units transfused and the frequency of transfusions at the time subjects request RBCT during the study period to the number of RBC units received and frequency using the standard threshold hemoglobin < 8 g/dL during a comparable period prior to enrollment.
III. To compare the quality of life (QoL) scores when subjects request RBCT to the scores obtained at the time hgb is 8 +/- 0.5 g/dL.
OUTLINE:
Patients undergo RBCT based on their perception and/or the presence of anemia symptoms for up to 6 months.
After completion of study treatment, patients are followed up for 30 days.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Supportive care (patient-driven RBCT) | Experimental | Procedure: Red Blood Cell Transfusions (RBCT)Patients undergo red blood cell transfusions (RBCT) based on their perception and/or the presence of anemia symptoms for up to 6 months. |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Diagnosed with hematological malignancy or marrow failure syndrome such as but not limited to: aplastic anemia, myelodysplastic syndrome or leukemia
- Chronic transfusion-dependent anemia with exposure to at least 5 RBCT
- Interested in reducing transfusion exposure
- Willing to sign informed consent
Key exclusion criteria (14 listed)
- Recent acute bleeding requiring intervention in less than 24 hours
- Hemoglobin levels < 6 g/dL
- Acute leukemia receiving induction chemotherapy
- Any patient with known ischemic heart disease, history of congestive heart failure, history of stroke, or cardiac arrhythmia for which the patient requires medication or a medical device
- Oxygen dependent
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 Transfusions (RBCT)Also: Blood Component Transfusion, Blood Transfusion, Transfusion, Transfusion of Blood ProductsUndergo patient-driven RBCT | Procedure or surgery | Supportive care (patient-driven RBCT) |
Primary outcome
Incidence of laboratory and clinical adverse events (AEs) assessed by the Common Terminology Criteria for Adverse Events version 4.03.
Time frame: Up to 6 months of study participation
Frequency, duration, and severity of AEs and severe adverse events will be determined. The proportion of subjects with serious side effects will be calculated with 95% confidence interval. Descriptive statistics will be used to characterize subjects enrolled on the trial: age, sex, race and diagnosis.
5 secondary outcomes
Frequency of RBC units transfused
Time frame: Up to 6 months of study participation
Hemoglobin concentration at the time subject requests RBCT
Time frame: Up to 6 months of study participation
Number of RBC units transfused
Time frame: Up to 6 months of study participation
QoL scores from the Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaire at the time hemoglobin is 8 +/- 0.5 g/dL
Time frame: Up to 6 months of study participation
QoL scores from the Functional Assessment of Cancer Therapy - Anemia/Fatigue (FACT-An) questionnaire at the time subjects request RBCT
Time frame: Up to 6 months of study participation
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jul 2018.
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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.
Ana Antun, MD principal investigator
Emory 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 Red Blood Cell Transfusions (RBCT) in people with Anemia and Aplastic Anemia, run by Emory University at 1 site in United States.
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 4 inclusion and 14 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: withdrawn, as of the sponsor's last update on 26 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 1 site in United States. 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 was withdrawn before enrolling participants, so there are no results.
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?
Incidence of laboratory and clinical adverse events (AEs) assessed by the Common Terminology Criteria for Adverse Events version 4.03. (time frame: Up to 6 months of study participation). 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 26 Jul 2018.