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TerminatedNCT03277183Updated Aug 13, 2020Results posted

Frequent, Low-Dose Erythropoietin A Mechanistic Approach to Mitigate Adverse Cardiovascular Effects of Erythropoietin

A Phase 4 interventional study of Low dose erythropoietin and High dose erythropoietin in Anemia, CKD and Atherosclerosis, sponsored by VA Office of Research and Development. Terminated at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-08-13.

Sponsored by VA Office of Research and Development · Phase 4, Interventional, and Prevention

Why this study was terminated
Lack of enrollment
Phase
Phase 4
Study type
Interventional
Enrollment
5
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

Although several large well designed clinical trials have shown that erythropoietin which is commonly used to treat anemia associated with kidney disease, increases the risk of stroke and heart disease, the mechanism for this increased risk is unknown. The investigators' preliminary studies show that the adverse effects of erythropoietin are from activation of the heterodimeric erythropoietin/ beta common receptor which only occurs with high doses of erythropoietin. The investigators propose a clinical trial of 120 patients assigned to low doses of erythropoietin given more frequently or the same cumulative dose of erythropoietin administered as a high dose once every two weeks and assess effects on the beta common receptor activation, inflammation and vascular disease as evidence by MRI of the carotid arteries.

Read the detailed description

Erythropoietin (EPO) is the most widely prescribed cytokine, yet the benefits and potential side effects of different dosing regimens are poorly understood. It is now recognized that erythropoietin administered at high doses to patients with chronic kidney disease, results in an increased risk of morbidity and mortality from heart disease and stroke. However, the mechanisms that mediate this increased risk of cardiovascular disease is not known. There are two receptors for erythropoietin the homodimeric EPO receptor (EPOR) and the heterodimeric beta common receptor ( CR)/EPOR. The investigators have demonstrated that activation of the heterodimeric CR/EPOR only occurs with high doses of EPO. Our exciting, published, preliminary data also demonstrates that the CR is in a complex with vascular endothelial growth factor receptor-2 (VEGFR-2) and that high doses of EPO activate VEGFR-2 through the CR, resulting in the deleterious effects of VEGFR-2 activation on the cardiovascular system. This is particularly important in patients with kidney disease since they are already at a high risk of cardiovascular disease. Moreover in advanced kidney disease cyanate derived from the high urea levels can non-enzymatically form an amide bond with EPO. This carbamylated EPO (cEPO) has no effect on hemoglobin, but still activates the heterodimeric CR/EPOR. To date there have been no studies that have directly measured levels of cEPO or activation of the CR/EPOR in patients with kidney disease. Our hypothesis is that the administration of low-doses of EPO more frequently will result in lower levels of total and carbamylated erythropoietin decreased activation of VEGFR-2 via the heterodimeric CR/EPOR and consequently decreased inflammation and atherosclerosis. The investigators will directly test this hypothesis by randomly allocating 120 patients with chronic kidney disease to either low- dose EPO given thrice weekly or the same cumulative dose, a high-dose, administered once every 2 weeks. Our hypothesis predicts that low-dose EPO will be as effective at correcting anemia, but will demonstrate less progression of carotid artery plaque, as assessed by non-contrast magnetic resonance imaging, as compared to the high-dose, EPO given every 2 weeks. To delineate how EPO affects blood vessels, the investigators will isolate endothelial cells from blood vessels in 20 patients who are assigned to low-dose EPO and 20 allocated to high-dose EPO. Within these cells the investigators will investigate the signaling pathways that are triggered by activation of CR/EPOR. In a substudy of 20 subjects with kidney disease randomized to low-dose EPO or to high-dose EPO, as well as 20 healthy controls receiving a single dose of high- or low-dose EPO, the investigators will determine how kidney function and dosing affects levels of total and carbamylated erythropoietin. The investigators' study will not only provide us with a thorough understanding of the mechanism by which EPO mediates the increased risk of atherosclerosis, but a clinical strategy to avoid the side effects of EPO therapy and a tool to quantify the cardiovascular risk of EPO and newer erythropoiesis stimulating agents by assessing activation of the heterodimeric CR/EPOR.

02

Conditions studied

  • Anemia
  • CKD
  • Atherosclerosis
  • Cardiovascular

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Keywords

  • erythropoietin
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

The investigators will enroll Veterans who fulfill the following criteria:

  • stage 3, 4, or 5 CKD (estimated glomerular filtration rate of less than 60 ml/min/1.73 m2) on at least two separate occasions greater than 3 months apart; and
  • candidates for EPO therapy as per the National Kidney Foundation's Kidney Disease Outcomes Quality Initiative guidelines (hemoglobin \< 10 gm/dL and anemia of CKD).

Exclusion criteria

Exclusion Criteria:

The investigators will exclude any Veteran who meets any of the following criteria:

  1. pregnant, planning to become pregnant in the next year, or breast feeding;
  2. uncontrolled hypertension (blood pressure > 180/100 mm Hg despite optimal antihypertensive medications);
  3. active gastrointestinal bleeding (visible blood or positive tests for stool occult blood accompanied by a decrease in hemoglobin);
  4. likely to have EPO resistance;
  5. an adverse cardiovascular event in the prior three months;
  6. active or recent (within the last 3 months) severe, systemic infection;
  7. active inflammatory disease such as lupus, rheumatoid arthritis, or vasculitis requiring immunosuppressive or immunomodulatory medications;
  8. history of solid organ transplantation;
  9. expected off-dialysis survival of less than one year (as determined by the estimated glomerular filtration slope and the treating physician;
  10. active cancer (undergoing chemotherapy or radiation within the last 3 months) or primary bone marrow disease such as myelofibrosis; or
  11. a contraindication for an MRI or individuals who cannot comply with the study protocol. The investigators will exclude healthy subjects that meet a, b, f, g, h, or j.
04

Study design

Phase
Phase 4
Primary purpose
Prevention
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Single (Outcomes assessor)
Enrollment
5 participants (actual)

Study arms

  • Placebo comparator
    Low dose erythropoietin

    Subjects randomized to this arm will receive low-dose of EPO administered thrice weekly

    Drug: Low dose erythropoietin

  • Experimental
    High dose erythropoietin

    Subjects randomized to this arm will receive the same cumulative dose of EPO administered as a high-dose of EPO every 2 weeks

    Drug: High dose erythropoietin

Interventions

  • DrugLow dose erythropoietin

    Subjects randomized to this arm will receive low-dose of EPO administered thrice weekly

    Also known as: Low dose

  • DrugHigh dose erythropoietin

    Subjects randomized to this arm will receive the same cumulative dose of EPO administered as a high-dose of EPO every 2 weeks

    Also known as: High dose

05

What researchers measure

Primary outcomes

  1. Change in Carotid Total Plaque Volume From Baseline to Approximately 1 Year, as Assessed by Non-contrast MRI

    The plaque characteristics will be analyzed by MRI-PlaqueViewTM (VP diagnostics Inc., WA). Planimetry will be performed on the image data sets, using histogram equalization to improve edge detection for plaque, arterial wall and lumen. Differences in image contrast between T1-weighted, T2-weighted, Time of Flight, and proton density will be used to characterize the plaque as fibrous, stable, or unstable.

    Time frame: 1 year

Secondary outcomes

  1. Severity of Maximal Stenosis at Baseline and Upon Follow-up.

    The plaque characteristics will be analyzed by MRI-PlaqueViewTM (VP diagnostics Inc., WA). Planimetry will be performed on the image data sets, using histogram equalization to improve edge detection for plaque, arterial wall and lumen. Differences in image contrast between T1-weighted, T2-weighted, Time of Flight, and proton density will be used to characterize the plaque as fibrous, stable, or unstable.

    Time frame: 1 year

  2. Percentage of Total Plaque Area at Baseline and Upon Follow-up.

    The plaque characteristics will be analyzed by MRI-PlaqueViewTM (VP diagnostics Inc., WA). Planimetry will be performed on the image data sets, using histogram equalization to improve edge detection for plaque, arterial wall and lumen. Differences in image contrast between T1-weighted, T2-weighted, Time of Flight, and proton density will be used to characterize the plaque as fibrous, stable, or unstable.

    Time frame: 1 year

  3. Characteristics of Plaques (Soft or Fibrous and Stable or Unstable) at Baseline and Upon Follow-up.

    The plaque characteristics will be analyzed by MRI-PlaqueViewTM (VP diagnostics Inc., WA). Planimetry will be performed on the image data sets, using histogram equalization to improve edge detection for plaque, arterial wall and lumen. Differences in image contrast between T1-weighted, T2-weighted, Time of Flight, and proton density will be used to characterize the plaque as fibrous, stable, or unstable.

    Time frame: 1 year

06

Results

Posted Aug 13, 2020

Participant flow

Participant flow — Overall Study
MilestoneLow Dose ErythropoietinHigh Dose Erythropoietin
Started32
Completed00
Not completed32

Outcome measures

PrimaryChange in Carotid Total Plaque Volume From Baseline to Approximately 1 Year, as Assessed by Non-contrast MRI

The plaque characteristics will be analyzed by MRI-PlaqueViewTM (VP diagnostics Inc., WA). Planimetry will be performed on the image data sets, using histogram equalization to improve edge detection for plaque, arterial wall and lumen. Differences in image contrast between T1-weighted, T2-weighted, Time of Flight, and proton density will be used to characterize the plaque as fibrous, stable, or unstable.

Time frame:
1 year

No measurements were reported for this outcome.

SecondarySeverity of Maximal Stenosis at Baseline and Upon Follow-up.

The plaque characteristics will be analyzed by MRI-PlaqueViewTM (VP diagnostics Inc., WA). Planimetry will be performed on the image data sets, using histogram equalization to improve edge detection for plaque, arterial wall and lumen. Differences in image contrast between T1-weighted, T2-weighted, Time of Flight, and proton density will be used to characterize the plaque as fibrous, stable, or unstable.

Time frame:
1 year

No measurements were reported for this outcome.

SecondaryPercentage of Total Plaque Area at Baseline and Upon Follow-up.

The plaque characteristics will be analyzed by MRI-PlaqueViewTM (VP diagnostics Inc., WA). Planimetry will be performed on the image data sets, using histogram equalization to improve edge detection for plaque, arterial wall and lumen. Differences in image contrast between T1-weighted, T2-weighted, Time of Flight, and proton density will be used to characterize the plaque as fibrous, stable, or unstable.

Time frame:
1 year

No measurements were reported for this outcome.

SecondaryCharacteristics of Plaques (Soft or Fibrous and Stable or Unstable) at Baseline and Upon Follow-up.

The plaque characteristics will be analyzed by MRI-PlaqueViewTM (VP diagnostics Inc., WA). Planimetry will be performed on the image data sets, using histogram equalization to improve edge detection for plaque, arterial wall and lumen. Differences in image contrast between T1-weighted, T2-weighted, Time of Flight, and proton density will be used to characterize the plaque as fibrous, stable, or unstable.

Time frame:
1 year

No measurements were reported for this outcome.

Adverse events

Collected over The time period of adverse events where for the time that each subject was enrolled in the study. No study completed the study since the study was closed prematurely.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Low Dose Erythropoietin0/3 (0%)0/3 (0%)0/3 (0%)
High Dose Erythropoietin0/2 (0%)0/2 (0%)0/2 (0%)

Baseline characteristics

Although baseline MRI's were performed they were not analyzed due to study termination due to lack of enrollment.

Age, Categorical
Age, Categorical(Participants)Low Dose ErythropoietinHigh Dose ErythropoietinTotal
<=18 years000
Between 18 and 65 years213
>=65 years112
Age, Continuous
Age, Continuous(years)Low Dose ErythropoietinHigh Dose ErythropoietinTotal
Mean65 ± 1068 ± 567 ± 8
Sex: Female, Male
Sex: Female, Male(Participants)Low Dose ErythropoietinHigh Dose ErythropoietinTotal
Female000
Male325
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Low Dose ErythropoietinHigh Dose ErythropoietinTotal
Hispanic or Latino000
Not Hispanic or Latino325
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Low Dose ErythropoietinHigh Dose ErythropoietinTotal
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American112
White213
More than one race000
Unknown or Not Reported000
Region of Enrollment
Region of Enrollment(Participants)Low Dose ErythropoietinHigh Dose ErythropoietinTotal
United States325
07

Study locations

1 site
  • North Florida/South Georgia Veterans Health System, Gainesville, FL
    Gainesville, Florida 32608, United States
08

References and documents

Study documents

  • Protocol and statistical analysis plan · Apr 23, 2019

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

09

Registry details

Key details

Study ID
NCT03277183
Lead sponsor
VA Office of Research and Development
Responsible party
Sponsor
First posted
Sep 8, 2017
Start date
Nov 2, 2017
Primary completion
Jun 3, 2019
Completion
Jun 3, 2019
Results posted
Aug 13, 2020
Last update
Aug 13, 2020

Study contacts

Mark S. Segal, MD PhD
principal investigator · North Florida/South Georgia Veterans Health System, Gainesville, FL

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
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