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CompletedNCT04415658Updated Jan 10, 2024Results posted

Intravenous Thyroxine for Heart-Eligible Organ Donors

A Phase 3 interventional study of Thyroxine and Saline in Brain Death and Heart Failure, sponsored by Washington University School of Medicine. Completed at 14 sites in United States. Open to participants aged 14 Years to 55 Years. Per ClinicalTrials.gov, last updated 2024-01-10.

Sponsored by Washington University School of Medicine · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
838
Allocation
Randomized
Ages
14 Years to 55 Years
Sex
All
01

Study summary

This randomized controlled trial will evaluate whether intravenous thyroxine infusion given to brain-dead organ donors who are eligible to donate hearts for 12 hours will result in more hearts transplanted than saline placebo

Read the detailed description

Background: Brain death frequently induces hemodynamic instability and cardiac stunning. Impairments in cardiac performance are major contributors to hearts from otherwise eligible organ donors not being transplanted. Deficiencies in pituitary hormones (including thyroid stimulating hormone) may contribute to hemodynamic instability and replacement of thyroid hormone has been proposed as a means of improving stability and increasing hearts available for transplantation. Intravenous thyroxine is commonly used in donor management. However, small controlled trials have not been able to demonstrate efficacy.

Methods: This multicenter study will involve organ procurement organizations (OPOs) across the country. A total of 800 heart-eligible brain dead organ donors who require vasopressor support will be randomly assigned to intravenous thyroxine for at least 12-hours or saline placebo. The primary study hypothesis is that thyroxine treatment results in more hearts transplanted. Additional outcome measures are time to achieve hemodynamic stability (weaning off vasopressors) and improvement in cardiac ejection fraction on echocardiography.

Discussion: This will be the largest randomized controlled study to evaluate the efficacy of thyroid hormone treatment for organ donor management. By collaborating across multiple OPOs, it will be able to enroll an adequate number of donors and be powered to definitively answer the critical question of whether treatment increases hearts transplanted and/or provides other hemodynamic benefits.

02

Conditions studied

  • Brain Death
  • Heart Failure

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Keywords

  • Organ donor
03

Who can participate

Ages eligible
14 Years to 55 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Declared dead by neurologic criteria (brain dead)
  • Authorization for organ donation and research
  • On one or more vasopressors and/or inotropes

Exclusion criteria

Exclusion Criteria:

  • Brain death declared more than 24 hours prior
  • Only vasopressor is vasopressin
  • Weight \< 45 kg (100 lbs)
  • Known coronary artery disease or history of myocardial infarction
  • Known valvular heart disease
  • Prior sternotomy or cardiac surgery
  • Donor at VA hospital
  • Received intravenous or oral thyroxine within past month
  • Known HIV+ status
  • Other reason donor is unable to receive study drug (determined by on-site personnel)
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
838 participants (actual)

Study arms

  • Experimental
    Thyroxine

    Intravenous thyroxine infusion

    Drug: Thyroxine

  • Placebo comparator
    Saline Placebo

    Intravenous saline infusion

    Drug: Saline

Interventions

  • DrugThyroxine

    Infusion prepared by mixing 500 μg of drug in 500-ml of normal saline (i.e. concentration of 1 μg/ml) and enclosing the bag in an opaque sleeve. Infusion started at 30 μg/hour (30 ml/hour) for twelve hours.

  • DrugSaline

    The placebo will be a 500-ml bag of normal saline (without active drug) also enclosed in an opaque sleeve. This infusion will also be started at 30 ml/hour for twelve hours.

05

What researchers measure

Primary outcomes

  1. Heart Transplanted

    Whether heart is transplanted into living recipient

    Time frame: One week

  2. Graft Function

    30-day graft survival of hearts transplanted from study donors, obtained from SRTR recipient registry

    Time frame: 30 days

Secondary outcomes

  1. Time Till Off Vasopressors

    Time in hours from randomization to when weaned off vasopressors (except vasopressin)

    Time frame: 72 hours

  2. Weaned Off Vasopressors

    Weaned off vasopressors within twelve hours

    Time frame: 12 hours

  3. Time to Order Echo

    Time till hemodynamic stability permits ordering initial echocardiogram

    Time frame: 72 hours

  4. Ejection Fraction

    Left ventricular ejection fraction measured on first echocardiography

    Time frame: 72 hours

  5. Lungs Transplanted

    Whether the lungs were transplanted into a living recipient

    Time frame: 72 hours

Other outcomes

  1. Total Organs Transplanted

    Total number of organs transplanted (incl. lungs, liver, kidneys, heart, pancreas)

    Time frame: One week

06

Results

Posted Jan 10, 2024
Limitations and caveats
Not blinded, open-label use of levothyroxine permitted in control group after 12 hours (only 50 donors in that group received open-label, plus three who crossed-over to levothyroxine immediately after randomization)

Participant flow

Initial Study Period (12 Hours)
Participant flow — Initial Study Period (12 Hours)
MilestoneThyroxineSaline Placebo
Started419419
Started assigned infusion411384
Per-protocol351374
Cross-over03
Stopped infusion early860
Completed333416
Not completed863
Open-Label (T4 Beyond 12 Hours)
Participant flow — Open-Label (T4 Beyond 12 Hours)
MilestoneThyroxineSaline Placebo
Started333416
Completed20950
Not completed124366

Outcome measures

PrimaryHeart Transplanted

Whether heart is transplanted into living recipient

Time frame:
One week
Reported as:
Count of participants · Participants
Heart Transplanted
ParticipantsThyroxineSaline Placebo
Heart Transplanted230223
Statistical analysis
  • Thyroxine vs Saline Placebo · Chi-squared, Corrected · p = 0.57 · Risk ratio (rr): 1.01 · 95% CI 0.97 to 1.07
PrimaryGraft Function

30-day graft survival of hearts transplanted from study donors, obtained from SRTR recipient registry

Time frame:
30 days
Reported as:
Count of units · hearts
Graft Function
heartsThyroxineSaline Placebo
Graft Function224213
Statistical analysis
  • Thyroxine vs Saline Placebo · Regression, Logistic · p = <0.001 · Mean difference (final values): 1.9 · 95% CI -2.3 to 6.0
SecondaryTime Till Off Vasopressors

Time in hours from randomization to when weaned off vasopressors (except vasopressin)

Time frame:
72 hours
Reported as:
Median · hours
Time Till Off Vasopressors
hoursThyroxineSaline Placebo
Time Till Off Vasopressors22 (19 to 31)25 (19 to 38)
SecondaryWeaned Off Vasopressors

Weaned off vasopressors within twelve hours

Time frame:
12 hours
Reported as:
Count of participants · Participants
Weaned Off Vasopressors
ParticipantsThyroxineSaline Placebo
Weaned Off Vasopressors143152
SecondaryTime to Order Echo

Time till hemodynamic stability permits ordering initial echocardiogram

Time frame:
72 hours
Reported as:
Median · hours
Time to Order Echo
hoursThyroxineSaline Placebo
Time to Order Echo12 (11 to 13)13 (12 to 14)
SecondaryEjection Fraction

Left ventricular ejection fraction measured on first echocardiography

Time frame:
72 hours
Reported as:
Mean · percentage of ejection fraction
Ejection Fraction
percentage of ejection fractionThyroxineSaline Placebo
Ejection Fraction59 ± 1158 ± 12
SecondaryLungs Transplanted

Whether the lungs were transplanted into a living recipient

Time frame:
72 hours
Reported as:
Count of participants · Participants
Lungs Transplanted
ParticipantsThyroxineSaline Placebo
Lungs Transplanted163149
Other pre-specifiedTotal Organs Transplanted

Total number of organs transplanted (incl. lungs, liver, kidneys, heart, pancreas)

Time frame:
One week
Reported as:
Median · organs
Total Organs Transplanted
organsThyroxineSaline Placebo
Total Organs Transplanted4 (3 to 5)4 (3 to 5)

Adverse events

Collected over 1 week. Non-serious events are listed at a 1% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Thyroxine419/419 (100%)2/419 (0.5%)51/419 (12.2%)
Saline Placebo419/419 (100%)3/419 (0.7%)16/419 (3.8%)
Most frequent serious events
Most frequent serious events
EventThyroxineSaline Placebo
Cardiac arrest / donor lossCardiac disorders2/4193/419
Most frequent other events
Most frequent other events
EventThyroxineSaline Placebo
HypertensionCardiac disorders26/4195/419
TachycardiaCardiac disorders16/4193/419
Atrial fibrillationCardiac disorders7/4197/419
Ventricular ectopyCardiac disorders5/4195/419
Ventricular tachycardiaCardiac disorders4/4191/419
Misc/OtherGeneral disorders4/4192/419

Baseline characteristics

Age, Continuous
Age, Continuous(years)ThyroxineSaline PlaceboTotal
Mean36 ± 1136 ± 1036 ± 10
Sex: Female, Male
Sex: Female, Male(Participants)ThyroxineSaline PlaceboTotal
Female127153280
Male276247523
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)ThyroxineSaline PlaceboTotal
White, non-Hispanic252278530
Black7549124
Hispanic6063123
Asian, Pacific-Islander, other16723
Region of Enrollment
Region of Enrollment(Participants)ThyroxineSaline PlaceboTotal
United States419419838
Blood Group
Blood Group(Participants)ThyroxineSaline PlaceboTotal
O224214438
A134148282
B504999
AB11819
Cause of Death
Cause of Death(Participants)ThyroxineSaline PlaceboTotal
Anoxia162195357
Stroke / Cerebrovascular8779166
Trauma143117260
Other11617
07

Study locations

14 sites
  • Donor Network of Arizona
    Phoenix, Arizona 85013, United States
  • Lifesharing
    San Diego, California 92108, United States
  • Donor Alliance
    Denver, Colorado 80230, United States
  • OurLegacy
    Maitland, Florida 32751, United States
  • Iowa Donor Network
    North Liberty, Iowa 52317, United States
  • Midwest Transplant Network
    Westwood, Kansas 66205, United States
  • Louisiana Organ Procurement Agency
    Covington, Louisiana 70433, United States
  • Mid-America Transplant Services
    Saint Louis, Missouri 63110, United States
  • Lifebanc
    Cleveland, Ohio 44128, United States
  • LifeShare of Oklahoma
    Oklahoma City, Oklahoma 73132, United States
  • Southwest Transplant Alliance
    Dallas, Texas 75231, United States
  • Texas Organ Sharing Alliance
    San Antonio, Texas 78229, United States
  • DonorConnect
    Murray, Utah 84107, United States
  • LifeCenter Northwest
    Bellevue, Washington 98006, United States
08

References and documents

Publications

  • Dhar R, Stahlschmidt E, Yan Y, Marklin G. A randomized trial comparing triiodothyronine (T3) with thyroxine (T4) for hemodynamically unstable brain-dead organ donors. Clin Transplant. 2019 Mar;33(3):e13486. doi: 10.1111/ctr.13486. Epub 2019 Feb 12. PubMed 30689222 ↗
  • Dhar R, Stahlschmidt E, Marklin G. A Randomized Trial of Intravenous Thyroxine for Brain-Dead Organ Donors With Impaired Cardiac Function. Prog Transplant. 2020 Mar;30(1):48-55. doi: 10.1177/1526924819893295. Epub 2019 Dec 5. PubMed 31802716 ↗
  • Dhar R, Marklin GF, Klinkenberg WD, Wang J, Goss CW, Lele AV, Kensinger CD, Lange PA, Lebovitz DJ. Intravenous Levothyroxine for Unstable Brain-Dead Heart Donors. N Engl J Med. 2023 Nov 30;389(22):2029-2038. doi: 10.1056/NEJMoa2305969. PubMed 38048188 ↗
  • Dhar R, Klinkenberg D, Marklin G. A multicenter randomized placebo-controlled trial of intravenous thyroxine for heart-eligible brain-dead organ donors. Trials. 2021 Nov 27;22(1):852. doi: 10.1186/s13063-021-05797-2. PubMed 34838132 ↗

Study documents

  • Study protocol · Dec 1, 2020
  • Protocol and statistical analysis plan · Nov 1, 2021

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

Individual participant data

Plan to share: No — Researchers within the study consortium only

09

Registry details

Key details

Study ID
NCT04415658
Lead sponsor
Washington University School of Medicine
Collaborators
Mid-America Transplant
Responsible party
Rajat Dhar (Professor, Washington University School of Medicine) — Principal investigator
First posted
Jun 4, 2020
Start date
Dec 1, 2020
Primary completion
Nov 6, 2022
Completion
Dec 6, 2022
Results posted
Jan 10, 2024
Last update
Jan 10, 2024

Study contacts

Rajat Dhar, MD
principal investigator · Washington University School of Medicine

Oversight

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

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