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CompletedNCT03440970Updated Sep 23, 2026Results posted

Mechanism and Effects of Manipulating Chloride Homeostasis in Stable Heart Failure

An Early Phase 1 interventional study of Lysine Chloride and Placebo in Decompensated Heart Failure, sponsored by Yale University. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-09-23.

Sponsored by Yale University · Early Phase 1, Interventional, and Diagnostic

Phase
Early Phase 1
Study type
Interventional
Enrollment
20
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study summary

This study is designed to investigate the quantitative effects of sodium-free chloride supplementation on electrolyte balance, volume status, and sodium avidity in stable heart failure patients in a highly controlled environment.

Read the detailed description

The overarching goal of this study is to develop a comprehensive understanding of the biology and therapeutic potential of sodium-free chloride supplementation. While sodium homeostasis has been the focus of substantial investigation, very little research has been devoted to understanding chloride homeostasis. Thus, this proposal is designed to obtain the full spectrum of information pertaining to chloride, such as novel areas with great interest by the scientific community (i.e. modulation of the WNK-kinase system and the use of exosomes), to more practical/basic questions (i.e. what happens to sodium chloride balance when a patient is challenged with chloride).

This study is designed as a highly controlled inpatient "GCRC" arm to be compared to a real world efficacy study that has been proposed as a separate study. With extensive biobanking and analysis of samples in the inpatient setting, we will be able to deliver a great wealth of information on the biology and therapeutic potential of manipulating chloride homeostasis in heart failure.

02

Conditions studied

  • Decompensated Heart Failure

Keywords

  • Chloride homeostasisH
03

Who can participate

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

Inclusion criteria

  • Meticulous history of medical compliance and attendance of appointments
  • Stable heart failure as defined by:

    1. Absence of hospitalizations for 90 days
    2. Stable diuretic and medical therapy for 30 days
    3. Opinion of the patient's treating physician (Heart Failure Cardiologist) that the patient is at optimal volume status
  • Evidence based heart failure treatment with maximally-tolerated doses of a beta blocker, ACE/ARB/neprilysin inhibitor and aldosterone antagonist
  • Chronic loop diuretic therapy with ≥ 40 mg of furosemide equivalents
  • Serum chloride \<102 mmol/L

Exclusion criteria

Exclusion Criteria:

  • Inability to commit to or comply with the rigorous study protocol
  • Use of a thiazide diuretic in the last 30 days
  • History of metabolic or respiratory acidosis
  • Use of metformin, acetazolamide, or any other agent that could predispose to acidosis. Patients who are on metformin may be enrolled if their metformin can be safely discontinued for the randomized periods in each arm. Any participants who have consistently elevated Blood glucose readings > 200 mg/dL while inpatient will not be enrolled.
  • Serum bicarbonate level \<24mmol/L
  • Serum pH \<7.3
  • Estimated glomerular filtration rate \<30 mL/min or prior or current history of renal replacement therapy
  • Anemia, as defined by Hemoglobin \<8.0 g/dL at screening visit
  • Urinary incontinence or significant bladder dysfunction (post-void residual at screening >300 mL)
  • Use of chloride containing medications that provide more than 5 mmol/day of chloride if the medication cannot be discontinued or substituted
  • Appears unlikely, or unable to participate in the required study procedures, as assessed by the study PI or research RN (ex: clinically-significant psychiatric, addictive, or neurological disease)
  • Inability to give written informed consent or follow study protocol
04

Study design

Phase
Early Phase 1
Primary purpose
Diagnostic
Allocation
Randomized
Intervention model
Crossover assignment
Masking
Triple (Participant, Investigator, Outcomes assessor)
Enrollment
20 participants (actual)

Study arms

  • Experimental
    Lysine Chloride

    Patients will be randomized to receive either lysine chloride or placebo. Patients will receive the study drug thrice daily for 5 days of randomized therapy, starting after the completion of a blood volume assessment.

    Drug: Lysine Chloride

  • Placebo comparator
    Placebo

    Patients will be randomized to receive either lysine chloride or placebo. Patients will receive the study drug thrice daily for 5 days of randomized therapy, starting after the completion of a blood volume assessment.

    Other: Placebo

Interventions

  • DrugLysine Chloride

    Patients will receive the study drug thrice daily for 5 days.

  • OtherPlacebo

    Patients will receive the placebo thrice daily for 5 days.

05

What researchers measure

Primary outcomes

  1. Mean Change in Blood Volume

    Volumex is albumin labeled with the iodine isotope I-131 and is an FDA-approved method used to determine total blood volume. A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Day 1 and day 7 measures of blood collection will be compared between intervention and placebo arms. Data presented here is the change from Day 1 to Day 7.

    Time frame: Day 7

  2. Mean Change in Plasma Volume

    Volumex is albumin labeled with the iodine isotope I-131 and is an FDA-approved method used to determine plasma volume. A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Day 1 and day 7 measures of blood collection will be compared between intervention and placebo arms. Data presented here is the change from Day 1 to Day 7.

    Time frame: Day 7

Secondary outcomes

  1. Mean Change in Serum Creatinine Concentration

    A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Daily measures of serum creatinine will be compared across the 7 day collection period between intervention and placebo arms.

    Time frame: Daily for 7-days

  2. Mean Change in Cystatin C Concentration

    A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Daily measures of cystatin C will be compared across the 7 day collection period between intervention and placebo arms.

    Time frame: Daily for 7-days

  3. Mean Change in Chloride Concentration

    A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Daily measures of chloride will be compared across the 7 day collection period between intervention and placebo arms.

    Time frame: Daily for 7-days

  4. Mean Change in Bicarbonate Concentration

    A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Daily measures of bicarbonate will be compared across the 7 day collection period between intervention and placebo arms.

    Time frame: Daily for 7-days

06

Results

Posted Sep 23, 2026

Participant flow

First intervention
Participant flow — First intervention
MilestoneLysine Chloride, Then PlaceboPlacebo, Then Lysine Chloride
Started119
Completed119
Not completed00
Second intervention
Participant flow — Second intervention
MilestoneLysine Chloride, Then PlaceboPlacebo, Then Lysine Chloride
Started119
Completed119
Not completed00

Outcome measures

PrimaryMean Change in Blood Volume

Volumex is albumin labeled with the iodine isotope I-131 and is an FDA-approved method used to determine total blood volume. A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Day 1 and day 7 measures of blood collection will be compared between intervention and placebo arms. Data presented here is the change from Day 1 to Day 7.

Time frame:
Day 7
Reported as:
Mean · milliliters
Mean Change in Blood Volume
millilitersLysine ChloridePlacebo
Mean Change in Blood Volume-275 (-453 to -97)73 (-161 to 308)
Statistical analysis
  • Lysine Chloride vs Placebo · Mixed Models Analysis · p = 0.036Linear mixed models
PrimaryMean Change in Plasma Volume

Volumex is albumin labeled with the iodine isotope I-131 and is an FDA-approved method used to determine plasma volume. A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Day 1 and day 7 measures of blood collection will be compared between intervention and placebo arms. Data presented here is the change from Day 1 to Day 7.

Time frame:
Day 7
Reported as:
Mean · milliliters
Mean Change in Plasma Volume
millilitersLysine ChloridePlacebo
Mean Change in Plasma Volume-152 (-277 to -26)91 (-77 to 259)
Statistical analysis
  • Lysine Chloride vs Placebo · Mixed Models Analysis · p = 0.018Linear mixed models
SecondaryMean Change in Serum Creatinine Concentration

A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Daily measures of serum creatinine will be compared across the 7 day collection period between intervention and placebo arms.

Time frame:
Daily for 7-days
Reported as:
Mean · mg/dL per day
Mean Change in Serum Creatinine Concentration
mg/dL per dayLysine ChloridePlacebo
Mean Change in Serum Creatinine Concentration-0.001 (-0.013 to 0.011)0.003 (-0.009 to 0.014)
Statistical analysis
  • Lysine Chloride vs Placebo · Mixed Models Analysis · p = 0.69Linear mixed models
SecondaryMean Change in Cystatin C Concentration

A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Daily measures of cystatin C will be compared across the 7 day collection period between intervention and placebo arms.

Time frame:
Daily for 7-days
Reported as:
Mean · mg/L per day
Mean Change in Cystatin C Concentration
mg/L per dayLysine ChloridePlacebo
Mean Change in Cystatin C Concentration-0.023 (-0.036 to -0.009)-0.009 (-0.023 to 0.004)
Statistical analysis
  • Lysine Chloride vs Placebo · Mixed Models Analysis · p = 0.16Linear mixed models
SecondaryMean Change in Chloride Concentration

A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Daily measures of chloride will be compared across the 7 day collection period between intervention and placebo arms.

Time frame:
Daily for 7-days
Reported as:
Mean · mmol/L per day
Mean Change in Chloride Concentration
mmol/L per dayLysine ChloridePlacebo
Mean Change in Chloride Concentration1.2 (0.9 to 1.6)0.0 (-0.4 to 0.4)
Statistical analysis
  • Lysine Chloride vs Placebo · Mixed Models Analysis · p = <0.001Linear mixed models
SecondaryMean Change in Bicarbonate Concentration

A linear mixed effect model will be used to analyze the trial with class variables of time and treatment group. Daily measures of bicarbonate will be compared across the 7 day collection period between intervention and placebo arms.

Time frame:
Daily for 7-days
Reported as:
Mean · mmol/L per day
Mean Change in Bicarbonate Concentration
mmol/L per dayLysine ChloridePlacebo
Mean Change in Bicarbonate Concentration-1.4 (-1.7 to -1.0)-0.4 (-0.7 to -0.01)
Statistical analysis
  • Lysine Chloride vs Placebo · Mixed Models Analysis · p = <0.001Linear mixed models

Adverse events

Collected over approximately 20 days. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Lysine Chloride0/20 (0%)0/20 (0%)13/20 (65%)
Placebo0/20 (0%)0/20 (0%)7/20 (35%)
Most frequent other events
Showing 10 of 11
Most frequent other events
EventLysine ChloridePlacebo
DiarrheaGastrointestinal disorders7/202/20
ConstipationGastrointestinal disorders0/201/20
TremorsNervous system disorders0/201/20
DizzinessNervous system disorders1/201/20
HypertensionVascular disorders0/201/20
HypotensionVascular disorders0/201/20
Sore throatRespiratory, thoracic and mediastinal disorders1/200/20
MalaiseGeneral disorders1/200/20
NauseaGastrointestinal disorders1/200/20
CrampingMusculoskeletal and connective tissue disorders1/200/20

Baseline characteristics

Age, Continuous
Age, Continuous(years)Lysine Chloride, Then PlaceboPlacebo, Then Lysine ChlorideTotal
Mean57.1 ± 8.551.4 ± 15.354.5 ± 12
Sex: Female, Male
Sex: Female, Male(Participants)Lysine Chloride, Then PlaceboPlacebo, Then Lysine ChlorideTotal
Female426
Male7714
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Lysine Chloride, Then PlaceboPlacebo, Then Lysine ChlorideTotal
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American527
White5712
More than one race000
Unknown or Not Reported101
Region of Enrollment
Region of Enrollment(participants)Lysine Chloride, Then PlaceboPlacebo, Then Lysine ChlorideTotal
United States11920
07

Study locations

1 site
  • Yale University
    New Haven, Connecticut 06510, United States
08

References and documents

Study documents

  • Protocol and statistical analysis plan · Sep 24, 2024
  • Informed consent form · Jul 27, 2022

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
NCT03440970
Lead sponsor
Yale University
Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
Responsible party
Sponsor
First posted
Feb 22, 2018
Start date
Jan 15, 2019
Primary completion
Aug 30, 2025
Completion
Aug 30, 2025
Results posted
Sep 23, 2026
Last update
Sep 23, 2026

Study contacts

Jeffrey M Testani, MD
principal investigator · Yale University

Oversight

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

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