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TerminatedNCT03565328Updated Oct 28, 2020Results posted

The Effect of Nicotinamide Riboside on Skeletal Muscle Function in Heart Failure Subjects

A Phase 2 interventional study of Nicotinamide Riboside in Heart Failure, sponsored by National Heart, Lung, and Blood Institute (NHLBI). Terminated at 2 sites in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2020-10-28.

Sponsored by National Heart, Lung, and Blood Institute (NHLBI) · Phase 2, Interventional, and Basic science

Why this study was terminated
Slow/insufficient accrual and investigators have left the study.
Phase
Phase 2
Study type
Interventional
Enrollment
2
Allocation
Not applicable
Ages
18 Years to 75 Years
Sex
All
01

Study summary

Background:

People are living longer and are more likely to survive a heart attack if they have one. Longer life expectancy is good but it also means more people get chronic heart failure over time. This is a condition in which the heart doesn't pump blood as well as it should. Treatment of chronic heart failure has not improved much in a few decades. Researchers want to see if giving a dietary supplement to people with heart failure can help their heart function. The supplement is nicotinamide riboside (NR).

Objective:

To study how NR affects skeletal muscle function in people with heart failure.

Eligibility:

Adults ages 18-70 with clinically stable systolic heart failure

Design:

Participants will be screened with a medical history and physical exam. They will answer demographic questions and review their current medical treatments. They will have blood and urine tests. They will have an echocardiogram. This uses sound waves to test heart function.

Participants will have 8 study visits over 16 weeks. At these visits, they will have some of the following:

Repeat of screening tests

Skin sample taken

Skeletal muscle exercise Nuclear magnetic resonance (NMR) spectroscopy. Muscles will be measured while participants do foot exercises.

Cardiopulmonary exercise testing. Participants may ride a stationary bike or walk on a treadmill. A facemask will analyze their breath. Heart and blood pressure measurements will be taken.

Participants will take the supplement in pill form each day for 12 weeks. Pill bottles will be checked at study visits.

Participants should not significantly change their activity levels during the study.

Read the detailed description

As life expectancy increases and acute cardiac mortality decreases, the incidence of chronic heart failure (HF) continues to rise, and despite this, conceptual advances in the treatment of chronic heart failure have not increased substantially over last few decades. One intracellular component of heart failure progression is mitochondrial bioenergetic dysfunction. Although the mechanism underpinning this is not completely understood, recent metabolomics data demonstrated an incomplete flux of metabolites through oxidative phosphorylation (OX PHOS) in HF. In parallel, data has shown that hyperacetylation of mitochondrial bioenergetic enzymes, with the concomitant blunting of enzymatic activity is evident in HF. Putting these together, an emerging hypothesis implicates excessive acetylation of mitochondrial proteins with the subsequent blunting of bioenergetic enzyme function, as a mechanism underpinning incomplete flux through OX PHOS resulting in HF progression.

In parallel with cardiac bioenergetic deficiency chronic HF subjects display disrupted skeletal muscle OX PHOS, which is thought to contribute towards overall fatigue and reduced exercise tolerance. Interestingly exercise training in HF subjects improves skeletal muscle mitochondrial OX PHOS capacity and subject activity levels. Exercise training additionally increases activity of the mitochondrial regulatory deacetylase sirtuin enzymes SIRT1 and SIRT3, in parallel with improved skeletal muscle OX PHOS capacity. At the same time HF-associated disruption in skeletal muscle metabolic function activates skeletal muscle cytokine production. These inflammatory programs, in turn, are proposed to contribute towards impaired functional capacity in HF. Interestingly, and mirroring improved OX PHOS following exercise programs in HF studies, exercise training similarly reduces skeletal muscle inflammatory effects.

Biochemical and bioenergetic consequences of impaired mitochondrial OX PHOS leads to decreased NAD+ levels, which exacerbate mitochondrial dysfunction by inactivating the NAD+ dependent sirtuin enzymes. Experimental studies using NAD+ precursors to increase NAD+ production have been shown to normalize NADH/NAD+ ratios and activate Sirtuin enzymes, resulting in enhanced OX PHOS with beneficial effects in numerous systems including skeletal muscle and in the blunting of inflammation.

In this pilot study we will directly assess the effect of the NAD+ precursor, nicotinamide riboside (NR) on skeletal muscle mitochondrial OX PHOS in HF subjects using: skeletal muscle Nuclear magnetic resonance (NMR) spectroscopy assessment of the rate of high energy phosphate recovery in response to submaximal exercise; assessment of the effect of NR on functional capacity using cardiopulmonary exercise testing (CPET) to determine VO(2max) and anaerobic threshold; evaluation of the NR effect on serum metabolomics at rest and in response to CPET; and by measuring circulating cytokine levels pre- and post- NR administration. These studies would enable a more comprehensive assessment of the role for NR supplementation on skeletal muscle mitochondrial function in subjects with systolic HF

02

Conditions studied

  • Heart Failure

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Keywords

  • Mitochondrial bioenergetic dysfunction
  • Mitochondrial oxidative phosphorylation in skeletal muscles
  • Hyperacetylation of mitochondrial bioenergetic enzymes
  • NAD+ dependent sirtuin enzymes
  • NAD+/NADH ratio
03

In context

Heart Failure

5,701 studies on the registry are indexed under Heart Failure; 1,220 are open to participants now.

This study's enrollment of 2 is below the median of 72 across 3,736 interventional studies indexed under Heart Failure.

Browse Heart Failure studies →

Lead sponsor

National Heart, Lung, and Blood Institute (NHLBI) is the lead sponsor of 1,117 studies on the registry; 71 are open to participants now.

Of its 57 completed or terminated interventional studies of FDA-regulated products, 49 (86%) have results posted.

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

04

Who can participate

Ages eligible
18 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

Men and women between the ages of 18 and 75 years with New York Heart Association (NYHA) Functional Classification Class II-III systolic heart failure ( Left Ventricular Ejection Fraction (LVEF) by standard echocardiography or radionuclide ventriculography of less than or equal to 45%) deemed to be non-ischemic or ischemic in origin.

Clinically stable (no cardiac procedures or hospitalizations for hospitalizations for cardiac causes, including HF, ischemia or arrhythmia) within the previous 3 months

Ability to undergo study procedures, including scheduled visits, blood draws, skeletal muscle exercise NMR spectroscopy and CPET testing

Willingness/ability to provide informed consent

Must be DEERS eligible to be enrolled in a research protocol at Walter Reed National Military Medical Center (WRNMMC).

Exclusion criteria

EXCLUSION CRITERIA:

Heart failure with preserved ejection fraction (LVEF greater than 45%)

Change in heart failure medications due to deterioration of function with the exception of up- or down-titration of diuretic dose up to 100% of baseline dose.

Heart failure due to etiologies other than non-ischemic or ischemic. Examples of exclusionary heart failure etiologies include primary valvular disease, or infiltrative or inflammatory cardiomyopathies.

Cardiac surgery, percutaneous coronary intervention (PCI) or cardiac device implantation within the previous 3 months

Hospitalizations for cardiovascular causes, including heart failure, chest pain, stroke/TIA or arrhythmias within the previous 3 months

Inability to perform Study visits or procedures (e.g., physical inability to perform exercise testing)

Unwillingness/inability to provide informed consent

ALT greater than x3 upper limit of normal, hepatic insufficiency or active liver disease

Recent history of acute gout

Chronic renal insufficiency with creatinine greater than 2.5mg/dl

Pregnant (or likely to become pregnant) women

Significant co-morbidity likely to cause death in the 6 month follow-up period

Significant active history of substance abuse within the previous 5 years

Current participation in another drug study

History of intolerance to NR precursor compounds, including niacin or nicotinamide

MRI incompatible hardware including pacemakers or ICD s

Study adherence concerns

Individuals with diabetes type 1 and 2 who use insulin

Women of child-bearing potential unwilling to use contraception or unwilling to practice abstinence

Breastfeeding women unwilling to stop breastfeeding

05

Study design

Phase
Phase 2
Primary purpose
Basic science
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
2 participants (actual)

Study arms

  • Experimental
    Nicotinamide Riboside in patients with stable heart failure

    Nicotinamide Riboside (NR) will be started at 500 mg daily (250 mg BID) then increased at two weekly intervals by 250 mg/dose (BID) (500 mg/day) to a final dose of 1000 mg PO BID (2000 mg/day) in patients with stable, systolic heart failure.

    Dietary Supplement: Nicotinamide Riboside

Interventions

  • Dietary supplementNicotinamide Riboside

    Nicotinamide Riboside (NR) will be started at 500 mg daily (250 mg BID) be increased at two weekly intervals by 250 mg/dose (BID) (500 mg/day) to a final dose of 1000mg PO BID (2000 mg/day).

    Also known as: Niagen; Nicotinamide Riboside (NR)

06

What researchers measure

Primary outcomes

  1. Number of Participants Who Had Enhancement of Mitochondrial Function in Skeletal Muscle

    Number of participants who had enhancement mitochondrial function in skeletal muscle Nuclear magnetic resonance (NMR) spectroscopy will be performed at baseline, at the end of the 12-week Nicotinamide Riboside (NR) supplementation period and repeated 4 weeks post-NR washout.

    Time frame: baseline-12 week-16 week

Secondary outcomes

  1. Measure the Effects of Nicotinamide Riboside (NR) on Oxidative Phosphorylation and Inflammation in Respective Subject Primary Skin Fibroblasts

    Measure the effects in participants of Nicotinamide Riboside (NR) on oxidative phosphorylation and inflammation in respective subject primary skin fibroblasts

    Time frame: Baseline, 12 week

  2. Number of Participants Without Heart Failure Linked Inflammation

    To quantitative serum cytokine immunoassay profiling to assess whether Nicotinamide Riboside (NR) blunts heart failure (HF) linked inflammation

    Time frame: Baseline, 12 week

  3. Number of Participants Who Experienced an Increased Rate of Oxidative Phosphorylation

    Serum quantitative metabolomic profiling, pre and post-cardiopulmonary exercise testing (CPET) to evaluate whether Nicotinamide Riboside (NR) increases the rate of oxidative phosphorylation

    Time frame: Baseline, 12 week

  4. VO2 Max and Anaerobic Thresholds

    Analysis of VO2max and the anaerobic threshold will be measured.

    Time frame: Baseline, 12 week

07

Results

Posted Oct 28, 2020

Participant flow

Participant flow — Overall Study
MilestoneNicotinamide Riboside in Patients With Stable Heart Failure
Started1
Completed1
Not completed0

Outcome measures

PrimaryNumber of Participants Who Had Enhancement of Mitochondrial Function in Skeletal Muscle

Number of participants who had enhancement mitochondrial function in skeletal muscle Nuclear magnetic resonance (NMR) spectroscopy will be performed at baseline, at the end of the 12-week Nicotinamide Riboside (NR) supplementation period and repeated 4 weeks post-NR washout.

Time frame:
baseline-12 week-16 week

No measurements were reported for this outcome.

SecondaryMeasure the Effects of Nicotinamide Riboside (NR) on Oxidative Phosphorylation and Inflammation in Respective Subject Primary Skin Fibroblasts

Measure the effects in participants of Nicotinamide Riboside (NR) on oxidative phosphorylation and inflammation in respective subject primary skin fibroblasts

Time frame:
Baseline, 12 week

No measurements were reported for this outcome.

SecondaryNumber of Participants Without Heart Failure Linked Inflammation

To quantitative serum cytokine immunoassay profiling to assess whether Nicotinamide Riboside (NR) blunts heart failure (HF) linked inflammation

Time frame:
Baseline, 12 week

No measurements were reported for this outcome.

SecondaryNumber of Participants Who Experienced an Increased Rate of Oxidative Phosphorylation

Serum quantitative metabolomic profiling, pre and post-cardiopulmonary exercise testing (CPET) to evaluate whether Nicotinamide Riboside (NR) increases the rate of oxidative phosphorylation

Time frame:
Baseline, 12 week

No measurements were reported for this outcome.

SecondaryVO2 Max and Anaerobic Thresholds

Analysis of VO2max and the anaerobic threshold will be measured.

Time frame:
Baseline, 12 week

No measurements were reported for this outcome.

Adverse events

Collected over 16 weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Nicotinamide Riboside in Patients With Stable Heart Failure0/1 (0%)0/1 (0%)0/1 (0%)

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Nicotinamide Riboside in Patients With Stable Heart Failure
<=18 years0
Between 18 and 65 years1
>=65 years0
Sex: Female, Male
Sex: Female, Male(Participants)Nicotinamide Riboside in Patients With Stable Heart Failure
Female1
Male0
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Nicotinamide Riboside in Patients With Stable Heart Failure
Hispanic or Latino0
Not Hispanic or Latino1
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Nicotinamide Riboside in Patients With Stable Heart Failure
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American1
White0
More than one race0
Unknown or Not Reported0
08

Study locations

2 sites
  • Walter Reed National Military Medical Center
    Bethesda, Maryland 20889, United States
  • National Institutes of Health Clinical Center
    Bethesda, Maryland 20892, United States
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Feb 15, 2019

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

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 28, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03565328
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Responsible party
Sponsor
First posted
Jun 21, 2018
Start date
Sep 27, 2018
Primary completion
Nov 18, 2019
Completion
Nov 18, 2019
Results posted
Oct 28, 2020
Last update
Oct 28, 2020

Study contacts

Michael N Sack, M.D.
principal investigator · National Heart, Lung, and Blood Institute (NHLBI)

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Nov 2019. You cannot join it, but the record below documents what was studied.

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