PEDIatric Cardiac Dysfunction Treated With Injectable Nicotinamide Adenine Dinucleotide

NCT07839130 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: [2026]-Y-110-D
• Study Type: interventional
• Target: 180
• Locations: 1 country
• Sites: 1

Brief Summary

Brief Summary This study is a multicenter, randomized, double-blind, placebo-controlled trial designed to evaluate whether an injectable form of Coenzyme I (also known as nicotinamide adenine dinucleotide, or NAD⁺) is safe and effective for treating children with cardiac dysfunction (weak heart pumping). Who can join? We plan to enroll 180 children under 18 years of age who are hospitalized with cardiac dysfunction, meaning their heart's pumping ability (left ventricular ejection fraction, or LVEF) is below 55%. Children will be recruited from five hospitals in China. Children with certain serious conditions, such as end-stage heart failure, severe liver or kidney problems, known allergy to the study drug, or active participation in another drug trial, will not be eligible. What will participants receive? All children will receive standard heart failure care as recommended by current guidelines. In addition, they will be randomly assigned (like drawing lots) to one of three groups: Placebo group - receives standard care plus a saltwater (normal saline) infusion that looks like the study drug but contains no active medicine (60 children). Low-dose group - receives standard care plus Coenzyme I injection at 2 mg per kg of body weight per day (maximum 100 mg) (60 children). High-dose group - receives standard care plus Coenzyme I injection at 4 mg per kg of body weight per day (maximum 200 mg) (60 children). The study drug or placebo is given as a slow intravenous (IV) infusion once daily for 7 days. Each infusion takes at least 2 hours. What is the main goal? The main question is whether adding Coenzyme I improves heart function more than standard care alone. The primary endpoint is the change in LVEF from baseline to 4 weeks after treatment, measured by echocardiography. We will also assess NT-proBNP, a blood test that reflects the strain on the heart, at several time points. What else will we look at? We will also evaluate quality of life (using a child-friendly questionnaire), heart function grade (NYHA/ROSS), exercise capacity (6-minute walk test), growth and development, cardiac structure and remodeling (by echocardiography), global longitudinal strain (GLS), and clinical events such as heart failure-related rehospitalization, emergency interventions, and death. We will carefully monitor for any side effects or safety concerns throughout the study. Why is this important? Children with cardiac dysfunction often do not respond well enough to current treatments, and there are few specific therapies designed for them. Coenzyme I (NAD⁺) is a natural substance in the body that helps heart cells produce energy. Studies in adults and in laboratory models suggest it may protect the heart and improve its function. This trial is the first large-scale pediatric study to test whether it can help children with heart pumping problems. How long will participation last? Each child will be in the study for about 48 weeks - 7 days of treatment in the hospital, followed by follow-up visits at 4 weeks, 12 weeks, and 24 weeks after treatment, and a final telephone or clinic visit at 48 weeks to collect clinical event data. Safety and oversight An independent Data and Safety Monitoring Board (DSMB) will regularly review all safety information. The study has been approved by the ethics committees of all participating hospitals and is conducted in accordance with the Declaration of Helsinki and Chinese Good Clinical Practice guidelines. All enrolled children are covered by clinical trial liability insurance.

Conditions

Cardiac Dysfunction

Keywords

Pediatric cardiac dysfunction; Nicotinamide adenine dinucleotide (NAD⁺); Randomized controlled trial

Outcome Measures

Primary Outcomes (1)

• Change in Left Ventricular Ejection Fraction (LVEF) from Baseline to 4 Weeks

  Change in LVEF from baseline to 4 weeks after treatment, assessed by echocardiography as a continuous variable. The primary analysis compares combined NAD⁺ groups (low-dose and high-dose) versus placebo using a one-sided difference test (δ₀ = 0) with ANCOVA, adjusting for baseline LVEF and center as a random effect (one-sided α = 0.025). Least-squares mean difference and 95% confidence interval will be estimated.

  Baseline to 4 weeks after treatment.

Secondary Outcomes (12)

• Change in N-terminal pro-B-type Natriuretic Peptide (NT-proBNP) from Baseline

  Baseline, 4 weeks, 12 weeks, and 24 weeks after treatment.

• Change in LVEF from Baseline at Multiple Time Points

  Baseline, 4 weeks, 12 weeks, and 24 weeks after treatment.

• Change in Left Ventricular End-Diastolic Diameter (LVEDD) from Baseline

  Baseline and 24 weeks after treatment.

• Change in Left Ventricular End-Systolic Diameter (LVESD) from Baseline

  Baseline and 24 weeks after treatment.

• Change in Left Ventricular Wall Thickness from Baseline

  Baseline and 24 weeks after treatment.

Other Outcomes (4)

• Incidence of Adverse Events (AEs)

  From baseline through 48 weeks after treatment.

• Incidence of Serious Adverse Events (SAEs)

  From baseline through 48 weeks after treatment.

• Exploratory Comparison of LVEF Change Between Low-Dose and High-Dose NAD⁺

  Baseline, 4 weeks, 12 weeks, and 24 weeks after treatment.

Study Arms (3)

Placebo Group

PLACEBO COMPARATOR

Participants receive standard guideline-directed care for cardiac dysfunction (including diuretics, ACEIs/ARBs/ARNIs, β-blockers, and mineralocorticoid receptor antagonists, adjusted according to clinical status) plus an intravenous placebo (normal saline) administered once daily for 7 consecutive days. Each infusion is delivered over a minimum of 2 hours. The maximum volume is 125 mL per infusion.

Other: Placebo

Low-Dose NAD⁺ Group

EXPERIMENTAL

Participants receive standard guideline-directed care for cardiac dysfunction (including diuretics, ACEIs/ARBs/ARNIs, β-blockers, and mineralocorticoid receptor antagonists, adjusted according to clinical status) plus intravenous Coenzyme I (nicotinamide adenine dinucleotide, NAD⁺) administered once daily for 7 consecutive days. The dose is 2 mg/kg of body weight per day, with a maximum of 100 mg per day. The drug is diluted with normal saline to a concentration of 1.6 mg/mL. Each infusion is delivered over a minimum of 2 hours.

Drug: Nicotinamide Adenine Dinucleotide

High-Dose NAD⁺ Group

EXPERIMENTAL

Participants receive standard guideline-directed care for cardiac dysfunction (including diuretics, ACEIs/ARBs/ARNIs, β-blockers, and mineralocorticoid receptor antagonists, adjusted according to clinical status) plus intravenous Coenzyme I (nicotinamide adenine dinucleotide, NAD⁺) administered once daily for 7 consecutive days. The dose is 4 mg/kg of body weight per day, with a maximum of 200 mg per day. The drug is diluted with normal saline to a concentration of 1.6 mg/mL. Each infusion is delivered over a minimum of 2 hours.

Drug: Nicotinamide Adenine Dinucleotide

Interventions

Placebo OTHER

Intravenous normal saline (0.9% sodium chloride), matching appearance and volume to the active drug. Administered once daily for 7 consecutive days. Each infusion is delivered over a minimum of 2 hours. Maximum volume is 125 mL per infusion.

Also known as: Normal saline, 0.9% sodium chloride

Placebo Group

Nicotinamide Adenine Dinucleotide DRUG

Powder for injection, supplied as 5 mg/vial. Administered intravenously once daily for 7 consecutive days at a dose of either 2 mg/kg/day (maximum 100 mg) or 4 mg/kg/day (maximum 200 mg), diluted with normal saline to a concentration of 1.6 mg/mL. Each infusion is delivered over a minimum of 2 hours.

Also known as: Coenzyme I, NAD⁺

High-Dose NAD⁺ Group; Low-Dose NAD⁺ Group

Eligibility Criteria

• Age: 0 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age \< 18 years.
• Hospitalized children diagnosed with cardiac dysfunction (LVEF \< 55%).
• Voluntary participation; written informed consent signed and dated by the legal guardian before enrollment in accordance with Good Clinical Practice (GCP) and local laws; assent from the age-appropriate child, if applicable.

You may not qualify if:

• Concomitant malignant arrhythmia; ischemic cardiomyopathy due to congenital or acquired coronary artery disease; cardiac dysfunction caused by systemic diseases (e.g., systemic lupus erythematosus, thyroid dysfunction).
• End-stage/refractory heart failure: stage D heart failure with no improvement or worsening of symptoms and signs after adequate and standardized anti-heart-failure therapy (including but not limited to continuous intravenous infusion of positive inotropic agents such as dopamine, dobutamine, milrinone, or vasoactive agents).
• Planned or prior advanced cardiac therapy: prior heart transplantation, or planned heart transplantation within 3 months after randomization, or implantation of a ventricular assist device (VAD, including LVAD/RVAD/BiVAD).
• Known allergy to Coenzyme I; lactose intolerance or lactose allergy.
• Severe hepatic or renal insufficiency: ALT or AST \> 5× upper limit of normal, or estimated glomerular filtration rate ≤ 30 mL/min/1.73 m².
• History of severe allergy or infusion reaction.
• Concurrent tumors or cardiac dysfunction caused by chemotherapy/radiotherapy.
• Current participation in another drug clinical trial.
• Any other condition deemed unsuitable for this clinical trial by the investigator.

Sponsors & Collaborators

• Beijing Children's Hospital: lead

Study Sites (1)

Beijing Children's Hospital, Capital Medical University

Beijing, Beijing Municipality, 100045, China

Location

MeSH Terms

Interventions

Saline Solution; Sodium Chloride; NAD

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds; Adenine Nucleotides; Purine Nucleotides; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Coenzymes; Enzymes and Coenzymes; Nucleotides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleotides

Central Study Contacts

Jinsong Jiang, Dr

CONTACT

86+19511430078; jiang_js2023@foxmail.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This multicenter, randomized, double-blind, placebo-controlled trial will enroll 180 children (60/arm): placebo, low-dose NAD⁺ (2 mg/kg/day, max 100 mg), and high-dose NAD⁺ (4 mg/kg/day, max 200 mg). Randomization is center-stratified block randomization (block size 6; 2/arm/block), yielding 2:1 combined NAD⁺ versus placebo. Participants, outcome and adverse-event assessors are blinded; unblinded staff do not administer drugs. All receive standard care. Treatment: IV once daily for 7 days; follow-up to 48 weeks. Primary endpoint: change in LVEF from baseline to 4 weeks. A one-sided difference test (δ₀=0) compares combined NAD⁺ groups versus placebo using ANCOVA (baseline LVEF covariate; center random effect; one-sided α=0.025). Exploratory: low- versus high-dose LVEF/NT-proBNP changes.

Study Record Dates

• First Submitted: September 18, 2026
• First Posted: September 24, 2026
• Study Start: October 1, 2026
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): December 1, 2028
• Last Updated: September 24, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)