PEDIatric Cardiac Dysfunction Treated With Injectable Nicotinamide Adenine Dinucleotide

Status

Not yet recruiting

Phase

Phase 2

Enrollment

180 participants (estimated)

Sponsor

Beijing Children's Hospital · Other

Design

Randomized · Quadruple masking

Countries

1 country

China

Start

1 Oct 2026 (estimated)

Primary completion

1 Apr 2028 (estimated)

Completion

1 Dec 2028 (estimated)

First posted

24 Sept 2026

Last update posted

24 Sept 2026

Conditions

Cardiac Dysfunction

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.

• Placebo comparator

  Placebo Group

  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.

• Experimental

  Low-Dose NAD⁺ Group

  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.

• Experimental

  High-Dose NAD⁺ Group

  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.

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

   Time frame · Baseline to 4 weeks after treatment.

   Registry description

   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.

No study cites this trial.