Nitric Oxide for Congenital Heart Defects

Overseen By Alistair Hilton, BA

Age: < 18

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Vanderbilt University Medical Center

Disqualifiers: Emergency surgery, Severe developmental delay, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether nitric oxide can protect the brain and kidneys during heart surgery for babies under one year old with congenital heart defects. Researchers aim to determine if nitric oxide reduces brain stress and kidney damage post-surgery, potentially affecting ICU recovery time. The trial compares two groups: one receiving standard care and the other receiving nitric oxide during surgery. Babies with heart defects scheduled for corrective surgery, who do not require inhaled nitric oxide immediately before surgery, might be suitable candidates for this study. As a Phase 2, Phase 3 trial, this study evaluates the treatment's effectiveness in an initial group and represents the final step before FDA approval, offering a chance to contribute to significant medical advancements.

Will I have to stop taking my current medications?

The trial information does not specify whether participants need to stop taking their current medications.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that nitric oxide is generally safe for people with congenital heart conditions. One study found that using nitric oxide in adults with these heart conditions and high blood pressure in the lungs after surgery was safe and effective. However, another study found that nitric oxide did not help children recover faster after heart surgery.

These results suggest that while nitric oxide is usually well-tolerated, it may not always accelerate recovery. Participants should consider this when deciding to join the trial. In terms of safety, nitric oxide has proven to be a dependable option in similar treatments. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for congenital heart defects?

Unlike the standard treatment options for congenital heart defects, which typically involve surgery and cardiopulmonary bypass procedures, the use of Nitric Oxide (NO) offers a novel approach. Nitric Oxide is unique because it acts as a vasodilator, helping to relax and widen blood vessels, which may improve blood flow during the critical phases of surgery. Researchers are excited about this treatment because it could potentially reduce complications and improve recovery times for patients undergoing heart surgery, thanks to its ability to better manage blood circulation during procedures. This represents a significant advancement, offering hope for safer and more effective surgical outcomes.

What evidence suggests that nitric oxide might be an effective treatment for congenital heart defects?

Studies have shown that nitric oxide can benefit patients with congenital heart defects by reducing heart and lung injuries during heart surgery. Research also indicates that nitric oxide can lower troponin levels, a marker of heart damage, after surgery. In this trial, participants in the nitric oxide group will receive Nitric Oxide (NO) during cardiopulmonary bypass, potentially protecting the heart and lungs and easing recovery. Meanwhile, the standard of care group will undergo the standard cardiopulmonary bypass procedure without nitric oxide. ^{4 6 7 8 9}

Who Is on the Research Team?

David P Bichell, MD

Principal Investigator

Vanderbilt University Medical Center

Are You a Good Fit for This Trial?

This trial is for babies under 1 year old with congenital heart defects (CHD) who are scheduled for corrective surgery using cardiopulmonary bypass. It's not open to those needing emergency surgery, with severe developmental delays, pre-existing kidney disease, or a need for nitric oxide before surgery.

Inclusion Criteria

I am under 1 year old.

My baby is having heart surgery with a heart-lung machine.

Exclusion Criteria

I need inhaled nitric oxide right before surgery.

You have a severe developmental delay, as determined by a specific scale that measures cognitive abilities before a serious illness.

I require emergency surgery.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants undergo cardiopulmonary bypass with or without nitric oxide administration

1 day

1 visit (in-person)

ICU Monitoring

Participants are monitored in the ICU for delirium and organ dysfunction

14 days

Daily monitoring in ICU

Follow-up

Participants are monitored for safety and effectiveness after treatment

30 days

What Are the Treatments Tested in This Trial?

Interventions

• Nitric Oxide (NO) 20 part per million (ppm)

Trial Overview The study tests if inhaling low doses of Nitric Oxide (NO) can protect the brain and kidneys during heart surgery. Researchers will measure specific proteins and monitor patients' recovery in the ICU to see if NO makes a difference.

How Is the Trial Designed?

Group I: Nitric oxide group Experimental Treatment 1 Intervention

Participants in this group will receive Nitric Oxide (NO) while undergoing Cardiopulmonary bypass (CPB)

Group II: Standard of care cardiopulmonary bypass procedure Active Control 1 Intervention

Participants in this group will receive standard of care

Who Is Running the Clinical Trial?

Vanderbilt University Medical Center

Lead Sponsor

Trials

922

Recruited

939,000+

Published Research Related to This Trial

Inhaled nitric oxide is an effective treatment for pulmonary hypertension in patients with congenital heart disease, particularly benefiting newborns, who made up 37% of the 400 patients treated at the center.

While it helps in diagnosing and managing conditions like total anomalous pulmonary venous connection and congenital mitral stenosis, the efficacy of inhaled nitric oxide has not been rigorously tested in randomized, placebo-controlled trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Inhaled nitric oxide in the neonate with cardiac disease. Atz, AM., Wessel, DL.[2022]

Citations

1. sciencedirect.com sciencedirect.com/science/article/pii/S1441277223002892

Statistical analysis plan for the NITric oxide during ... Statistical analysis plan for the NITric oxide during cardiopulmonary bypass to improve Recovery in Infants with Congenital heart defects (NITRIC) trial.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9237803/

Effect of Nitric Oxide via Cardiopulmonary Bypass on ... Studies have suggested that nitric oxide added to the gas inflow of the cardiopulmonary bypass oxygenator may decrease postoperative troponin ...

3. cardiothoracicsurgery.biomedcentral.com cardiothoracicsurgery.biomedcentral.com/articles/10.1186/s13019-024-02953-y

Impact of nitric oxide via cardiopulmonary bypass on pediatric ... In the past decade, numerous studies had reported that adding NO to the CPB circle can reduce heart and lung injuries [29, 30]. Additionally, it ...

4. withpower.com withpower.com/trial/phase-3-heart-diseases-5-2021-77924

Nitric Oxide for Congenital Heart Disease Inhaled nitric oxide is an effective treatment for pulmonary hypertension in patients with congenital heart disease, particularly benefiting newborns, who made ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT03946462

Nitric Oxide During CPB to Reduce AKI in Neonates Gestational age: 38 weeks; Diagnosis: Congenital Heart Disease (CHD); Planned surgery to include cardiopulmonary bypass (CPB )for underlying CHD; Consent of ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10692519/

Statistical analysis plan for the NITric oxide during ... Statistical analysis plan for the NITric oxide during cardiopulmonary bypass to improve Recovery in Infants with Congenital heart defects (NITRIC) trial.

7. bmccardiovascdisord.biomedcentral.com bmccardiovascdisord.biomedcentral.com/articles/10.1186/s12872-025-04967-3

Innovative nitric oxide delivery technology for postoperative ... This device has proven to be safe and effective for use in adult patients with congenital heart disease and pulmonary hypertension post-surgery.

8. innovationdistrict.childrensnational.org innovationdistrict.childrensnational.org/jama-study-shows-no-benefit-to-nitric-oxide-in-cardiopulmonary-bypass-for-young-children/

JAMA study shows no benefit to nitric oxide in ... An international clinical trial showed that nitric oxide doesn't help kids recover faster from cardiac surgery with cardiopulmonary bypass.

9. sciencedirect.com sciencedirect.com/science/article/pii/S1053077025007323

Efficacy and Safety of Pulmonary Vasodilator and Inodilator ... Vasodilators and inodilators did not significantly reduce perioperative mortality in paediatric CHD. Some agents, such as intravenous sildenafil ...