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Milrinone for Prevention of Post-ligation Cardiac Syndrome Trial

Phase 3
Recruiting
Conditions
Post-ligation Cardiac Syndrome
Interventions
Drug: Placebo infusion
Registration Number
NCT06679855
Lead Sponsor
NICHD Neonatal Research Network
Brief Summary

The goal of this Phase 3, randomized, masked clinical trial is to is to find out whether milrinone, when given to infants after PDA closure, will help the heart work better by supplying oxygen to the lungs and tissues.

The main questions it aims to answer are:

1. to determine if milrinone decreases the risk of death or PLCS within 7 days of the procedure, compared to standard treatment; and

2. to determine the effects of milrinone on two-year survival and neurodevelopmental outcome.

Detailed Description

Researchers will compare milrinone to a placebo saline solution to see if it helps the heart work better by supplying oxygen to the lungs and tissues.

After randomization the following will happen in both the control and treatment groups:

* Infant will start receiving either a low dose (0.33 μg/kg/min) of milrinone in the treatment group, OR saline solution in the control group through an intravenous (IV) line. The study drug will be started within 2 hours of the PDA closure procedure.

* For the first 2 to 4 hours, the study team will monitor very closely to see if there are any major side effects from the study drug.

* If the infant continues to have high blood pressure, which places an added stress on the heart, the dose will go up to 0.66 μg/kg/min. One more increase will be allowed to 0.75 μg/kg/min. The study drug will then stay at this dose. No more changes will happen to the dose. If there are side effects, then the study drug will be stopped and will not be started again.

* The study drug will be stopped after 24 hours if the infant only required the lowest drug and remains well. If the required higher doses of milrinone, it will take longer to wean them off the medication. Some infants may receive milrinone for 3-5 days. If the infant's heart starts to work better by using less oxygen from the breathing machine (ventilator), the study drug may be stopped before 3 days.

* Information will be collected from the infant's medical record including demographic information, gestational age, blood pressures, heart rates, respiratory rates, information about his or her breathing, medications, details of medical treatment for PDA, medical procedures including echocardiograms (ultrasound of the baby's heart), details of PDA closure (by surgery or transcatheter closure), head ultrasounds, and diagnoses. The investigators will also be collecting information from the maternal medical record including ultrasounds that may be relevant to the infant's course.

* Right after the study drug is stopped (which could be 5 days or less) the doctor may decide to give the infant the drug milrinone. This is a decision that will be made by the infant's doctor.

* After the infant goes home from the hospital, they will be scheduled for follow-up exams in the clinic. The follow-up visit will be done when the infant is about 2 years old. The visit will take about two hours to do. During the follow-up visit the investigators will test the infant's movement, behavior, and development. The investigators will also check the infant to make sure they do not have high blood pressure or any evidence of kidney disease.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
316
Inclusion Criteria
  • Gestational age at birth ≤27 weeks (and 6 days) and postnatal age < 3 months at intervention
  • Invasive or non-invasive positive pressure respiratory support (does not include low flow nasal cannula)
  • Hemodynamically significant PDA with minimum transductal diameter ≥1.0 mm within 2 days of intervention
  • Decision by clinical team to proceed with PDA closure via surgical ligation or percutaneous cardiac catheterization based on clinical and echocardiography features of hemodynamic significance.
Exclusion Criteria
  • Any major congenital malformation
  • Congenital heart disease (except small (≤1mm) muscular ventricular septal defects, or small/moderate (<3mm) atrial septal defect)
  • Acute renal failure defined by urine output < 0.5 mL/kg/hour OR rise of serum creatinine by 0.3 mg/dL within 48 hours OR rise of serum creatinine more than 40% above baseline serum creatinine within prior 72 hours.
  • Systemic administration of vasodilator/inodilator agents
  • Prior history of arrhythmia

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlacebo infusionAn iv infusion of placebo (0.9% saline) of equivalent volume will be administered. The infusion will be accompanied by an iv bolus of 10 mL/kg of 0.9% NaCl (administered over 60 minutes) to ensure blinding is maintained.
MilrinoneMilrinone infusionAn intravenous (iv) infusion of milrinone will be administered at an initial dose of 0.33 mcg/kg/min and accompanied by an iv bolus of 10 mL/kg of 0.9% NaCl (administered over 60 minutes).
Primary Outcome Measures
NameTimeMethod
Post-ligation cardiac syndrome (PLCS) or death within 7 days of PDA closureOnset within 48 hours but may last up to 7 days

Composite outcome of post-ligation cardiac syndrome (PLCS) or death within 7 days of PDA closure. Onset within 48 hours but may last up to 7 days.

Secondary Outcome Measures
NameTimeMethod
Severe vision impairment22 to 26 months

Severe vision impairment

Severe hearing impairment22 to 26 months

Severe hearing impairment

Bayley-4 cognitive, language, motor scores22 to 26 months

Bayley-4 cognitive, language, motor scores

Gross Motor Function level ≥II22 to 26 months

Gross Motor Function level ≥II

CBCL Internalizing, Externalizing, and Total Problems aggregate T scores of >64 (clinical range) and 60-63 (borderline range).22 to 26 months

CBCL Internalizing, Externalizing, and Total Problems aggregate T scores of \>64 (clinical range) and 60-63 (borderline range).

Death before 22-26-month follow-up22 to 26 months

Death before 22-26-month follow-up

Height, weight, or head circumference growth failure22 to 26 months

Height, weight, or head circumference growth failure (decline by \>2 z-scores since discharge)

Moderate or severe cerebral palsy22 to 26 months

Moderate or severe cerebral palsy

Systemic hypotensionOnset within 48 hours but may last up to 7 days

Either systolic blood pressure below the 3rd percentile for gestational age or mean blood pressure below the postmenstrual age equivalent

Systemic hypertensionOnset within 48 hours but may last up to 7 days

Systolic blood pressure above the 97th percentile

Oxygenation failureOnset within 48 hours but may last up to 7 days

An absolute increase of at least 20% in the fraction of inspired oxygen or mean airway pressure compared with the one-hour post-intervention value that persists for a minimum of 1 hour and occurs within 72 hours of PDA closure

Ventilation failureWithin 72 hours of PDA closure

Need for high frequency oscillatory ventilation when conventional ventilation strategies fail or a 20% rise in amplitude compared with the one-hour post-intervention value that persists for a minimum of 1 hour and occurs within 72 hours of PDA closure

Vasopressor scoreWithin 7 days of PDA closure

Vasopressor score. Minimum score is zero. Higher score is worse, meaning need for higher level of support.

VISmax will be calculated as follows: (VIS=dopamine dose \[μg kg-1 min-1\]+dobutamine \[μg kg-1 min-1\]+100×epinephrine dose \[μg kg-1 min-1\]+50×levosimendan dose \[μg kg-1 min-1\]+10×milrinone dose \[μg kg-1 min-1\]+10 000×vasopressin \[units kg-1 min-1\]+100×norepinephrine dose \[μg kg-1 min-1\]) using the maximum dosing rates of vasoactive and inotropic medications (μg kg-1 min-1 or IU kg-1 min-1)

Use of open-label milrinone or systemic vasodilator after cessation of study drug administrationAfter study drug cessation and within 7 days of PDA closure

Number of participants using open-label milrinone or systemic vasodilator after cessation of study drug administration

Time to successful extubation36 weeks postmenstrual age

Time to successful extubation, which is defined as extubation for at least 7 days

Post-intervention Moderate-severe bronchopulmonary dysplasia at 36 weeks' gestation36 weeks postmenstrual age

Need for at least 2 liters of high flow nasal cannula at 36 weeks postmenstrual age

Post-intervention Chronic Pulmonary hypertension36 weeks postmenstrual age

presence of septal flattening (or eccentricity index \> 1.3, right ventricular systolic pressure greater than 40 mmHg, or exclusive right to left atrial level shunt on 36-week echocardiography assessment. The presence of a large atrial septal defect, pulmonary vein stenosis or left ventricular diastolic dysfunction will be recorded.

Post-intervention Periventricular Leukomalacia (PVL)36 weeks postmenstrual age

Presence of cystic white matter changes on cranial ultrasound

Post-intervention Necrotizing Enterocolitis36 weeks postmenstrual age

At least Bells stage IIb disease

Post-intervention Retinopathy of Prematurity (ROP) ≥ stage 3 (according to the international classification)36 weeks postmenstrual age

Post-intervention Retinopathy of Prematurity (ROP) ≥ stage 3 (according to the international classification)

Weight or head circumference z-score change at 36 weeks36 weeks postmenstrual age

Weight or head circumference z-score change at 36 weeks (decline by two z-scores will be considered growth failure)

Death between randomization and discharge from NICURandomization to 120 days' postnatal age, death, discharge, or transfer outside of the Study Center, whichever occurs first (an average of 112 days postnatal age)

Death between randomization and discharge from NICU

Moderate-Severe neurodevelopmental impairment (NDI), using current NRN Follow-Up Study definition22 to 26 months

Moderate-Severe neurodevelopmental impairment (NDI), using current NRN Follow-Up Study definition

Moderate-Severe neurodevelopmental impairment (NDI) or death22 to 26 months

Moderate-Severe neurodevelopmental impairment (NDI) or death

Trial Locations

Locations (19)

University of Alabama - Birmingham

🇺🇸

Birmingham, Alabama, United States

Children's Hospital of Orange County

🇺🇸

Orange, California, United States

Stanford University

🇺🇸

Palo Alto, California, United States

Sharp Mary Birch Hospital for Women & Newborns

🇺🇸

San Diego, California, United States

Emory University

🇺🇸

Atlanta, Georgia, United States

Northwestern Lurie Children's Hospital of Chicago

🇺🇸

Chicago, Illinois, United States

University of Iowa

🇺🇸

Iowa City, Iowa, United States

Boston Children's Hospital

🇺🇸

Boston, Massachusetts, United States

University of Mississippi Medical Center

🇺🇸

Jackson, Mississippi, United States

University of New Mexico

🇺🇸

Albuquerque, New Mexico, United States

Duke University

🇺🇸

Durham, North Carolina, United States

Cincinnati Children's Hospital Medical Center

🇺🇸

Cincinnati, Ohio, United States

Case Western Reserve University

🇺🇸

Cleveland, Ohio, United States

University of Oklahoma Health Sciences

🇺🇸

Oklahoma City, Oklahoma, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

Le Bonheur Children's Hospital

🇺🇸

Memphis, Tennessee, United States

University of Texas Southwestern

🇺🇸

Dallas, Texas, United States

University of Texas at Houston

🇺🇸

Houston, Texas, United States

University of Utah

🇺🇸

Salt Lake City, Utah, United States

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