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Trial to Shorten Pharmacologic Treatment of Newborns With Neonatal Opioid Withdrawal Syndrome (NOWS)

Phase 3
Active, not recruiting
Conditions
Neonatal Opioid Withdrawal Syndrome
Interventions
Registration Number
NCT04214834
Lead Sponsor
Advancing Clinical Trials in Neonatal Opioid Withdrawal (ACT NOW) Program
Brief Summary

The objective of this study is to evaluate the efficacy of a rapid wean intervention compared with a slow-wean intervention in reducing the number of days of opioid treatment from the first dose of weaning to cessation of opioid among infants receiving an opioid (defined as morphine or methadone) as the primary treatment for neonatal opioid withdrawal syndrome (NOWS).

Detailed Description

This will be a pragmatic, randomized, blinded trial comparing a rapid-wean intervention (15% decrements from the stabilization dose) to a slow-wean intervention (10% decrements from the stabilization dose) to determine whether rapid weaning will reduce the number of treatment days among infants receiving morphine or methadone orally as the primary treatment for NOWS. Participating hospitals must provide pharmacologic treatment to at least an average of 12 opioid exposed infants each year, use a scoring system to assess for signs of NOWS (original or modified Finnegan Neonatal Abstinence Scoring system, Eat-Sleep or Console), and provide opioid replacement therapy with either morphine or methadone as the primary drug for treating NOWS. Hospitals may change use of these two opioids during the trial period. The investigators will stratify randomization by hospital.

The study protocol will commence after NOWS signs have been controlled with an opioid (stabilization) and weaning of pharmacologic treatment is to be started. At or before each 24-hour interval, clinical team members will evaluate and score infants, per hospital practice, for signs of NOWS to determine if the infant will tolerate weaning of the study drug. After study drug cessation, the clinical team will observe infants in the hospital for at least 48 hours prior to discharge, which is similar to clinical practice. A trained examiner will administer the Neonatal Intensive Care Unit (NICU) Network Neurobehavioral Scale (NNNS) to assess neurobehavioral profiles after infants cease study drug and prior to discharge.

At one month post discharge, primary caregivers will complete the Brief Symptom Inventory (BSI), the Maternal Postnatal Attachment Questionnaire (MPAQ) and a caregiver questionnaire. The site research team will contact the primary caregiver(s) to update contact information and/or complete questionnaires when the infant is 6 months, 12 months, 18 months, and 24 months of age. The questionnaires will assess infant wellness, neurobehavioral functioning and development, postnatal attachment and bonding, and caregiver well-being. At 24 months, the infants will be seen during which a, certified developmental specialists, blinded to the intervention, will administer the Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) to assess infant neurodevelopment. The BSI and the Brief Infant Toddler Social Emotional Assessment (BITSEA) will also be administered during the 24 month visit along with measures of growth.

Recruitment & Eligibility

Status
ACTIVE_NOT_RECRUITING
Sex
All
Target Recruitment
190
Inclusion Criteria
  • Hospital Level

    1. Hospital provides pharmacologic treatment to at least an average of 12 opioid exposed infants each year
    2. Hospital uses a scoring system to assess for signs of NOWS (original or modified Finnegan Neonatal Abstinence Scoring system, Eat-Sleep or Console)
    3. Hospital provides opioid replacement therapy with either morphine or methadone as part of pharmacologic treatment of NOWS
  • Infant Level

    1. Gestational age ≥ 36 weeks
    2. Receiving scheduled pharmacological therapy with morphine or methadone as the primary drug treatment for NOWS secondary to maternal opioid use
    3. Tolerating enteral feeds and medications by mouth
Read More
Exclusion Criteria
  • Hospital Level

    1. Hospitals discharge > 10% of infants from the hospital on opioid replacement therapy on average per year
  • Infant Level

    1. Major birth defect (e.g. gastroschisis)
    2. Any major surgery (minor surgery [e.g., circumcision, digit ligation, frenulectomy] is not an exclusion criterion)
    3. Hypoxic-ischemic encephalopathy
    4. Seizures from etiologies other than NOWS
    5. Treatment with opioids for reasons other than NOWS
    6. Respiratory support (nasal cannula or greater) for > 72 hours
    7. Planned discharge from the hospital on opioids
    8. Use of other opioids (e.g., buprenorphine) as primary drugs for treatment of NOWS
    9. Weaning of morphine or methadone as the primary treatment of NOWS has started
Read More

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Rapid-weanMethadone15% decrements from the stabilization dose of morphine/methadone
Slow-weanMorphine10% decrements from the stabilization dose of morphine/methadone
Rapid-weanMorphine15% decrements from the stabilization dose of morphine/methadone
Slow-weanMethadone10% decrements from the stabilization dose of morphine/methadone
Primary Outcome Measures
NameTimeMethod
Number of days of opioid treatmentFrom date of birth until hospital discharge or 1 year whichever comes first

The number of days of opioid treatment (used as primary treatment), including escalation, resumption, and spot treatment, from the first weaning dose to cessation of opioid.

Secondary Outcome Measures
NameTimeMethod
Number of days of morphine treatmentFrom date of birth until hospital discharge or 1 year whichever comes first

The numbers of days of opioid treatment from the first weaning dose to cessation of opioid with a rapid and slow-wean interventions among infants treated with morphine.

Proportion of infants in each intervention arm with an atypical NNNS neurobehavioral profileFrom date of birth until hospital discharge or 1 year whichever comes first

The proportion of infants in each intervention arm with an atypical NNNS neurobehavioral profile prior to discharge.

Number of days of methadone treatmentFrom date of birth until hospital discharge or 1 year whichever comes first

The numbers of days of opioid treatment from the first weaning dose to cessation of opioid with a rapid and slow-wean interventions among infants treated with methadone.

Proportion of infants who have an escalation or resumption of opioid medication during weaningFrom date of birth until hospital discharge or 1 year whichever comes first

The proportions of infants in the rapid and slow-wean intervention arms who have an escalation or resumption of opioid medication during weaning.

Total amounts of opioid from the first weaning dose to cessation of opioidFrom date of birth until hospital discharge or 1 year whichever comes first

The total amounts of opioid from the first weaning dose to cessation of opioid among infants in the rapid and slow-wean intervention arms.

proportion of infants in each intervention arm with safety outcomes of seizures (clinical or EEG), excessive stool output, respiratory disturbances, and feeding toleranceFrom date of birth until hospital discharge or 1 year whichever comes first

The proportion of infants in each intervention arm with safety outcomes of seizures (clinical or EEG), excessive stool output, respiratory disturbances, and feeding tolerance.

Length of hospital stayFrom date of birth until hospital discharge or 1 year whichever comes first

The lengths of hospital stay for infants in each intervention arm.

proportion of infants who experience initiation and/or escalation of second-line or third-line drugs to treat NOWSFrom date of birth until hospital discharge or 1 year whichever comes first

The proportion of infants who experience initiation and/or escalation of second-line or third-line drugs to treat NOWS signs from the first weaning dose to cessation of opioid in the rapid-wean and slow-wean intervention arms.

Maternal Well-being1 month Post Discharge and 24 months of age

The Brief Symptom Inventory (BSI) will be the measurement tool used to asses maternal well-being in each intervention arm. A regression model will be used to analyze BSI total scores. This model will include a fixed treatment effect (intervention arms), an adjustment for the stratifying variable, and fixed effects for the covariates of maternal treatment and stabilization dose. The BSI outcomes are measured at 1-month after discharge and 24-months of age. The models for BSI outcomes will include the 1-month after discharge BSI outcome as a covariate. The F-test of the intervention arm effect will be the primary test of interest. The intervention arm difference will be reported along with 95% CI. Analyses will be conducted among the entire group and among those where the biologic mother is the primary caretaker.

Maternal-Infant attachment1 month post discharge

The Maternal Postnatal Attachment Questionnaire (MPAQ) will be the measurement tool used to asses maternal-infant attachment in each intervention arm. A regression model will be used to analyze MPAQ total scores. This model will include a fixed treatment effect (intervention arms), an adjustment for the stratifying variable, and fixed effects for the covariates of maternal treatment and stabilization dose. The intervention arm difference will be reported along with 95% CI. Analyses will be conducted among the entire group and among those where the biologic mother is the primary caretaker.

Infant growthBirth through 24 months of age

Anthropometric outcomes will be measured at birth, time of discharge, and 24 months. We will calculate anthropometric z-scores at each of the three assessment periods for the purpose of analysis based on age and gender specific WHO norms.We will provide the mean and SD of infants' weights (z-scores) separately for each treatment group. We will use a mixed linear model to evaluate the effect of treatment arm on weight (z-scores). We will examine the impact of the treatment arm on length, HC, and infant weight for length (z-scores). We will provide the mean and SD of infant BMI-z at 24-months for each treatment group. To compare Bayley-IV scores between intervention arms, we will perform a linear mixed-effects model with a fixed effect for the intervention group and a random effect for study site. We will report point estimates for the group mean difference along with a 95% CI, and the team will repeat this analytical approach for each of the Bayley-IV domains.

Infant wellnessBirth through 24 months of age

We will analyze the caregiver questionnaire outcomes and the death outcome using a longitudinal GLMM or GEE model appropriate for the outcome type since the data will be collected at multiple time points after discharge. Count data that tend to have more than 0 or 1 events counted will be analyzed using a Poisson model while binary or count data that rarely goes beyond 1 occurrence will be analyzed using a Logistic model. In case of count data that rarely goes beyond 1 occurrence, this data will be transformed to binary data (occurrence/no occurrence). We will present mean outcome ratios for count data (from Poisson models) and odds ratios for binary data (from logistic models) with respect to the intervention effect as well as 95% CI of the intervention effect. All analyses will be adjusted for repeated measures over time, so that patterns of change for these outcomes over time can be assessed by treatment group.

Infant developmentBirth through 24 months of age

To compare Bayley-IV scores between intervention arms, we will perform a linear mixed-effects model with a fixed effect for the intervention group and a random effect for study site. We will report point estimates for the group mean difference along with a 95% CI, and the team will repeat this analytical approach for each of the Bayley-IV domains.

Descriptive statistics (means, medians, SD, percentiles) for continuous secondary outcomes and frequency based statistics (N and percentages) for binary secondary outcomes will be generated and summarized in a tabular form by treatment group.

Trial Locations

Locations (24)

Loma Linda University Medical Center

🇺🇸

Loma Linda, California, United States

University of Arizona

🇺🇸

Tucson, Arizona, United States

West Virginia University Hospital

🇺🇸

Morgantown, West Virginia, United States

Women & Infants Hospital of Rhode Island

🇺🇸

Providence, Rhode Island, United States

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Central Michigan University

🇺🇸

Detroit, Michigan, United States

Metrohealth

🇺🇸

Cleveland, Ohio, United States

Nationwide Childeren's Hospital

🇺🇸

Columbus, Ohio, United States

RTI International

🇺🇸

Durham, North Carolina, United States

Washington University School of Medicine

🇺🇸

Saint Louis, Missouri, United States

Ohio State University Hospital

🇺🇸

Columbus, Ohio, United States

Sanford Health

🇺🇸

Sioux Falls, South Dakota, United States

University of Arkansas Medical Sciences

🇺🇸

Little Rock, Arkansas, United States

University of Iowa

🇺🇸

Iowa City, Iowa, United States

Sharp Mary Birch Hospital for Women and Newborns

🇺🇸

San Diego, California, United States

Tulane University Health Science Center

🇺🇸

New Orleans, Louisiana, United States

Ochsner Baptist Clinical Trials Unit

🇺🇸

New Orleans, Louisiana, United States

Children's Mercy Hospital

🇺🇸

Kansas City, Missouri, United States

University of Massachusetts Memorial Medical Center-West Campus

🇺🇸

Worcester, Massachusetts, United States

Ochsner Medical Regional Hospital

🇺🇸

Kenner, Louisiana, United States

MedStar Franklin Square

🇺🇸

Hyattsville, Maryland, United States

University of Tennessee Health Science Center

🇺🇸

Memphis, Tennessee, United States

The University of Tennessee Health Science Center

🇺🇸

Memphis, Tennessee, United States

University of New Mexico

🇺🇸

Albuquerque, New Mexico, United States

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