Efficacy and Safety of Stannsoporfin in Neonates

Study Overview
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Brief Summary
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This is a multi-center, randomized, sham injection-controlled (placebo) masked trial of a single intramuscular injection of Stannsoporfin compared to sham (placebo) in healthy term and near-term newborns admitted to the well-baby nursery and enrolled with intention to treat.

Detailed Description
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For purposes of analysis, 2 patient populations are defined. One population consists of those babies who did not develop severe hyperbilirubinemia (TSB <9 mg/dL) during the first 36 hours of age. This population is referred to as the untreated population of screened but not randomized patients. The second population of babies was defined as those infants who develop severe hyperbilirubinemia. These infants were randomized to treatment with either stannsoporfin or the sham injection and will be the focus of the efficacy and safety analysis. This population was referred to as the treated population. The treated population was used for the efficacy and safety analysis. These infants were randomized to either stannsoporfin or the sham injection treatment group.

Official Title
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A Multicenter Study to Evaluate the Efficacy and Safety of Tin Mesoporphyrin (Stannsoporfin) to Reduce the Need for Phototherapy in Term and Near Term Infants.

Conditions
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Nutritional and Metabolic Diseases

Intervention / Treatment
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* Drug: stannsoporfin
* Other: Sham Injection


Participation Criteria
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Eligibility Criteria
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Patients were enrolled who meet the following criteria: The infant's family may be approached at anytime from 6 hours after birth onward to discuss the possibility of enrollment. However, Informed Consent may not be signed until the baby is between 12 and 36 hours of age. Consent may be obtained as soon as possible after 12 hours of age, when the baby enters the well-baby nursery. An infant may be included if the mother has received, or the baby is receiving, antibiotics and the infant is asymptomatic. For example, the baby has no signs of cardiorespiratory distress and is feeding well. Symptomatic infants are excluded (see Exclusions, below). Born at the study hospital site and admitted to the well-baby nursery or admitted to the intensive-care nursery for <12 hours for post delivery observation (delayed transition, temperature or dextrose instability, meconium staining and/or parental concern). All babies on oral feeding with stable cardiorespiratory status and deemed healthy upon clinical examination, with normal perfusion as defined by capillary filling of the fingernail. Term neonate (≥38 weeks of completed gestation), OR Near-term neonate (>36<38 weeks of completed gestation; >2,000g birth weight), OR Near-term neonate (>35<38 weeks of completed gestation; >2,500g birth weight) Care provided (primarily) in the well-baby nursery population; Absence of concurrent cardiorespiratory distress, sepsis, major congenital anomalies or need for care in an intensive care nursery; Not participating in another concurrent unrelated study. Exclusion Criteria The study enrolled patients who did not have any of the following exclusion criteria: Any condition that in the opinion of the investigator would make the subject unsuitable for the study. Infants receiving antibiotics who are also symptomatic are excluded. For example, a baby who is feeding poorly or is in cardiorespiratory distress is excluded. Mother has received phenobarbital in past 30 days.

Ages Eligible for Study
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Minimum Age: 12 Hours
Maximum Age: 36 Hours

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Active drug-Stannsoporfin<br>Stannsoporfin, single dose 4.5mg/kg administered Intramuscular (parental injection in the thigh) for treatment of jaundice 20 mg/mL 1.5 mL/vial | Drug: stannsoporfin<br>* Intramuscular injection of stannsoporfin to treat jaundice.<br>|
| Sham Comparator: Reference Therapy-Sham<br>Sham Injection, no injection followed by a Band-Aid to thigh | Other: Sham Injection<br>* nothing<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Need for Phototherapy | proportion of cases which need phototherapy in the treated population | 30 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| hyperbilirubinemia | Change in serum bilirubin values | 12 hrs | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Kernicterus, Neonatal Jaundice, Newborn Jaundice, Hyperbilirubinemia, Stannsoporfin