A Safety and Efficacy Trial of Stannsoporfin in Neonates With Hyperbilirubinemia

Study Overview
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Brief Summary
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It is a normal process in the human body for red blood cells to die, which makes bilirubin. Bilirubin is cleared away through the liver. Some babies are born with livers that don't work well enough yet, or their red blood cells are dying too fast, so the baby looks yellow (jaundice). This means there is too much bilirubin in the body. It can be dangerous if a baby's bilirubin gets too high. Phototherapy is what they call the lights they shine on newborn babies to help the liver get rid of bilirubin. This study tests an experimental drug to see if it can reduce how much bilirubin is being made in the first place.

Detailed Description
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The purpose of this study is to determine if an experimental drug, stannsoporfin, is safe and effective in the treatment of hyperbilirubinemia in hemolyzing neonates.

Official Title
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A Phase 2b, Multicenter, Single-dose, Blinded, Randomized, Placebo-controlled, Dose-escalation, Safety and Efficacy Trial of Stannsoporfin in Neonates With Hyperbilirubinemia

Conditions
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Hyperbilirubinemia, Neonatal

Intervention / Treatment
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* Drug: Stannsoporfin
* Other: Placebo
* Other: PhotoTherapy (as needed)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Babies may only participate if they meet all the following criteria: Is a term or late preterm baby Is at risk for protocol-defined hemolytic disease Weighs at least 2500 g (5.5 lbs) Has total serum bilirubin (TSB) a specified amount lower than the phototherapy threshold for the age Has parents/guardians who are willing to follow light precautions and sign informed consent Exclusion Criteria: The following criteria will make a baby not eligible to participate: Needs medications that may prolong the QT interval Has family history or risk factors for Long QT Syndrome, Sudden Infant Death Syndrome, or Porphyrias Has an Apgar score of 6 or below at age 5 minutes Has abnormalities or infections (in mother or child) that per protocol or in the opinion of the investigator may compromise the safety and well-being of the baby or analysis of study results

Ages Eligible for Study
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Minimum Age: 1 Minute
Maximum Age: 48 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: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Sequential experimental cohorts are run in parallel with placebo controls.
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort 1: Stannsoporfin 1.5 mg/kg<br>Participants receive a single dose of 1.5 mg/kg by intramuscular (IM) injection, along with PhotoTherapy (PT) if and when needed. | Drug: Stannsoporfin<br>* Stannsoporfin administered as a single IM injection<br>* Other names: Experimental product;Other: PhotoTherapy (as needed)<br>* PT standard care administered as needed, based on bilirubin levels throughout the treatment period<br>* Other names: PT;|
| Experimental: Cohort 2: Stannsoporfin 3.0 mg/kg<br>Participants receive a single dose of 3.0 mg/kg by intramuscular (IM) injection, along with PT if and when needed. | Drug: Stannsoporfin<br>* Stannsoporfin administered as a single IM injection<br>* Other names: Experimental product;Other: PhotoTherapy (as needed)<br>* PT standard care administered as needed, based on bilirubin levels throughout the treatment period<br>* Other names: PT;|
| Experimental: Cohort 3: Stannsoporfin 4.5 mg/kg<br>Participants receive a single dose of 4.5 mg/kg by intramuscular (IM) injection, along with PT if and when needed. | Drug: Stannsoporfin<br>* Stannsoporfin administered as a single IM injection<br>* Other names: Experimental product;Other: PhotoTherapy (as needed)<br>* PT standard care administered as needed, based on bilirubin levels throughout the treatment period<br>* Other names: PT;|
| Placebo Comparator: Cohort 4: Placebo<br>Participants receive a single dose of placebo (sterile saline solution) by IM injection, along with PT if and when needed. | Other: Placebo<br>* Placebo (sterile saline solution) administered as a single IM injection<br>* Other names: Saline;Other: PhotoTherapy (as needed)<br>* PT standard care administered as needed, based on bilirubin levels throughout the treatment period<br>* Other names: PT;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Adjusted Total Serum Bilirubin (TSB) From Baseline to 48 Hours After Treatment. | The adjusted TSB is a calculation of the percentage difference of the TSB level from the age-specific threshold for PT initiation per the American Academy of Pediatrics (AAP) Guidelines, ie, an indication of the distance below the PT threshold at the time [(TSB - PT threshold/PT threshold) x 100%). | Baseline, 48 hours | 
| Change From Baseline in Total Serum Bilirubin (TSB) at 48 Hours (ITT Population | Total bilirubin in blood serum was measured at baseline and at 48 hours after the shot. Change from baseline is calculated by subtracting the amount at baseline from the amount at 48 hours. Lower numbers are better. | Baseline, 48 hrs | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Hemolysis