Table of Contents
- Overview of the Allopurinol Sodium trials
- Trial in hypoxic-ischemic encephalopathy
- Trial in critical congenital heart disease
- Main outcomes measured
- Who the studies are for
- Study design and phases
Overview of the Allopurinol Sodium trials
These clinical trials are studying Allopurinol Sodium in newborn babies at high risk of brain injury.[1][2] The studies are looking at whether early treatment can improve short-term brain findings and longer-term development.[1][2]
Trial in hypoxic-ischemic encephalopathy
NCT03162653 is a Phase 3 interventional trial in newborns with hypoxic-ischemic encephalopathy, also called HIE, which is brain injury linked to low oxygen and poor blood flow around birth.[1] The study includes babies with asphyxia and early clinical signs of HIE, and it evaluates early postnatal Allopurinol compared with placebo in addition to standard care, including therapeutic hypothermia if needed.[1]
The main outcome is a combined result at 2 years of age: death, severe neurodevelopmental impairment, or survival without severe neurodevelopmental impairment.[1] Severe neurodevelopmental impairment is defined in the trial as cerebral palsy or cognitive or language impairment, with language and cognitive scores below 85 on the Bayley Scales of Infant and Toddler Development, 3rd edition.[1]
Trial in critical congenital heart disease
NCT04217421 is a Phase 3 interventional trial in neonates with critical congenital heart disease who require cardiac surgery with cardiopulmonary bypass.[2] This study tests early postnatal and perioperative Allopurinol compared with placebo, which is listed as mannitol in the trial record.[2]
The trial is focused on brain protection during a very high-risk period around surgery.[2] Its main outcome is a composite endpoint of moderate or severe brain injury on postoperative MRI, being too unstable for MRI, or death.[2]
Main outcomes measured
The two trials use outcomes that matter to families and doctors because they reflect survival, brain injury, and later development.[1][2] In the HIE study, the key outcome is measured at 24 months, which helps show whether early treatment may affect long-term child development.[1]
In the heart disease study, the outcome is measured soon after surgery with MRI and includes clinical instability and death, which are important early safety and effectiveness signals.[2]
Who the studies are for
Both trials are for newborns, also called neonates.[1][2] One study includes babies with birth-related asphyxia and early signs of HIE, while the other includes babies with critical congenital heart disease needing surgery with bypass support.[1][2]
Babies with HIE are enrolled because the trial wants to see if early treatment can reduce death or serious developmental problems later in life.[1]
Babies with critical congenital heart disease are enrolled because the trial wants to see if treatment can reduce brain injury around the time of surgery.[2]
Study design and phases
Both studies are interventional trials, which means the researchers give a treatment and then compare outcomes between groups.[1][2] Both are also Phase 3 studies, with enrolment of 503 in the HIE trial and 236 in the congenital heart disease trial.[1][2]
The status of both trials is Authorised, showing that the studies have been approved to proceed in the source data.[1][2]
