Clinical Research Directory

Healing Tiny Minds: Rural Recovery Through Virtual and Lived Experience Care

The goal of this behavioral-interventional study is to learn if the Abecedarian Approach implemented virtually for children ages 0-5 with a history of Hypoxic Ischaemic Encephalopathy (HIE) and/or premature birth produces the same effects as when administered at in-person facilities.

Intranasal AAV9-PHP.eB Gene Therapy in Cerebral Palsy (CP) & Hypoxic Ischemic Encephalopathy (HIE)

This international study, organized by Healing Hope International, is an observational registry designed to collect real-world data on participants living with chronic hypoxic ischemic encephalopathy (HIE) who receive an emerging intranasal gene therapy based on the AAV9-PHP.eB viral vector. The investigational therapy delivers a panel of 15 restorative genes that support brain repair, reduce inflammation, promote myelination, and improve neural communication. It is administered intranasally in one or three sessions by participating international clinical teams. Because the therapy is already being offered abroad, this registry does not assign treatment but instead follows participants who have received it as part of their existing medical care. The GEN HOPE Study aims to understand how this gene therapy affects movement, cognition, spasticity, and seizure frequency over time. Families and clinicians will share outcomes such as changes in gross motor function (GMFM-66/88), cognitive assessments (Bayley or WISC tests), and quality-of-life measures. Information on safety, laboratory results, MRI findings, and caregiver-reported experiences will also be collected. By combining data from multiple countries, the registry seeks to evaluate whether this novel gene based approach can meaningfully improve daily function and comfort for participants with chronic HIE. Results will guide future clinical trial development and help define safe and effective standards of care for regenerative neurologic therapies.

Genetics of Neonatal Encephalopathy and Related Disorders

Investigators at Boston Children's Hospital are conducting research in order to better understand the genetic factors which may contribute to neonatal encephalopathy (NE) and related disorders. These findings may help explain the broad spectrum of clinical features and outcomes seen in individuals with a history of NE.

Measuring Heart Health in Both Term, Preterm and Unwell Newborn Babies With an Advanced Ultrasound Method: Speckle Tracking Echocardiography

This study aims to improve how neonatologists check the heart function of newborn babies, especially those who are sick. While standard heart ultrasound scans are useful, a more advanced and sensitive technique called 2D speckle tracking echocardiography (STE) can detect subtle problems with how the heart muscle squeezes and relaxes. This may allow doctors to spot potential issues earlier. Our research will take place at Birmingham Women's Hospital. The investigators will perform these advanced, non-invasive heart scans on several groups of babies: 1. Healthy term and premature babies, to establish a "normal" range of heart function. 2. Babies who are unwell with specific conditions, including those with brain injury due to lack of oxygen at birth (HIE), chronic lung disease of prematurity (BPD), a hole in the diaphragm (CDH), or high blood pressure in their lungs (aPHN). The heart scan is a standard, painless procedure. Using STE does not require any extra scanning time or cause any additional discomfort to the baby; the special images are taken during the routine scan. For many of the sick babies, these scans are already part of their normal clinical care. The main goals of this observational study are to see if STE is a feasible and reliable tool in newborns, to establish normal values for healthy babies, and to track how heart function changes in sick babies during their illness and recovery. Ultimately, the investigators hope this research will provide doctors with a better tool to assess heart health in newborns. This could lead to earlier, more accurate detection of heart problems and help guide treatment decisions to improve outcomes for these vulnerable infants.

EFFICACY OF MELATONIN IN MANAGEMENT OF HYPOXIC ISCHEMIC ENCEPHALOPATHY (HIE) IN NEONATES

To compare the survival rate and improvement in stage of hypoxic ischemic encephalopathy (HIE) at day 7 of treatment in neonates treated with versus without melatonin in addition to standard supportive therapy.

Feasibility and Safety of Intranasally Administered Breast Milk in HIE

This is a prospective intervention single center study to evaluate the feasibility and safety of intranasal breast milk in hypoxic-ischaemic encephalopathic neonates receiving therapeutic hypothermia.