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

Sponsor
Healing Hope International
Study ID
NCT07264166
Status
Enrolling By Invitation

Conditions

  • Cerebral Palsy
  • Cerebral Palsy (CP)
  • Cerebral Palsy Hemiparetic Cerebral Palsy Spasticity Gait Disorders, Neurologic Postural Balance Impairment
  • Cerebral Palsy Quadriplegic
  • Cerebral Palsy Spastic Diplegia
  • Hypoxic Brain Damage
  • Hypoxic Ischaemic Encephalopathy (HIE)
  • Hypoxic Ischaemic Encephalopathy Due to Cardiac Arrest
  • Hypoxic Ischemic Encephalopathy of Newborn

Eligibility Criteria

Sex
ALL
Age
2 Years - 65 Years
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

First listed
Dec 4, 2025
Start date
Mar 1, 2026
Status verified
Mar 2026
Primary completion
Jan 1, 2032
Completion
Jun 15, 2032

Study Design

Enrollment
25 participants (estimated)

Arms

  • Arm: Intranasal 15-Gene AAV9-PHP.eB Therapy Cohort
    This cohort includes patients diagnosed with chronic hypoxic-ischemic encephalopathy (HIE) who receive an intranasal 15-gene AAV9-PHP.eB therapy as part of their existing medical care at participating international sites.

Primary Outcome Measure

Change in Gross Motor Function Measure (GMFM-66/88) Score From Baseline to 12 Months [ Time Frame: Baseline to 12 months after treatment ]

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