NCT07264166Enrolling by invitation
Intranasal AAV9-PHP.eB Gene Therapy in Cerebral Palsy (CP) & Hypoxic Ischemic Encephalopathy (HIE)
Healing Hope International Multinational Observational Registry Evaluating Intranasal 15-Gene AAV9-PHP.eB Therapy and Functional Outcomes in Participants With Cerebral Palsy & Chronic Hypoxic-Ischemic Encephalopathy (HIE) (GEN-HOPE Study)
Source: ClinicalTrials.gov · Last updated 23 Mar 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Enrolling by invitation
- Study type
- Observational
- Enrollment
- 25 (estimated)
- First submitted
- 24 Nov 2025
- Start date
- 1 Mar 2026
- Primary completion date
- 1 Jan 2032
- Lead sponsor
- Healing Hope International
- NCT number
- NCT07264166
What you commit to
- Study type: Observational
- Planned participants: 25
- From 1 Mar 2026 to the primary completion date, 1 Jan 2032
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Known positive anti-AAV9 neutralizing antibody titer at baseline exceeding threshold values that may preclude effective vector transduction (if testing performed locally).
- Previous gene therapy or investigational stem cell therapy within the past 12 months.
Other illness
- Active systemic infection, immune deficiency, or ongoing use of immunosuppressive agents (other than short-term rapamycin used per treating physician's protocol).
- Uncontrolled seizure activity exceeding five episodes per day at baseline despite medical therapy.
- Known or suspected malignancy, severe hepatic or renal dysfunction, or other conditions that would confound safety monitoring.
- Any condition that, in the opinion of the local investigator or registry sponsor, may interfere with participation, data reliability, or patient safety.
Other
- Known pregnancy or breastfeeding in post-pubertal female participants.
Who the study is looking for
- Age 2 to 65 years, at the time of enrollment.
- Documented diagnosis of chronic hypoxic-ischemic encephalopathy (HIE), confirmed by medical history, MRI findings, or neonatal records.
- Stable medical condition for at least 6 months prior to enrollment (no major surgeries or hospitalizations related to HIE within that period).
- Baseline Gross Motor Function Measure (GMFM-66 or GMFM-88) score between 40% and 70%, representing moderate functional impairment.
- Completion or active receipt of intranasal 15-gene AAV9-PHP.eB therapy at a participating international clinical site under local physician supervision.
- Parent(s) or legal guardian(s) willing and able to provide written informed consent for participation in the observational registry and data sharing.
- Access to clinical follow-up and ability to participate in scheduled assessments or data submissions at 3, 6, 12, 18, and 24 months after treatment.
Age in the registry: 2 years to 65 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Stem Solutions Coordinator of this site? Confirm its status | Monterrey | Mexico | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.