Registry following people with cerebral palsy or brain oxygen injury after nasal gene therapy
Follows people with cerebral palsy or oxygen-related brain injury who received nasal gene therapy.
Plain-language title generated from the registry record
Registry title: Intranasal AAV9-PHP.eB Gene Therapy in Cerebral Palsy (CP) & Hypoxic Ischemic Encephalopathy (HIE)
Key facts
- Where
- Monterrey, MexicoSee the site
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The registry is looking for people aged 2 to 65 with long-term brain damage from lack of oxygen at birth or later (chronic HIE). They must have received or be receiving the intranasal 15-gene AAV9-PHP.eB gene therapy at a participating clinic.
- Age
- 2 to 65 years
- Sex
- All sexes can join.
- Disease stage
- Documented chronic hypoxic-ischemic encephalopathy (HIE), confirmed by medical history, MRI or newborn records. Stable condition for at least 6 months. Moderate movement difficulty, with a baseline GMFM-66 or GMFM-88 score between 40% and 70%.
- Earlier treatment
- Must have completed or be receiving intranasal 15-gene AAV9-PHP.eB therapy at a participating international clinical site. No previous gene therapy or investigational stem cell therapy within the past 12 months.
Main reasons for not taking part
- Active infection, immune deficiency, or ongoing use of immunosuppressive drugs (short-term rapamycin per the treating doctor is allowed)
- Known high anti-AAV9 neutralizing antibody level at baseline, if tested locally
- Uncontrolled seizures of more than five episodes per day despite treatment
- Known or suspected cancer, or severe liver or kidney problems
- Known pregnancy or breastfeeding in females after puberty
Read by machine on 12 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 65 years old?
Source: NCT07264166, who can take part
Where in Mexico
- Stem SolutionsMonterrey, MexicoNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Healing Hope International
- Trial code
- NCT07264166
- Record updated
- 23 March 2026
- Original description
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.Read full original descriptionShow less
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)
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.
Inclusion: 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.
Exclusion: 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; 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; Known pregnancy or breastfeeding in post-pubertal female participants.