Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The goal of this observational study is to learn about the effects of nazally applied exosome treatment on visual functions in children with Phase 1 Cerebral visual impairment (CVÄ°).
The main question it aims to answer is:
Does exosome therapy administered via the nasal route for neurological disorders in children with Phase 1 CVI also show a beneficial effect on visual functions? Researchers will compare visual and visual function findings before and after the exosome application to determine whether the exosome application is also effective in visual function.
Participants:
Purpose Cortical visual impairment (CVI) denotes a pediatric visual deficit arising from non-ocular etiologies, purportedly linked to perturbations in visual cortical processing regions. This syndrome characteristically ensues from perinatal insults-such as asphyxia, prematurity, neonatal hypoglycemia, or hypoxia-inflicting damage upon retro-geniculate visual pathways and cortical centers. Empirical evidence underscores cerebral neuroplasticity, facilitating reorganization despite congenital retro-geniculate lesions; nonetheless, CVI manifests heterogeneous visual and cognitive sequelae. With 17% of neonates necessitating intensive care, advancements in neonatal care have augmented survival, concomitantly escalating the prevalence of neurodevelopmental disorders including CVI and cerebral palsy (CP), thereby spurring investigational reparative modalities such as stem cell and molecular therapeutics.
Methods This investigation prospectively enrolled 32 children aged 0.6-13 years diagnosed with CVI, who received intranasal exosome therapy (5 × 10^6 particles/dose) between 2023 and 2024. Administration occurred in 4-6 iterations at monthly intervals. Comprehensive neuro-ophthalmic evaluations encompassed dynamic retinoscopy, preferential looking assessments (LEA and Cardiff cards) for visual acuity and Visual Function Index, alongside surveillance of ventral stream deficits (e.g., delayed gaze, complexity aversion) and dorsal stream impairments (e.g., visuomotor orienting). Statistical analyses employed IBM SPSS Statistics version 25.0 (IBM Corp., Armonk, NY, USA).
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| study group | The cohort comprised 32 pediatric volunteers aged 12-34 months diagnosed with Phase 1 cortical visual impairment (CVI) concomitant with simultaneous perception (SP) deficit at Zatay Paediatric Neurology Clinic between June 2024 and July 2025. All participants underwent intranasal exosome therapy administered in 4-6 monthly doses over a 12-month surveillance interval. Data were prospectively accrued via clinical files and serial neuro-ophthalmic examination records, with comprehensive follow-up spanning 12 months to delineate therapeutic trajectory and neuroplastic adaptations. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Molecularly matched therapy | Other | Derived from mesenchymal stem cells, exosomes are nano-scale vesicles characterized by their regenerative and anti-inflammatory capabilities. These vesicles can traverse the blood-brain barrier to transport functional cargo, including lipids, mRNA, and proteins, to target cells. Over the course of 12 months, volunteers were administered 5 million units of exosomes in 7 ml via nasal spray once a month for a total of 4-6 doses, with an infusion rate of 1 ml per hour. |
| Measure | Description | Time Frame |
|---|---|---|
| Whether exosome therapy combined with standardized neuro-visual rehabilitation improves functional vision outcomes compared to rehabilitation plus placebo. | Change in Visual Function Index (VFI) total score The total score for visual function is 15, comprising the sum of 13 functions, each worth 1 point, and fixation, which is worth 2 points. Missing functions are scored as 0. | Up to 1 year |
| Primary Objective ( Safety) | To evaluate the advers effects of repeated intranasal exosome administration in pediatric patients with CVI over a 12-week treatment period. Epileptic attack Allergic reactions Treatment discontinuation due to adverse events | Up to 1 year |
| Affected Visual fields (Totally impaired /delayed) | Impaired Visual Field Before, n (%) 0/1 | Up to 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Exploratory Objectives (Neuroplasticity Mechanisms) | Quantification of "Delayed Field → Functional Field" transition rate | Up to 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Exploratory Objectives (Neuroplasticity Mechanisms) | Learning-curve slope analysis during rehabilitation tasks | Up to 1 year |
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
The study population consisted of pediatric patients aged 12-34 months who dere diagnosed with Phase 1 CVI and SP and who were referred to the Zatay Pediatric Neurology Clinic. All participants recieved exosome therapy 4-6 times over a 12 month period, administered monthly.
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Istanbul Okan University | Istanbul | Tuzla | Turkey (Türkiye) |
Not provided
| Label | URL |
|---|---|
| Current Modalities for Low Vision Rehabilitation | View source |
| Recent Advances in Extracellular Vesicle-Based Therapies Using Induced Pluripotent Stem Cell-Derived Mesenchymal Stromal Cells | View source |
Not provided
All IPD that underlie results in a publication
Not provided
Open access for academics
Not provided
Not provided
| ID | Term |
|---|---|
| D019575 | Blindness, Cortical |
| ID | Term |
|---|---|
| D001766 | Blindness |
| D014786 | Vision Disorders |
| D012678 | Sensation Disorders |
| D009461 | Neurologic Manifestations |
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Extracellular vesicle-based therapy application and efficacy in ophthalmic diseases: from origin to target |
| View source |
| Extracellular vesicles as a new horizon in the diagnosis and treatment of inflammatory eye diseases: A narrative review of the literature | View source |
| Safety and efficacy outcomes after intranasal administration of neural stem cells in cerebral palsy: a randomized phase 1/2 controlled trial | View source |
| Mesenchymal stem cell-derived exosomes therapy: research progress and mechanism of action | View source |
| Updates on neonatal cell and novel therapeutics: Proceedings of the Second Neonatal Cell Therapies Symposium (2024) | View source |
| Successful treatment of cortical visual impairment in children using anti-amblyopia treatment despite the absence of amblyopia: a case report | View source |
| Possible Effect of the use of Mesenchymal Stromal Cells in the Treatment of Autism Spectrum Disorders: A Review | View source |
| Research progress of stem cell therapy for neurological diseases | View source |
| Extracellular Vesicles: Biomarkers, Therapeutics, and Vehicles in the Visual System | View source |
| Exosomes derived from microRNA-22-3p-overexpressed mesenchymal stem cells protect retinal ganglion cells by regulating MAPK pathway | View source |
| Human amniotic mesenchymal stromal cell-derived exosomes promote neuronal function by inhibiting excessive apoptosis in a hypoxia/ischemia-induced cerebral palsy model: A preclinical study | View source |
| D009422 |
| Nervous System Diseases |
| D005128 | Eye Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |