Exosomes Effect on Visual Function in CVI

The Effect of Nasal Mesenchymal Exosome Administration on Vision Function in CVI

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 (CVİ).

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:

• They will receive exosomes via the nasal route every month for 6 months.
• They will visit the clinic every month for monitoring and tests.

Study Overview

• Status: Completed
• Conditions: Cortical Visual Impairment; Visual Function
• Intervention / Treatment: Other: Molecularly matched therapy

Detailed Description

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).

• Study Type: Observational
• Enrollment (Actual): 32

Contacts and Locations

Study Locations

• Turkey (Türkiye)
  • Tuzla
    • Istanbul, Tuzla, Turkey (Türkiye)
      • Istanbul Okan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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.

Description

Inclusion Criteria:

• Phase I Cerebral Palsy and Cortical Visual Impairment
• During the 12 months period recieved 4-6 times intranasal exosome

Exclusion Criteria:

• Irregular follow-up
• Abondened the study

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

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.

Other: Molecularly matched therapy; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Exploratory Objectives (Neuroplasticity Mechanisms)	Quantification of "Delayed Field → Functional Field" transition rate	Up to 1 year

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Exploratory Objectives (Neuroplasticity Mechanisms)	Learning-curve slope analysis during rehabilitation tasks	Up to 1 year

Collaborators and Investigators

• Sponsor: Hatice Semrin Timlioglu İper

Publications and helpful links

Helpful Links

• Current Modalities for Low Vision Rehabilitation
• Recent Advances in Extracellular Vesicle-Based Therapies Using Induced Pluripotent Stem Cell-Derived Mesenchymal Stromal Cells
• Extracellular vesicle-based therapy application and efficacy in ophthalmic diseases: from origin to target
• Extracellular vesicles as a new horizon in the diagnosis and treatment of inflammatory eye diseases: A narrative review of the literature
• Safety and efficacy outcomes after intranasal administration of neural stem cells in cerebral palsy: a randomized phase 1/2 controlled trial
• Mesenchymal stem cell-derived exosomes therapy: research progress and mechanism of action
• Updates on neonatal cell and novel therapeutics: Proceedings of the Second Neonatal Cell Therapies Symposium (2024)
• Successful treatment of cortical visual impairment in children using anti-amblyopia treatment despite the absence of amblyopia: a case report
• Possible Effect of the use of Mesenchymal Stromal Cells in the Treatment of Autism Spectrum Disorders: A Review
• Research progress of stem cell therapy for neurological diseases
• Extracellular Vesicles: Biomarkers, Therapeutics, and Vehicles in the Visual System
• Exosomes derived from microRNA-22-3p-overexpressed mesenchymal stem cells protect retinal ganglion cells by regulating MAPK pathway
• 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

Study record dates

Study Major Dates

• Study Start (Actual): June 30, 2024
• Primary Completion (Actual): July 19, 2025
• Study Completion (Actual): September 24, 2025

Study Registration Dates

• First Submitted: February 8, 2026
• First Submitted That Met QC Criteria: March 20, 2026
• First Posted (Actual): March 25, 2026

Study Record Updates

• Last Update Posted (Actual): March 25, 2026
• Last Update Submitted That Met QC Criteria: March 20, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Exosome; Cortical Visual Impairment; Visual Function; Nasal Mesenchymal Exosome
• Additional Relevant MeSH Terms: Neurologic Manifestations; Nervous System Diseases; Eye Diseases; Vision Disorders; Sensation Disorders; Blindness; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Blindness, Cortical
• Other Study ID Numbers: 2025/12-05

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: All IPD that underlie results in a publication
• IPD Sharing Access Criteria: Open access for academics
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; ICF; ANALYTIC_CODE; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No