Exosomes Effect on Visual Function in CVI

NCT07492966 | Completed DMC

• 1 other identifier: 2025/12-05
• Study Type: observational
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cortical Visual Impairment; Visual Function

Keywords

Cortical Visual Impairment; Visual Function; Nasal Mesenchymal Exosome; Exosome

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (1)

• Exploratory Objectives (Neuroplasticity Mechanisms)

  Up to 1 year

Other Outcomes (1)

• Exploratory Objectives (Neuroplasticity Mechanisms)

  Up to 1 year

Study Arms (1)

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

Interventions

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.

study group

Eligibility Criteria

• Age: 12 Months - 34 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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.

You may qualify if:

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

You may not qualify if:

• Irregular follow-up
• Abondened the study

Sponsors & Collaborators

• Hatice Semrin Timlioglu İper: lead

Study Sites (1)

Istanbul Okan University

Istanbul, Tuzla, Turkey (Türkiye)

Location

Related 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

MeSH Terms

Conditions

Blindness, Cortical

Condition Hierarchy (Ancestors)

Blindness; Vision Disorders; Sensation Disorders; Neurologic Manifestations; Nervous System Diseases; Eye Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: CROSS SECTIONAL
• Target Duration: 12 Months
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: February 8, 2026
• First Posted: March 25, 2026
• Study Start: June 30, 2024
• Primary Completion: July 19, 2025
• Study Completion: September 24, 2025
• Last Updated: March 25, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF, CSR, ANALYTIC CODE
• Access Criteria: Open access for academics

Locations

TU (1)