Safety and Efficacy of Stem Cell Small Extracellular Vesicles in Patients With Retinitis Pigmentosa

NCT06242379 | Recruiting Phase 1

• 1 other identifier: R016534006
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of this clinical trials is to evaluate the safety and efficacy of intravitreal injection of GMP-compliant BM-MSC-derived sEVs in patients with retinitis pigmentosa.

Conditions

Retinitis Pigmentosa

Keywords

Retinitis pigmentosa; small extracellular vesicles

Outcome Measures

Primary Outcomes (12)

• Incidence of treatment-emergent adverse events

  To evaluate the safety of the intervention. The evaluations include the intraocular pressure in mmHg, cell value in number of cells in 0.5 cubic millimeter and flare value in photons per millisecond, anterior segment examination using slit lamp biomicroscopy, fundus evaluation using fundus photography, retinal assessment, and subjective complaints of study participants.

  1 year

• Ophthalmological parameters

  To observe the efficacy of the intervention. The evaluations comprise the best corrected visual acuity (BCVA) in the Logarithm of the Minimum Angle of Resolution (LogMAR) score. The value "0" indicates "no loss", that is visual acuity equal to the reference standard (1.0, 20/20). Visual acuity better tans 1.0 (20/20) is represented by negative LogMAR values. LogMAR 1.0 is equivalent to 20/200. Blindness is defined as a best-corrected visual acuity worse than 1.3 LogMAR.

  1 year

• Ophthalmological parameters

  contrast sensitivity test using the Stereo Optical Functional Vision Analyzer (FVA) to measure contrast sensitivity by generating a contrast sensitivity function curve that portrays sensitivity on the Y-axis and spatial frequency on the X-axis.

  1 year

• Ophthalmological parameters

  sweep visual evoked potential in Snellen unit

  1 year

• Ophthalmological parameters

  kinetic visual field test in degree field of vision at four quadrants (superior, nasal, inferior, temporal)

  1 year

• Ophthalmological parameters

  optic disc and cup volume in cubic millimeter

  1 year

• Ophthalmological parameters

  retinal nerve fiber layer thickness in micron

  1 year

• Ophthalmological parameters

  electrophysiology (electroretinography in microvolt, visual evoked potential in millisecond and microvolt, multifocal electroretinography in signal strength response)

  1 year

• Ophthalmological parameters

  optical coherence tomography of the macula in micron

  1 year

• Ophthalmological parameters

  optical coherence tomography angiography in percent of vessel flow density

  1 year

• Ophthalmological parameters

  foveal avascular zone in square millimeter

  1 year

• Ophthalmological parameters

  macular thickness in micron

  1 year

Study Arms (1)

Bone marrow-mesenchymal stem cell-derived small extracellular vesicles

EXPERIMENTAL

Single intravitreal injection of Good Manufacturing Practice (GMP) compliant bone marrow (BM)-mesenchymal stem cell (MSC)-derived small extracellular vesicles (sEVs) (50 μg) for single eye

Biological: GMP compliant-BM-MSC derived sEVs

Interventions

GMP compliant-BM-MSC derived sEVs BIOLOGICAL

The procedure will be performed under topical anesthesia (0.5% tetracaine hydrochloride ophthalmic solution). The intravitreal injection will be performed by the retina specialist. Topical antiseptic (5% povidone iodine solution) will be applied on the periorbital and ocular surface. Eyelid speculum will be inserted to expose the injection area. It will include an intravitreal injection at the superotemporal quadrant (right eye) and superonasal quadrant (left eye), 3.5 to 4 mm posterior to the limbus. A 30-gauge needle will be used to deliver a 0.05 to 0.1 ml sEV suspension into the vitreous cavity. Indirect ophthalmoscopy will be performed immediately after the procedure to ensure no occlusion of the central retinal artery. The eye will be rinsed thoroughly by normal saline to wash out remaining antiseptic. The total duration for an intravitreal injection will be approximately 30 minutes.

Bone marrow-mesenchymal stem cell-derived small extracellular vesicles

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18 years or above
• Clinically diagnosed with RP by experienced ophthalmologists or having documented mutations in the genes responsible for RP
• Central visual field in the better eye less than or equal to 20 degrees
• Best corrected visual acuity (BCVA) in the worse eye 6/18 (logMAR 0.48) to 6/120 (logMAR 1.3) by Snellen visual acuity chart
• Electroretinogram in the worse eye nonrecordable or the amplitudes were less than 25% of normal
• Willing and able to give informed consent for participation in the study

You may not qualify if:

• Intolerance and/or contraindication to local anesthesia and other substances used during the procedure
• Pregnant or lactating woman
• Having blood-borne infections, i.e. Human immunodeficiency virus (HIV), hepatitis B or C, Human T-lymphotropic viruses (HTLV)
• Having any other significant ocular or non-ocular disease/disorder which may either put the subjects at risk because of participation in the study, or may influence the results of the study, or the subjects ability to participate in the study. This includes
• Inherited or acquired bleeding disorders, including the use of anticoagulant medications that cannot be stopped prior the procedure
• Autoimmune diseases, i.e., systemic lupus erythematosus, multiple sclerosis, fibromyalgia, Guillain-Barre syndrome
• Severe/uncontrolled chronic/metabolic diseases, e.g., diabetes mellitus, cardiovascular disease, chronic kidney disease, transient ischemic attack (TIA)/stroke
• Unable to complete the full course of the study or failed to return for follow up

Sponsors & Collaborators

• Mahidol University: lead

Study Sites (1)

Siriraj Hospital

Bangkok Noi, Bangkok, 10700, Thailand

RECRUITING

Related Publications (22)

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MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Laongsri Atchaneeyasakul, Professor

  Mahidol University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Laongsri Atchaneeyasakul, Professor

CONTACT

+66 893138367; atchanee@hotmail.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 12, 2024
• First Posted: February 5, 2024
• Study Start: May 23, 2024
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: May 15, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

TH (1)