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RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

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

Safety and Efficacy Evaluation of Intravitreal Injection of GMP-compliant Bone Marrow Mesenchymal Stem Cell-derived Small Extracellular Vesicles in Patients With Retinitis Pigmentosa

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 willing to sign a consent form for participation in the study Who Should NOT Join This Trial: - 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 weakened immune system 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 1. Inherited or acquired bleeding disorders, including the use of anticoagulant medications that cannot be stopped prior the procedure 2. autoimmune conditions (where your immune system attacks your own body)s, i.e., systemic lupus erythematosus, multiple sclerosis, fibromyalgia, Guillain-Barre syndrome 3. 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 Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * 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 Exclusion Criteria: * 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 1. Inherited or acquired bleeding disorders, including the use of anticoagulant medications that cannot be stopped prior the procedure 2. Autoimmune diseases, i.e., systemic lupus erythematosus, multiple sclerosis, fibromyalgia, Guillain-Barre syndrome 3. 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

Treatments Being Tested

BIOLOGICAL

GMP compliant-BM-MSC derived sEVs

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.

Locations (1)

Siriraj Hospital
Bangkok Noi, Bangkok, Thailand