Using stem cell-derived vesicles to treat 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
This study is testing if injections of tiny particles from stem cells can help people with retinitis pigmentosa improve their vision and stay safe during treatment.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Mahidol University Academic / other |
| Locations | 1 site (Bangkok Noi, Bangkok) |
| Trial ID | NCT06242379 on ClinicalTrials.gov |
What this trial studies
This clinical trial evaluates the safety and efficacy of intravitreal injections of small extracellular vesicles derived from bone marrow mesenchymal stem cells in patients with retinitis pigmentosa. The study builds on previous research that indicated potential benefits from stem cell treatments for inherited retinal diseases. Participants will be monitored for safety and visual acuity improvements following the treatment. The trial aims to minimize adverse effects while maintaining therapeutic benefits through a cell-free approach.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older with a clinical diagnosis of retinitis pigmentosa and specific visual acuity and field criteria.
Not a fit: Patients with significant ocular or non-ocular diseases, or those who are pregnant or lactating, may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve vision and quality of life for patients suffering from retinitis pigmentosa.
How similar studies have performed: Previous studies using stem cell therapies for retinal diseases have shown promising results, suggesting potential for success in this novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Bangkok Noi, Bangkok
- Siriraj Hospital — Bangkok Noi, Bangkok, Thailand (Recruiting)
Study contacts
- Principal investigator: Laongsri Atchaneeyasakul, Professor — Mahidol University
- Study coordinator: Laongsri Atchaneeyasakul, Professor
- Email: atchanee@hotmail.com
- Phone: +66 893138367
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.