Intravitreal Injection of MSCs in Retinitis Pigmentosa

Phase 1

Completed

• Conditions: Retinitis Pigmentosa
• Interventions: Other: BM-MSC

• Registration Number: NCT01531348
• Lead Sponsor: Mahidol University

Brief Summary

The purpose of this study is to determine the feasibility and safety of adult human bone marrow-derived mesenchymal stem cells by intravitreal injection in patients with retinitis pigmentosa.

Detailed Description

Retinitis pigmentosa (RP) is an inherited disorder of the photoreceptor cells in the retina. Patients may lose vision since they were young or later in life. Currently, there are more than 60 genes identified as the cause of this condition, one of which, RPE65, has been studied in several gene therapy trials for Leber congenital amaurosis with promising results. Another treatment approach for RP is stem cell therapy. Studies in animal models of RP have shown that subretinal injection of bone marrow-derived mesenchymal stem cells may delay degenerative changes of photoreceptor cells.

Study Timeline

• Study First Submitted: 2012-02-06
• Study First Submitted QC: 2012-02-08
• Study First Posted: 2012-02-10
• Study Start: 2012-04
• Primary Completion: 2020-07
• Study Completion: 2020-07
• Status Verified: 2021-08
• Last Update Submitted: 2021-08-31
• Last Update Posted: 2021-09-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 14

Inclusion Criteria

• Retinitis pigmentosa patients diagnosed by ophthalmologists
• Age 18-65 years old
• Central visual field less than or equal to 20 degrees
• Best corrected visual acuity less than 6/120 by Snellen visual acuity chart
• Electroretinogram nonrecordable or the amplitudes were less than 25% of normal

Exclusion Criteria

• Other eye conditions that could mask the interpretation of the results
• Unable to return for follow up
• Underlying diseases including asthma, heart failure, myocardial infarction, liver failure, renal failure
• Pregnant and lactating women

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BM-MSC	BM-MSC	Bone marrow-derived mesenchymal stem cells 1 million cells in balanced salt solution 100 microlitres will be injected into the vitreous cavity.

Primary Outcome Measures

Name	Time	Method
Change from baseline in laser flare and cell measurements	up to 12 months

Secondary Outcome Measures

Name	Time	Method
Change from baseline in visual function tests	up to 12 months

Trial Locations

Locations (1)

Siriraj Hospital Mahidol University; 🇹🇭 Bangkoknoi, Bangkok, Thailand