Intravitreal Injection of MSCs in Retinitis Pigmentosa

Retinitis Pigmentosa Clinical Trial

Official title:

Feasibility and Safety of Adult Human Bone Marrow-Derived Mesenchymal Stem Cells by Intravitreal Injection in Patients With Retinitis Pigmentosa

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01531348
• Other study ID #: RP-001
• Status: Completed
• Phase: Phase 1
• Start date: April 2012
• Est. completion date: July 2020

Study information

• Verified date: August 2021
• Source: Mahidol University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: July 2020
• Est. primary completion date: July 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa

Intervention

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

Locations

Country	Name	City	State
Thailand	Siriraj Hospital Mahidol University	Bangkoknoi	Bangkok

Sponsors (2)

Lead Sponsor	Collaborator
Mahidol University	Ministry of Health, Thailand

Country where clinical trial is conducted

Thailand, 

References & Publications (4)

Arnhold S, Absenger Y, Klein H, Addicks K, Schraermeyer U. Transplantation of bone marrow-derived mesenchymal stem cells rescue photoreceptor cells in the dystrophic retina of the rhodopsin knockout mouse. Graefes Arch Clin Exp Ophthalmol. 2007 Mar;245(3) — View Citation

Hartong DT, Berson EL, Dryja TP. Retinitis pigmentosa. Lancet. 2006 Nov 18;368(9549):1795-809. Review. — View Citation

Kociok N. Can the injection of the patient's own bone marrow-derived stem cells preserve cone vision in retinitis pigmentosa and other diseases of the eye? Graefes Arch Clin Exp Ophthalmol. 2005 Mar;243(3):187-8. Epub 2004 Nov 23. — View Citation

Wang S, Lu B, Girman S, Duan J, McFarland T, Zhang QS, Grompe M, Adamus G, Appukuttan B, Lund R. Non-invasive stem cell therapy in a rat model for retinal degeneration and vascular pathology. PLoS One. 2010 Feb 15;5(2):e9200. doi: 10.1371/journal.pone.000 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in laser flare and cell measurements		up to 12 months
Secondary	Change from baseline in visual function tests		up to 12 months