Clinical trial NCT01531348: Intravitreal Injection of MSCs in Retinitis Pigmentosa
Official title: Feasibility and Safety of Adult Human Bone Marrow-Derived Mesenchymal Stem Cells by Intravitreal Injection in Patients With Retinitis Pigmentosa
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Retinitis Pigmentosa
Keywords: Retinitis pigmentosa
Bone marrow-derived mesenchymal stem cells
Intravitreal injection
Lead sponsor: Mahidol University
Sponsor class: OTHER
Collaborators: name: Ministry of Health, Thailand
First posted: 2012-02-10
Enrollment: 14
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change from baseline in laser flare and cell measurements
timeFrame: up to 12 months
Secondary outcomes:
measure: Change from baseline in visual function tests
timeFrame: up to 12 months
Other outcomes:
Locations:
city: Bangkoknoi
country: Thailand
facility: Siriraj Hospital Mahidol University
state: Bangkok
Study officials:
affiliation: Siriraj Hospital
name: La-ongsri Atchaneeyasakul, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Hartong DT, Berson EL, Dryja TP. Retinitis pigmentosa. Lancet. 2006 Nov 18;368(9549):1795-809. doi: 10.1016/S0140-6736(06)69740-7.
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. doi: 10.1007/s00417-004-1062-0. Epub 2004 Nov 23. No abstract available.
citation: 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):414-22. doi: 10.1007/s00417-006-0382-7. Epub 2006 Aug 4.
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.0009200.
citation: Tuekprakhon A, Sangkitporn S, Trinavarat A, Pawestri AR, Vamvanij V, Ruangchainikom M, Luksanapruksa P, Pongpaksupasin P, Khorchai A, Dambua A, Boonchu P, Yodtup C, Uiprasertkul M, Sangkitporn S, Atchaneeyasakul LO. Intravitreal autologous mesenchymal stem cell transplantation: a non-randomized phase I clinical trial in patients with retinitis pigmentosa. Stem Cell Res Ther. 2021 Jan 9;12(1):52. doi: 10.1186/s13287-020-02122-7.