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

This study is enrolling participants by invitation only.

Sponsor:

Collaborator:

Ministry of Health, Thailand

Information provided by (Responsible Party):

La-ongsri Atchaneeyasakul, Mahidol University

ClinicalTrials.gov Identifier:

NCT01531348

First received: February 6, 2012

Last updated: February 8, 2012

Last verified: February 2012

History of Changes

Purpose

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.

Condition	Intervention	Phase
Retinitis Pigmentosa	Other: Intravitreal injection of bone marrow-derived mesenchymal stem cells	Phase 1

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label
Official Title:	Feasibility and Safety of Adult Human Bone Marrow-Derived Mesenchymal Stem Cells by Intravitreal Injection in Patients With Retinitis Pigmentosa

Resource links provided by NLM:

Genetics Home Reference related topics: Lenz microphthalmia syndrome oculofaciocardiodental syndrome Peters plus syndrome retinitis pigmentosa X-linked juvenile retinoschisis

U.S. FDA Resources

Further study details as provided by Mahidol University:

Primary Outcome Measures:

Change from baseline in laser flare and cell measurements [ Time Frame: up to 12 months ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Change from baseline in visual function tests [ Time Frame: up to 12 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	10
Study Start Date:	February 2012
Estimated Primary Completion Date:	March 2014 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
BM-MSC	Other: Intravitreal injection of bone marrow-derived mesenchymal stem cells Bone marrow-derived mesenchymal stem cells 1 million cells in balanced salt solution 100 microlitres will be injected into the vitreous cavity.

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

Ages Eligible for Study:	18 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01531348

Locations

Thailand
Siriraj Hospital Mahidol University
Bangkoknoi, Bangkok, Thailand, 10700

Sponsors and Collaborators

Mahidol University

Ministry of Health, Thailand

Investigators

Principal Investigator:

La-ongsri Atchaneeyasakul, MD

Siriraj Hospital, Mahidol University

More Information

Publications:

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. Epub 2006 Aug 4.

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.

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

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. No abstract available.

Responsible Party:	La-ongsri Atchaneeyasakul, Professor, Mahidol University
ClinicalTrials.gov Identifier:	NCT01531348 History of Changes
Other Study ID Numbers:	RP-001
Study First Received:	February 6, 2012
Last Updated:	February 8, 2012
Health Authority:	Thailand: Siriraj Institutional Review Board Thailand: The Medical Council of Thailand

Keywords provided by Mahidol University:

Retinitis pigmentosa
Bone marrow-derived mesenchymal stem cells
Intravitreal injection

Additional relevant MeSH terms:

Retinitis
Retinitis Pigmentosa
Retinal Diseases
Eye Diseases

Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on May 19, 2013

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