Intravitreal Injection of MSCs in Retinitis Pigmentosa

NCT ID: NCT01531348

Last Updated: 2021-09-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-04-30

Study Completion Date

2020-07-31

Brief Summary

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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

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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.

Conditions

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Retinitis Pigmentosa

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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BM-MSC

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

Group Type EXPERIMENTAL

BM-MSC

Intervention Type OTHER

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

Interventions

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BM-MSC

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

Intervention Type OTHER

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ministry of Health, Thailand

OTHER_GOV

Sponsor Role collaborator

Mahidol University

OTHER

Sponsor Role lead

Responsible Party

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La-ongsri Atchaneeyasakul

Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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La-ongsri Atchaneeyasakul, MD

Role: PRINCIPAL_INVESTIGATOR

Siriraj Hospital

Locations

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Siriraj Hospital Mahidol University

Bangkoknoi, Bangkok, Thailand

Site Status

Countries

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Thailand

References

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Hartong DT, Berson EL, Dryja TP. Retinitis pigmentosa. Lancet. 2006 Nov 18;368(9549):1795-809. doi: 10.1016/S0140-6736(06)69740-7.

Reference Type BACKGROUND
PMID: 17113430 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 15565292 (View on PubMed)

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.

Reference Type RESULT
PMID: 16896916 (View on PubMed)

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.

Reference Type RESULT
PMID: 20169166 (View on PubMed)

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.

Reference Type DERIVED
PMID: 33422139 (View on PubMed)

Other Identifiers

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RP-001

Identifier Type: -

Identifier Source: org_study_id

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