NCT01531348

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Apr 2012

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 6, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 10, 2012

Completed
2 months until next milestone

Study Start

First participant enrolled

April 1, 2012

Completed
8.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2020

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2020

Completed
Last Updated

September 8, 2021

Status Verified

August 1, 2021

Enrollment Period

8.3 years

First QC Date

February 6, 2012

Last Update Submit

August 31, 2021

Conditions

Retinitis Pigmentosa

Keywords

Retinitis pigmentosaBone marrow-derived mesenchymal stem cellsIntravitreal injection

Outcome Measures

Primary Outcomes (1)

  • Change from baseline in laser flare and cell measurements

    up to 12 months

Secondary Outcomes (1)

  • Change from baseline in visual function tests

    up to 12 months

Study Arms (1)

BM-MSC

EXPERIMENTAL

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

Other: BM-MSC

Interventions

BM-MSCOTHER

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

BM-MSC

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Siriraj Hospital Mahidol University

Bangkoknoi, Bangkok, 10700, Thailand

Location

Related Publications (5)

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

    PMID: 17113430BACKGROUND

  • 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.

    PMID: 15565292BACKGROUND

  • 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.

    PMID: 16896916RESULT

  • 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.

    PMID: 20169166RESULT

  • 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.

    PMID: 33422139DERIVED

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

Eye Diseases, HereditaryEye DiseasesRetinal DystrophiesRetinal DegenerationRetinal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • La-ongsri Atchaneeyasakul, MD

    Siriraj Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
OTHER
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

February 6, 2012

First Posted

February 10, 2012

Study Start

April 1, 2012

Primary Completion

July 1, 2020

Study Completion

July 1, 2020

Last Updated

September 8, 2021

Record last verified: 2021-08

Locations

TH(1)