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
  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:


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:
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 April 16, 2014