Back to resultsAutologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis Pigmentosa
Primary Purpose
Retinitis Pigmentosa
Status
Unknown status
Phase
Phase 1
Locations
Jordan
Study Type
Interventional
Intervention
Stem Cell Transplantation
Sponsored by
About this trial
This is an interventional treatment trial for Retinitis Pigmentosa
Eligibility Criteria
Inclusion Criteria:
- Retinitis pigmentosa patients diagnosed by ophthalmologists
- Visual acuity (measured with ETDRS) less than or equal to 20/70 and visual field below 30° central in both eyes.
- Best corrected visual acuity less than 6/120 by Snellen visual acuity chart
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
Sites / Locations
- Stem Cells Arabia
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Stem Cells
Arm Description
Intravitreal Injection of bone marrow derived CD34+, CD133+, CD271+ stem cells.
Outcomes
Primary Outcome Measures
ETDRS Visual acuity change
Secondary Outcome Measures
Quality of Life: Questionnaire VFQ-25 (Visual Function Questionnaire-25)
Color Vision: Ishihara Color Test
Contrast sensitivity: CSV-1000E
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02709876
Brief Title
Autologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis Pigmentosa
Study Type
Interventional
2. Study Status
Record Verification Date
March 2020
Overall Recruitment Status
Unknown status
Study Start Date
April 2014 (undefined)
Primary Completion Date
September 2020 (Anticipated)
Study Completion Date
March 2021 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Stem Cells Arabia
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
A single arm, single center trial to evaluate the safety and efficacy of autologous purified populations of bone-marrow derived stem cells in patients with Retinitis Pigmentosa (BM-SCs) through a 48 month follow up period.
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. This is a single arm, single center trial to assess the safety and efficacy of purified adult autologous bone marrow derived CD34+, CD133+, and CD271+ stem cells through a 48 month follow-up period. The combination of these three cell types was based on their diverse potentialities to differentiate into specific functional cell types to regenerate damaged retinal tissue, and the availability of clinical-grade purification system (CliniMACS) and microbeads to purify the target cell populations in clinically-approved methods.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Retinitis Pigmentosa
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Stem Cells
Arm Type
Experimental
Arm Description
Intravitreal Injection of bone marrow derived CD34+, CD133+, CD271+ stem cells.
Intervention Type
Biological
Intervention Name(s)
Stem Cell Transplantation
Intervention Description
Bone marrow-derived CD34+, CD133+, CD271+ stem cells in 1.0 ml normal saline will be injected into the vitreous cavity.
Primary Outcome Measure Information:
Title
ETDRS Visual acuity change
Time Frame
12 months from baseline
Secondary Outcome Measure Information:
Title
Quality of Life: Questionnaire VFQ-25 (Visual Function Questionnaire-25)
Time Frame
12 months from baseline
Title
Color Vision: Ishihara Color Test
Time Frame
12 months from baseline
Title
Contrast sensitivity: CSV-1000E
Time Frame
12 months from baseline
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
55 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Retinitis pigmentosa patients diagnosed by ophthalmologists
Visual acuity (measured with ETDRS) less than or equal to 20/70 and visual field below 30° central in both eyes.
Best corrected visual acuity less than 6/120 by Snellen visual acuity chart
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
Facility Information:
Facility Name
Stem Cells Arabia
City
Amman
ZIP/Postal Code
11953
Country
Jordan
12. IPD Sharing Statement
Plan to Share IPD
Yes
Learn more about this trial
Autologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis Pigmentosa
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