- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02709876
Autologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis Pigmentosa
March 15, 2020 updated by: Stem Cells Arabia
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.
Study Overview
Status
Unknown
Conditions
Intervention / Treatment
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.
Study Type
Interventional
Enrollment (Anticipated)
50
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Amman, Jordan, 11953
- Stem Cells Arabia
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 55 years (Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Stem Cells
Intravitreal Injection of bone marrow derived CD34+, CD133+, CD271+ stem cells.
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Bone marrow-derived CD34+, CD133+, CD271+ stem cells in 1.0 ml normal saline will be injected into the vitreous cavity.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
ETDRS Visual acuity change
Time Frame: 12 months from baseline
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12 months from baseline
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Quality of Life: Questionnaire VFQ-25 (Visual Function Questionnaire-25)
Time Frame: 12 months from baseline
|
12 months from baseline
|
Color Vision: Ishihara Color Test
Time Frame: 12 months from baseline
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12 months from baseline
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Contrast sensitivity: CSV-1000E
Time Frame: 12 months from baseline
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12 months from baseline
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
April 1, 2014
Primary Completion (Anticipated)
September 1, 2020
Study Completion (Anticipated)
March 1, 2021
Study Registration Dates
First Submitted
February 26, 2016
First Submitted That Met QC Criteria
March 10, 2016
First Posted (Estimate)
March 16, 2016
Study Record Updates
Last Update Posted (Actual)
March 17, 2020
Last Update Submitted That Met QC Criteria
March 15, 2020
Last Verified
March 1, 2020
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- SCA-RP1
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Yes
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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-
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