Overview

This trial is active, not recruiting.

Condition retinitis pigmentosa
Treatment stem cell transplantation
Phase phase 1/phase 2
Sponsor Stem Cells Arabia
Start date April 2014
End date September 2018
Trial size 50 participants
Trial identifier NCT02709876, SCA-RP1

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Intravitreal Injection of bone marrow derived CD34+, CD133+, CD271+ stem cells.
stem cell transplantation
Bone marrow-derived CD34+, CD133+, CD271+ stem cells in 1.0 ml normal saline will be injected into the vitreous cavity.

Primary Outcomes

Measure
ETDRS Visual acuity change
time frame: 12 months from baseline

Secondary Outcomes

Measure
Quality of Life: Questionnaire VFQ-25 (Visual Function Questionnaire-25)
time frame: 12 months from baseline
Color Vision: Ishihara Color Test
time frame: 12 months from baseline
Contrast sensitivity: CSV-1000E
time frame: 12 months from baseline

Eligibility Criteria

Male or female participants from 18 years up to 55 years old.

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

Additional Information

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.
Trial information was received from ClinicalTrials.gov and was last updated in October 2016.
Information provided to ClinicalTrials.gov by Stem Cells Arabia.