NCT02709876

Autologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis Pigmentosa

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Stem Cells Arabia · Academic / Other
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Retinitis Pigmentosa

Interventions

Type	Name	Description
BIOLOGICAL	Stem Cell Transplantation	Bone marrow-derived CD34+, CD133+, CD271+ stem cells in 1.0 ml normal saline will be injected into the vitreous cavity.

Timeline

• Start date: 2014-04-01
• Primary completion: 2020-09-01
• Completion: 2021-03-01
• First posted: 2016-03-16
• Last updated: 2020-03-17

Locations

1 site across 1 country: Jordan