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

Stem Cells Arabia

Status and phase: Unknown; Phase 2; Phase 1

Conditions: Retinitis Pigmentosa

Treatments: Biological: Stem Cell Transplantation

Study type: Interventional

Funder types: Other

Identifiers

NCT02709876

SCA-RP1

Details and patient eligibility

About

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.

Full 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.

Enrollment: 50 estimated patients

Sex: All

Ages: 18 to 55 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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50 participants in 1 patient group

Stem Cells

Experimental group

Description:

Intravitreal Injection of bone marrow derived CD34+, CD133+, CD271+ stem cells.

Treatment:

Biological: Stem Cell Transplantation