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

NCT ID: NCT02709876

Last Updated: 2020-03-17

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-04-30
• Study Completion Date: 2021-03-31

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.

Conditions

Retinitis Pigmentosa

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Stem Cells

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

Group Type: EXPERIMENTAL

Stem Cell Transplantation

Intervention Type: BIOLOGICAL

Bone marrow-derived CD34+, CD133+, CD271+ stem cells in 1.0 ml normal saline will be injected into the vitreous cavity.

Interventions

Stem Cell Transplantation

Bone marrow-derived CD34+, CD133+, CD271+ stem cells in 1.0 ml normal saline will be injected into the vitreous cavity.

Intervention Type: BIOLOGICAL

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Stem Cells Arabia

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Stem Cells Arabia

Amman, , Jordan

Countries

Jordan

Other Identifiers

SCA-RP1

Identifier Type: -

Identifier Source: org_study_id