Phase 1 Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis Pigmentosa (BMSCRP1)

University of California (UC) Davis

Status and phase: Completed; Phase 1

Conditions: Retinitis Pigmentosa

Treatments: Biological: Intravitreal autologous CD34+ cells

Study type: Interventional

Funder types: Other

Identifiers

NCT04925687

1743714

Details and patient eligibility

About

In this Phase 1 open-labeled prospective study, one eye of each participant with vision loss from retinitis pigmentosa will be administered intravitreal injection of autologous CD34+ stem cells harvested from bone marrow. Each participant will be examined serially for 6 months after study injection to determine safety and feasibility of this intervention.

Enrollment: 4 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Age >18 years of age 2. Visual Acuity at Presentation of 20/100 to Counting Fingers or constriction of peripheral visual field 360 degrees to <10 degrees by perimetry if BCVA is better than 20/100, duration of vision loss > 3 months 3. Vision loss due to retinitis pigmentosa 4. The study eye has the worse visual acuity or peripheral constriction. 14. Able and willing to sign consent 15. Able to keep follow-up appointments for at least 6 months

Exclusion criteria

1. Other concurrent optic nerve or retinal disease in study eye affecting vision

2. History of active retinal or choroidal neovascularization or macular edema in study eye requiring on-going intravitreal or periocular therapy in study eye

3. Active eye or systemic infection

4. Significant media opacity in the study eye precluding view of the fundus for examination, photography or optical coherence tomography (OCT)

5. Other cause for vision loss in the study eye 10. History of coagulopathy or other hematologic abnormality 11. Use of coumadin within 3 days of enrollment 12. Concurrent immunosuppressive therapy 13. History of allergy to fluorescein dye

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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4 participants in 1 patient group

Intravitreal autologous CD34+ cells

Experimental group

Description:

Intravitreal injection of autologous CD34+ cells harvested from bone marrow under GMP conditions

Treatment:

Biological: Intravitreal autologous CD34+ cells