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

Phase 1

Completed

• Conditions: Retinitis Pigmentosa
• Interventions: Biological: Intravitreal autologous CD34+ cells

• Registration Number: NCT04925687
• Lead Sponsor: University of California, Davis

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2021-06-01
• Study First Submitted: 2021-06-02
• Study First Submitted QC: 2021-06-07
• Study First Posted: 2021-06-14
• Primary Completion: 2023-11-01
• Study Completion: 2023-11-01
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-06
• Last Update Posted: 2024-05-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 4

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Safety of intravitreal injection of autologous CD34+ cells	6 months	Number and severity of Ocular and systemic adverse events
Feasibility of intravitreal injection of autologous CD34+ cells	Baseline	Number of CD34+ cells harvested for intravitreal injection

Secondary Outcome Measures

Name	Time	Method
Visual Field	1 month	Change in visual field area in study eye from baseline
National Eye Institute Vision Questionnaire	1 month	Change in Vision Questionnaire compared to baseline
Best corrected visual acuity	1 month	Change in number of letters read in study eye from baseline
Microperimetry	1 month	Change in percent reduced sensitivity from baseline
Electroretinography	1 month	Change in a and b wave amplitude in study eye from baseline

Trial Locations

Locations (1)

University of California Davis; 🇺🇸 Sacramento, California, United States