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

Retinitis Pigmentosa Clinical Trial

— BMSCRP1  

Official title:

Phase 1 Study of the Feasibility and Safety of Intravitreal Autologous Bone Marrow CD34+ Stem Cell Therapy for Eyes With Vision Loss From Retinitis Pigmentosa

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04925687
• Other study ID #: 1743714-2
• Status: Completed
• Phase: Phase 1
• Start date: June 1, 2021
• Est. completion date: November 1, 2023

Study information

• Verified date: May 2024
• Source: University of California, Davis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: November 1, 2023
• Est. primary completion date: November 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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)

9. 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

Related Conditions & MeSH terms

• Retinitis
• Retinitis Pigmentosa

Intervention

Biological:
• Intravitreal autologous CD34+ cells
Autologous CD34+ cells harvested from bone marrow injected intravitreal

Locations

Country	Name	City	State
United States	University of California Davis	Sacramento	California

Sponsors (3)

Lead Sponsor	Collaborator
University of California, Davis	Cures Within Reach, Retina Society

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety of intravitreal injection of autologous CD34+ cells	Number and severity of Ocular and systemic adverse events	6 months
Primary	Feasibility of intravitreal injection of autologous CD34+ cells	Number of CD34+ cells harvested for intravitreal injection	Baseline
Secondary	Best corrected visual acuity	Change in number of letters read in study eye from baseline	6 months
Secondary	Best corrected visual acuity	Change in number of letters read in study eye from baseline	1 month
Secondary	Visual Field	Change in visual field area in study eye from baseline	6 months
Secondary	Visual Field	Change in visual field area in study eye from baseline	1 month
Secondary	Electroretinography	Change in a and b wave amplitude in study eye from baseline	6 months
Secondary	Electroretinography	Change in a and b wave amplitude in study eye from baseline	1 month
Secondary	National Eye Institute Vision Questionnaire	Change in Vision Questionnaire compared to baseline	6 months
Secondary	National Eye Institute Vision Questionnaire	Change in Vision Questionnaire compared to baseline	1 month
Secondary	Microperimetry	Change in average threshold from baseline	6 months
Secondary	Microperimetry	Change percent reduced sensitivity from baseline	6 months
Secondary	Microperimetry	Change in average threshold from baseline	1 month
Secondary	Microperimetry	Change in percent reduced sensitivity from baseline	1 month