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

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

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.

Study Overview

• Status: Completed
• Conditions: Retinitis Pigmentosa
• Intervention / Treatment: Biological: Intravitreal autologous CD34+ cells

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Sacramento, California, United States, 95817
      • University of California Davis

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Intravitreal autologous CD34+ cells; Intravitreal injection of autologous CD34+ cells harvested from bone marrow under GMP conditions	Biological: Intravitreal autologous CD34+ cells; Autologous CD34+ cells harvested from bone marrow injected intravitreal

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of intravitreal injection of autologous CD34+ cells	Number and severity of Ocular and systemic adverse events	6 months
Feasibility of intravitreal injection of autologous CD34+ cells	Number of CD34+ cells harvested for intravitreal injection	Baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Best corrected visual acuity	Change in number of letters read in study eye from baseline	6 months
Best corrected visual acuity	Change in number of letters read in study eye from baseline	1 month
Visual Field	Change in visual field area in study eye from baseline	6 months
Visual Field	Change in visual field area in study eye from baseline	1 month
Electroretinography	Change in a and b wave amplitude in study eye from baseline	6 months
Electroretinography	Change in a and b wave amplitude in study eye from baseline	1 month
National Eye Institute Vision Questionnaire	Change in Vision Questionnaire compared to baseline	6 months
National Eye Institute Vision Questionnaire	Change in Vision Questionnaire compared to baseline	1 month
Microperimetry	Change in average threshold from baseline	6 months
Microperimetry	Change percent reduced sensitivity from baseline	6 months
Microperimetry	Change in average threshold from baseline	1 month
Microperimetry	Change in percent reduced sensitivity from baseline	1 month

Collaborators and Investigators

• Sponsor: University of California, Davis
• Collaborators: Cures Within Reach; Retina Society
• Investigators: Principal Investigator: Susanna Park, MD PhD, University of California, Davis

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2021
• Primary Completion (Actual): November 1, 2023
• Study Completion (Actual): November 1, 2023

Study Registration Dates

• First Submitted: June 2, 2021
• First Submitted That Met QC Criteria: June 7, 2021
• First Posted (Actual): June 14, 2021

Study Record Updates

• Last Update Posted (Actual): May 8, 2024
• Last Update Submitted That Met QC Criteria: May 6, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Keywords: intravitreal; retinal degeneration; stem cell; retinitis pigmentosa
• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Eye Diseases, Hereditary; Retinal Dystrophies; Retinitis; Retinitis Pigmentosa
• Other Study ID Numbers: 1743714-2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No