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

• ClinicalTrials.gov Identifier: NCT04925687
• Recruitment Status: Active, not recruiting
• First Posted: June 14, 2021
• Last Update Posted: April 21, 2023
• Sponsor: University of California, Davis
• Collaborators: Cures Within Reach; Retina Society
• Information provided by (Responsible Party): Susanna Park, MD, PhD, University of California, Davis

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Retinitis Pigmentosa	Biological: Intravitreal autologous CD34+ cells	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 4 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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
• Actual Study Start Date: June 1, 2021
• Estimated Primary Completion Date: November 30, 2023
• Estimated Study Completion Date: November 30, 2023

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. Safety of intravitreal injection of autologous CD34+ cells [ Time Frame: 6 months ]
   Number and severity of Ocular and systemic adverse events
2. Feasibility of intravitreal injection of autologous CD34+ cells [ Time Frame: Baseline ]
   Number of CD34+ cells harvested for intravitreal injection

Secondary Outcome Measures :

1. Best corrected visual acuity [ Time Frame: 6 months ]
   Change in number of letters read in study eye from baseline
2. Best corrected visual acuity [ Time Frame: 1 month ]
   Change in number of letters read in study eye from baseline
3. Visual Field [ Time Frame: 6 months ]
   Change in visual field area in study eye from baseline
4. Visual Field [ Time Frame: 1 month ]
   Change in visual field area in study eye from baseline
5. Electroretinography [ Time Frame: 6 months ]
   Change in a and b wave amplitude in study eye from baseline
6. Electroretinography [ Time Frame: 1 month ]
   Change in a and b wave amplitude in study eye from baseline
7. National Eye Institute Vision Questionnaire [ Time Frame: 6 months ]
   Change in Vision Questionnaire compared to baseline
8. National Eye Institute Vision Questionnaire [ Time Frame: 1 month ]
   Change in Vision Questionnaire compared to baseline
9. Microperimetry [ Time Frame: 6 months ]
   Change in average threshold from baseline
10. Microperimetry [ Time Frame: 6 months ]
    Change percent reduced sensitivity from baseline
11. Microperimetry [ Time Frame: 1 month ]
    Change in average threshold from baseline
12. Microperimetry [ Time Frame: 1 month ]
    Change in percent reduced sensitivity from baseline

Eligibility Criteria

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

Criteria

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

Contacts and Locations

Locations

United States, California

University of California Davis

Sacramento, California, United States, 95817

Sponsors and Collaborators

University of California, Davis

Cures Within Reach

Retina Society

Investigators

• Principal Investigator: Susanna Park, MD PhD; University of California, Davis

More Information

• Responsible Party: Susanna Park, MD, PhD, Professor, University of California, Davis
• ClinicalTrials.gov Identifier: NCT04925687
• Other Study ID Numbers: 1743714-2
• First Posted: June 14, 2021
• Last Update Posted: April 21, 2023
• Last Verified: September 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Susanna Park, MD, PhD, University of California, Davis:

retinitis pigmentosa; stem cell; retinal degeneration; intravitreal

Additional relevant MeSH terms:

Retinitis; Retinitis Pigmentosa; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Retinal Dystrophies; Retinal Degeneration; Genetic Diseases, Inborn