BMSCRP1: Pilot Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis Pigmentosa

• Sponsor: University of California, Davis (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT04925687
• Collaborator: Cures Within Reach (Other)
• Enrollment: 4
• Location: 1
• Arm: 1
• Anticipated Duration (Months): 12.9
• Patients Per Site Per Month: 0.3

Study Details

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
• Anticipated Enrollment: 4 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Pilot 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: Jun 1, 2021
• Anticipated Primary Completion Date: Jun 30, 2022
• Anticipated Study Completion Date: Jun 30, 2022

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 [6 months]

   Number and severity of Ocular and systemic adverse events

2. Feasibility of intravitreal injection of autologous CD34+ cells [Baseline]

   Number of CD34+ cells harvested for intravitreal injection

Secondary Outcome Measures

1. Best corrected visual acuity [6 months]

   Change in number of letters read in study eye from baseline

2. Best corrected visual acuity [1 month]

   Change in number of letters read in study eye from baseline

3. Visual Field [6 months]

   Change in visual field area in study eye from baseline

4. Visual Field [1 month]

   Change in visual field area in study eye from baseline

5. Electroretinography [6 months]

   Change in a and b wave amplitude in study eye from baseline

6. Electroretinography [1 month]

   Change in a and b wave amplitude in study eye from baseline

7. National Eye Institute Vision Questionnaire [6 months]

   Change in Vision Questionnaire compared to baseline

8. National Eye Institute Vision Questionnaire [1 month]

   Change in Vision Questionnaire compared to baseline

9. Microperimetry [6 months]

   Change in average threshold from baseline

10. Microperimetry [6 months]

    Change percent reduced sensitivity from baseline

11. Microperimetry [1 month]

    Change in average threshold from baseline

12. Microperimetry [1 month]

    Change in percent reduced sensitivity from baseline

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of California Davis	Sacramento	California	United States	95817

Sponsors and Collaborators

• University of California, Davis
• Cures Within Reach

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Susanna Park, MD, PhD, Professor, University of California, Davis
• ClinicalTrials.gov Identifier: NCT04925687
• Other Study ID Numbers: 1743714-2
• First Posted: Jun 14, 2021
• Last Update Posted: Aug 23, 2021
• Last Verified: Aug 1, 2021

• Individual Participant Data (IPD) Sharing Statement: No
• 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, Professor, University of California, Davis: retinitis pigmentosa; stem cell; retinal degeneration; intravitreal
• Additional relevant MeSH terms: Retinitis; Retinitis Pigmentosa

Study Results

No Results Posted as of Aug 23, 2021