Clinical trial NCT04925687: Phase 1 Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis Pigmentosa
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Retinitis Pigmentosa
Keywords: retinitis pigmentosa
stem cell
retinal degeneration
intravitreal
Lead sponsor: University of California, Davis
Sponsor class: OTHER
Collaborators: name: Cures Within Reach
name: The Retina Society
First posted: 2021-06-14
Enrollment: 4
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Safety of intravitreal injection of autologous CD34+ cells
timeFrame: 6 months
measure: Feasibility of intravitreal injection of autologous CD34+ cells
timeFrame: Baseline
Secondary outcomes:
measure: Best corrected visual acuity
timeFrame: 6 months
measure: Best corrected visual acuity
timeFrame: 1 month
measure: Visual Field
timeFrame: 6 months
measure: Visual Field
timeFrame: 1 month
measure: Electroretinography
timeFrame: 6 months
measure: Electroretinography
timeFrame: 1 month
measure: National Eye Institute Vision Questionnaire
timeFrame: 6 months
measure: National Eye Institute Vision Questionnaire
timeFrame: 1 month
measure: Microperimetry
timeFrame: 6 months
measure: Microperimetry
timeFrame: 6 months
measure: Microperimetry
timeFrame: 1 month
measure: Microperimetry
timeFrame: 1 month
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: University of California Davis
state: California
Study officials:
affiliation: University of California, Davis
name: Susanna Park, MD PhD
role: PRINCIPAL_INVESTIGATOR
References: