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

NCT04925687 | Completed Phase 1 FDA Drug

• 1 other identifier: 1743714
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

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.

Conditions

Retinitis Pigmentosa

Keywords

retinitis pigmentosa; stem cell; retinal degeneration; intravitreal

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (12)

• Best corrected visual acuity

  6 months

• Best corrected visual acuity

  1 month

• Visual Field

  6 months

• Visual Field

  1 month

• Electroretinography

  6 months

Study Arms (1)

Intravitreal autologous CD34+ cells

EXPERIMENTAL

Intravitreal injection of autologous CD34+ cells harvested from bone marrow under GMP conditions

Biological: Intravitreal autologous CD34+ cells

Interventions

Intravitreal autologous CD34+ cells BIOLOGICAL

Autologous CD34+ cells harvested from bone marrow injected intravitreal

Intravitreal autologous CD34+ cells

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. 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

You may not qualify if:

• Other concurrent optic nerve or retinal disease in study eye affecting vision
• History of active retinal or choroidal neovascularization or macular edema in study eye requiring on-going intravitreal or periocular therapy in study eye
• Active eye or systemic infection
• Significant media opacity in the study eye precluding view of the fundus for examination, photography or optical coherence tomography (OCT)
• \. 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

Sponsors & Collaborators

• University of California, Davis: lead
• Cures Within Reach: collaborator
• The Retina Society: collaborator

Study Sites (1)

University of California Davis

Sacramento, California, 95817, United States

Location

MeSH Terms

Conditions

Retinitis Pigmentosa; Retinal Degeneration

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Retinal Dystrophies; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Susanna Park, MD PhD

  University of California, Davis

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 2, 2021
• First Posted: June 14, 2021
• Study Start: June 1, 2021
• Primary Completion: November 1, 2023
• Study Completion: November 1, 2023
• Last Updated: September 23, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)