# The Phase I/II Randomized, Prospective, Double-blinded, Sham-controlled Cross-over Study of Intravitreal Autologous Bone Marrow CD34+ Stem Cell Therapy for Retinal Vein Occlusion

> **NIH NIH UG1** · UNIVERSITY OF CALIFORNIA AT DAVIS · 2020 · $472,056

## Abstract

Project Summary:
 The goal of this phase I/II prospective, randomized, sham-controlled, double-blinded
clinical trial is to determine whether intravitreal autologous CD34+ cell therapy is safe, feasible
and potentially beneficial in eyes with vision loss from retinal vein occlusion (RVO). RVO is a
leading retinal vascular cause of vision loss in the elderly. CD34+ cells in human bone marrow
are mobilized into the circulation in response to tissue ischemia for tissue revascularization and
repair. Since local delivery of CD34+ cells benefits ischemic tissue, intravitreal delivery of
CD34+ cells may benefit vision and retinal ischemia in eyes with RVO. A pilot clinical trial has
shown no major safety or feasibility concerns using intravitreal autologous CD34+ bone marrow
cells. In this expanded phase I/II study, 15 subjects (15 eyes) with persistent vision loss from
RVO will be enrolled and follow for 2 years. Total study duration will be 60 months. This single-
center study will be at the University of California Davis. The Coordinating Center will be
Emmes Corporation. The Study Chair/Principal Investigator will be Dr. Susanna Park. The study
will be conducted under an IND cleared from FDA. A Data and Safety Monitoring Committee will
oversee the study. Subjects will be randomized at enrollment 2:1 to cell therapy or
sham/deferred cell therapy. The cell therapy involves bone marrow aspiration, isolation of
CD34+ cells from the aspirate under Good Manufacturing Practice conditions, and intravitreal
injection of isolated CD34+ cells. Sham therapy involves sham bone marrow aspiration and
intravitreal injection without penetrating the bone or eye. The subject, examining
ophthalmologist and visual acuity examiner will remain masked to study treatment
randomization till month six. Thereafter, the study will be unmasked and the sham-treated
subjects will undergo bone marrow aspiration and cell therapy. A comprehensive eye
examination with ETDRS best-corrected visual acuity, optical coherence tomography, auto-
fluorescence, fundus photography, fluorescein angiography, microperimetry and
electroretinography will be performed at baseline and serially. The primary outcome measures
will be the incidence and severity of ocular and systemic adverse events associated with cell
therapy and the number of CD34+ cells isolated for injection. The secondary outcome measures
will be the changes in visual function, retinal perfusion and morphology following cell therapy.
The long-term objective is to determine whether intravitreal autologous CD34+ cell therapy can
minimize or reverse vision loss associated with retinal ischemia without compromising safety.

## Key facts

- **NIH application ID:** 9948653
- **Project number:** 5UG1EY026876-03
- **Recipient organization:** UNIVERSITY OF CALIFORNIA AT DAVIS
- **Principal Investigator:** SUSANNA S PARK
- **Activity code:** UG1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $472,056
- **Award type:** 5
- **Project period:** 2018-06-15 → 2023-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9948653

## Citation

> US National Institutes of Health, RePORTER application 9948653, The Phase I/II Randomized, Prospective, Double-blinded, Sham-controlled Cross-over Study of Intravitreal Autologous Bone Marrow CD34+ Stem Cell Therapy for Retinal Vein Occlusion (5UG1EY026876-03). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/9948653. Licensed CC0.

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