NCT03073733

Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa

A Prospective, Multicenter, Randomized, Study of the Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 84 (actual)
• Sponsor: jCyte, Inc · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study evaluates the changes in visual function at 12 months following a single injection of human retinal progenitor cells compared to sham treated controls in a cohort of adult subjects with RP.

Detailed description

There is no effective treatment for RP; once photoreceptors are lost, they do not regenerate. The rate of deterioration of vision varies from person to person, with most people with RP legally blind by age 40. Preclinical studies demonstrated that transplantation of retinal progenitor cells into the eye can result in both photoreceptor replacement and significant slowing of host photoreceptor loss. Thus, the primary goal of this therapy is to preserve, and potentially improve, vision by intervening in the disease at a time when dystrophic host photoreceptors can be protected and reactivated. Based on the demonstration of acceptable safety and tolerability in a phase 1/2a study, this phase 2b study is designed as a controlled comparison of the changes in visual function and functional vision in subjects who receive a single jCell injection in comparison to a comparable sham-treated control group of subjects with RP.

Conditions

• Retinitis Pigmentosa

Interventions

Type	Name	Description
BIOLOGICAL	human retinal progenitor cells	live suspension of 3.0 or 6.0 x 10e6 human retinal progenitor cells (hRPC) suspended in clinical grade medium injected intravitreally under local anesthesia
OTHER	Mock injection	pressing the hub of a syringe with no needle against the eye to mimic intravitreal injection

Timeline

• Start date: 2017-03-01
• Primary completion: 2020-11-13
• Completion: 2020-11-13
• First posted: 2017-03-08
• Last updated: 2024-06-18
• Results posted: 2022-01-21

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study