Phase 2 N=83 Randomized Double-blind Treatment

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

Retinitis Pigmentosa

Bottom Line

View on ClinicalTrials.gov: NCT03073733 ↗

Enrolled (actual)

Serious AEs

1.2%

Results posted

Jan 2022

Primary outcome: Primary: Best Corrected Visual Acuity (BCVA) — 3.9; 1.3; 2.6 Letters correct — p=0.582

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: human retinal progenitor cells (Biological); Mock injection (Other)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: jCyte, Inc
Primary completion: Nov 2020

Outcome Measures

Outcome	Result	p-value
PRIMARY Best Corrected Visual Acuity (BCVA)	3.9; 1.3; 2.6	0.582
SECONDARY Contrast Sensitivity (CS) at 1.0 CPD	0.4331; 1.9145; 0.1142	0.290
SECONDARY Kinetic Visual Field (KVF)	260.19; -44.02; 560.19	0.515
SECONDARY Low Luminance Mobility Test (LLMT)	0.6; -0.3; 0.2	0.160
SECONDARY Low Vision Functional Questionnaire (Visual Ability)	0.225; 0.146; 0.360	0.442
SECONDARY Safety of Intravitreal Injection of Retinal Progenitor Cells (RPC)	15; 20; 21; 7; 14; 13	—

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.

Eligibility Criteria

Inclusion Criteria

Clinical diagnosis of RP confirmed by ERG and willing to consent to mutation typing, if not already done Best corrected visual acuity (BCVA) 20/80 or worse and no worse than 20/800 Adequate organ function and negative infectious disease screen Female of childbearing potential must have negative pregnancy test and be willing to use medically accepted methods of contraception throughout the study

Exclusion Criteria

Eye disease other than RP that impairs visual function Pseudo-RP, cancer-associated retinopathies History of malignancy or other end-stage organ disease, or any chronic disease requiring continuous treatment with system steroids, anticoagulants or immunosuppressive agents Known allergy to penicillin or streptomycin Treatment with corticosteroids or any investigational or neuroprotectant therapy within 90 days of enrollment Cataract surgery within 3 months prior to enrollment

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03073733). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.