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

NCT03073733 | Completed Phase 2 Results FDA Drug

• 1 other identifier: JC-02
• Study Type: interventional
• Target: 84
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Retinitis Pigmentosa

Outcome Measures

Primary Outcomes (1)

• Best Corrected Visual Acuity (BCVA)

  Mean change in BCVA in study eye from baseline to month 12 as assessed by E-ETDRS in ITT population. A letter score is used to compare change over time, with a higher number of letters representing better visual function, and a lower number of letters representing worse visual function. For example, 85 letters is equivalent to 20/20 visual acuity and 5 letters is equivalent to 20/800 visual acuity. A change value is derived for each subject by taking the letter score at 12 months and subtracting the letter score at baseline. A mean of all change values is then calculated for each arm.

  12 months

Secondary Outcomes (5)

• Contrast Sensitivity (CS) at 1.0 CPD

  12 months

• Kinetic Visual Field (KVF)

  12 months

• Low Luminance Mobility Test (LLMT)

  12 months

• Low Vision Functional Questionnaire (Visual Ability)

  12 months

• Safety of Intravitreal Injection of Retinal Progenitor Cells (RPC)

  12 months

Study Arms (3)

Test (jCell injection) dose level 1

EXPERIMENTAL

single intravitreal injection of 3.0 x 10e6 human retinal progenitor cells into the eye with the poorest visual acuity or, if vision is comparable in both eyes, the non-dominant eye

Biological: human retinal progenitor cells

Sham treated Control

OTHER

a mock injection will be performed on the eye with the poorest vision in each Control subject (designated as the "study eye")

Other: Mock injection

test (jCell injection) dose level 2

EXPERIMENTAL

single intravitreal injection of 6.0 x 10e6 human retinal progenitor cells into the eye with the poorest visual acuity or, if vision is comparable in both eyes, the non-dominant eye

Biological: human retinal progenitor cells

Interventions

human retinal progenitor cells BIOLOGICAL

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

Also known as: jCell

Test (jCell injection) dose level 1; test (jCell injection) dose level 2

Mock injection OTHER

pressing the hub of a syringe with no needle against the eye to mimic intravitreal injection

Sham treated Control

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• jCyte, Inc: lead
• California Institute for Regenerative Medicine (CIRM): collaborator

Study Sites (3)

Gavin Herbert Eye Inst, Univ Cal Irvine

Irvine, California, 92697, United States

Location

Retina-Vitreous Associates Medical Group

Los Angeles, California, 90074, United States

Location

Ophthalmic Consultants of Boston

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Retinitis Pigmentosa

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Robert Beathard
• Organization: jCyte, Inc.

rob.beathard@jcyte.com

9492430687

Study Officials

• Mitul Mehta, MD

  UCI

  PRINCIPAL INVESTIGATOR

• David Liao, MD

  RVA

  PRINCIPAL INVESTIGATOR

• Anthony Joseph, MD

  OCB

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Masking Details: Subjects, their family members and clinical staff performing key efficacy assessments will be masked to the randomization assignment of subjects. Due to the nature of some safety assessments and the sham treatment, not all personnel can be masked.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 2, 2017
• First Posted: March 8, 2017
• Study Start: March 1, 2017
• Primary Completion: November 13, 2020
• Study Completion: November 13, 2020
• Last Updated: June 18, 2024
• Results First Posted: January 21, 2022

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)