Clinical trial NCT03073733: Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis Pigmentosa
Official title: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Retinitis Pigmentosa
Lead sponsor: jCyte, Inc
Sponsor class: INDUSTRY
Collaborators: name: California Institute for Regenerative Medicine (CIRM)
First posted: 2017-03-08
Enrollment: 84
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

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

Primary outcomes:
measure: Best Corrected Visual Acuity (BCVA)
timeFrame: 12 months
Secondary outcomes:
measure: Contrast Sensitivity (CS) at 1.0 CPD
timeFrame: 12 months
measure: Kinetic Visual Field (KVF)
timeFrame: 12 months
measure: Low Luminance Mobility Test (LLMT)
timeFrame: 12 months
measure: Low Vision Functional Questionnaire (Visual Ability)
timeFrame: 12 months
measure: Safety of Intravitreal Injection of Retinal Progenitor Cells (RPC)
timeFrame: 12 months
Other outcomes:
Locations:
city: Irvine
country: United States
facility: Gavin Herbert Eye Inst, Univ Cal Irvine
state: California
city: Los Angeles
country: United States
facility: Retina-Vitreous Associates Medical Group
state: California
city: Boston
country: United States
facility: Ophthalmic Consultants of Boston
state: Massachusetts
Study officials:
affiliation: UCI
name: Mitul Mehta, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: RVA
name: David Liao, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: OCB
name: Anthony Joseph, MD
role: PRINCIPAL_INVESTIGATOR
References: