Study of Subretinal Implantation of Human Embryonic Stem Cell-Derived RPE Cells in Advanced Dry AMD

NCT02590692 | Unknown Phase 1 DMC

• 1 other identifier: RPT-14-01
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 6

Brief Summary

The Phase I/IIa clinical trial is designed to assess the feasibility of delivery and safety of Human Embryonic Stem Cell-Derived RPE Cells on a parylene membrane (CPCB-RPE1) in patients with advanced, dry age-related macular degeneration. Primary Objective:

• To test the safety and tolerability of CPCB-RPE1 during and after subretinal implantation in patients with geographic atrophy with evidence of involvement of the central fovea. Secondary Objective:
• To assess visual acuity, visual field, and retinal function after CPCB-RPE1 implantation. Implanted and fellow eyes will be compared post-implantation to assess the ability of the implant to prevent disease progression. Exploratory Objectives:
• To assess the feasibility of measuring the change in area of geographic atrophy over time using spectral domain optical coherence tomography or fundus autofluorescence.

Conditions

Dry Macular Degeneration; Geographic Atrophy

Outcome Measures

Primary Outcomes (1)

• Frequency and Severity of Treatment-Related Adverse Events [Safety and Tolerability]

  Comparison of product, procedure and immunosuppression related adverse events in the implanted eye to those experienced in the non-treated eye

  1 year

Secondary Outcomes (3)

• Visual Acuity

  1 year

• Visual Field

  1 year

• Photoreceptor Electrical Responses

  1 year

Study Arms (1)

CPCB-RPE1 treatment

EXPERIMENTAL

Subretinal implantation of CPCB-RPE1 in dry AMD patients

Biological: CPCB-RPE1

Interventions

CPCB-RPE1 BIOLOGICAL

Patients will receive one CPCB-RPE1 implant of approximately 100,000 differentiated RPE cells attached to a small parylene membrane. The density of cells on the membrane represents the approximate density of RPE cells in the human eye. The membrane size was chosen to cover a substantial portion of the macular region of the retina.

CPCB-RPE1 treatment

Eligibility Criteria

• Age: 55 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients able to understand and willing to sign the informed consent
• Adult male or female patients with the age of 55 to 85 (inclusive) years who are not employees of the trial sites
• In sufficiently good health to reasonably expect survival for at least five years after treatment
• Clinical findings consistent with advanced dry AMD with evidence of one or more areas of ≥1.25 square millimeter of geographic atrophy involving the central fovea
• Geographic atrophy defined as attenuation or loss of RPE as observed by biomicroscopy, OCT, or FAF
• The best-corrected visual acuity (BCVA) of the eye to receive the implant will be equal or worse than 20/200 in the first half of the study patients and between 20/80 and 20/400 (inclusive) in the second half of the patients. The BCVA of the eye that is NOT to receive the implant will be better or equal to the eye that will receive the implant
• Medically suitable to undergo pars plana vitrectomy and the surgical implant procedure, including being able to position post-operatively and use post-operative medications as required
• Medically suitable for general anesthesia or monitored intravenous sedation, if needed
• Patients who are pseudophakic or aphakic in the study eye
• If designated as an organ donor, willing to forego live organ donation
• Willing to consent to the post-mortem removal of the implant from the treated eye for the sponsor's analysis. The patient may also elect to donate the implanted and fellow, untreated eye, for histological analysis.
• Able to understand the requirements of the study and willing and able to participate in long term follow up.

You may not qualify if:

• Presence of active or inactive choroidal neovascularization (CNV)
• Presence or history of retinal dystrophy, retinitis pigmentosa, chorioretinitis, central serous choroidopathy or any other inflammatory ocular disease except dry eye syndrome
• Presence or history of severe, end-stage corneal dystrophy
• History of steroid induced ocular hypertension or glaucoma
• Presence of moderate to severe glaucomatous optic neuropathy in the study eye, uncontrolled IOP, use of two or more topical agents to control intraocular pressure; history of glaucoma-filtering surgery
• Presence of moderate to severe non-proliferative diabetic retinopathy in the study eye
• Presence of any proliferative diabetic retinopathy in the study eye
• Presence of uncontrolled diabetes mellitus (HbA1c \> 8) at the time of screening
• History of retinal detachment or retinal detachment repair in the study eye other than peripheral retinal tears or holes treated exclusively with laser or cryotherapy
• Presence of any other sight-threatening ocular disease
• History of cognitive impairments or dementia which may impact the patient's ability to participate in the informed consent process and to appropriately complete evaluations
• History of any immunodeficiency
• Evidence of herpetic or other viral eye disease
• Any current use of immunosuppressive therapy other than intermittent or low dose corticosteroids
• Participation within previous 3 months in any clinical trial of a drug by ocular or systemic administration (within previous 18 months for sustained release products)

Sponsors & Collaborators

• Regenerative Patch Technologies, LLC: lead

Study Sites (6)

Retinal Consultants of Arizona LTD

Phoenix, Arizona, 85053, United States

Location

Retina-Vitreous Associates Medical Group

Beverly Hills, California, 90211, United States

Location

USC Keck School of Medicine / Eye Institute

Los Angeles, California, 90033, United States

Location

Southern California Desert Retina Consultants

Palm Desert, California, 92211, United States

Location

Orange County Retina Medical Group

Santa Ana, California, 92705, United States

Location

California Retina Consultants

Santa Barbara, California, 93103, United States

Location

MeSH Terms

Conditions

Geographic Atrophy

Condition Hierarchy (Ancestors)

Macular Degeneration; Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Jane Lebkowski, Ph.D.

  Regenerative Patch Technologies

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 23, 2015
• First Posted: October 29, 2015
• Study Start: February 16, 2016
• Primary Completion: July 1, 2019
• Study Completion: June 1, 2023
• Last Updated: May 29, 2020

Record last verified: 2020-05

Locations

US (6)