Trial record 2 of 2 for:    rpe advanced cell technology

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2015 by Regenerative Patch Technologies, LLC
Information provided by (Responsible Party):
Regenerative Patch Technologies, LLC Identifier:
First received: October 23, 2015
Last updated: November 12, 2015
Last verified: November 2015

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.

Condition Intervention Phase
Dry Macular Degeneration
Geographic Atrophy
Biological: CPCB-RPE1
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Official Title: A Phase I/IIa Safety Study of Subretinal Implantation of CPCB-RPE1 (Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells Seeded on a Polymeric Substrate) in Subjects With Advanced, Dry Age-Related Macular Degeneration (AMD)

Resource links provided by NLM:

Further study details as provided by Regenerative Patch Technologies, LLC:

Primary Outcome Measures:
  • Frequency and Severity of Treatment-Related Adverse Events [Safety and Tolerability] [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Comparison of product, procedure and immunosuppression related adverse events in the implanted eye to those experienced in the non-treated eye

Secondary Outcome Measures:
  • Visual Acuity [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Comparison of VA changes in the treated eye versus baseline and versus the non-treated eye

  • Visual Field [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Comparison of visual field changes in the treated eye versus baseline and versus the non-treated eye

  • Photoreceptor Electrical Responses [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Comparison of multifocal electroretinogram changes in the treated eye versus baseline and versus the non-treated eye

Estimated Enrollment: 20
Study Start Date: October 2015
Estimated Study Completion Date: September 2022
Estimated Primary Completion Date: September 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: CPCB-RPE1 treatment
Subretinal implantation of CPCB-RPE1 in dry AMD patients
Biological: CPCB-RPE1
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.

Detailed Description:

The study will include two cohorts, each of 10 patients. For the first cohort, the study population will be patients with advanced, dry AMD with evidence of significant geographic atrophy involving the fovea. These patients will have significant central vision loss with best-corrected visual acuity (BCVA) of the eye to be implanted of BCVA ≤ 20/400. Each of these patients will have substantial RPE and photoreceptor loss. Patients will be screened for overall health status to minimize risks associated with retinal surgery and any subsequent immunosuppression.

As the safety and tolerability of CPCB-RPE1 is demonstrated in the first cohort, patients with less advanced disease will be recruited into a second cohort in this Phase I/IIa clinical trial. In this cohort patients will have significant central vision loss with BCVA of the eye to be implanted of ≤ 20/100 (but better than 20/400) with comparably less damage to the RPE/photoreceptor complex than Cohort 1. These patients will be screened in the same manner for overall health status to minimize risks associated with retinal surgery and any subsequent immunosuppression. Assessments of visual function will be the same as in Cohort 1.


Ages Eligible for Study:   55 Years to 85 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Patients able to understand and willing to sign the informed consent
  2. Adult male or female patients with the age of 55 to 85 years who are not employees of the trial sites
  3. The patient should be in sufficiently good health to reasonably expect survival for at least five years after treatment
  4. 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
  5. Geographic atrophy defined as attenuation or loss of RPE as observed by biomicroscopy, OCT, or FAF
  6. The best-corrected visual acuity (BCVA) of the eye to receive the implant will be equal or worse than 20/400 in the first half and no better than 20/100 but better than 20/400 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
  7. 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
  8. Medically suitable for general anesthesia or monitored intravenous sedation, if needed
  9. If phakic, and when indicated, medically capable and willing to undergo cataract surgery post implantation
  10. If designated as an organ donor, willing to forego live organ donation
  11. Willing to donate the treated eye post-mortem to the sponsor for histological analysis. The patient may also elect to donate the fellow, untreated eye for analysis
  12. Patients able to understand the requirements of the study and willing and able to participate in long term follow up.

Exclusion Criteria:

  1. Presence of active or inactive choroidal neovascularization (CNV)
  2. Presence or history of retinal dystrophy, retinitis pigmentosa, chorioretinitis, central serous choroidopathy or any other inflammatory ocular disease except dry eye syndrome
  3. Presence or history of severe, end-stage corneal dystrophy
  4. 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 trabeculectomy, Baerveldt or Ahmed tube shunts
  5. Presence of moderate to severe non-proliferative diabetic retinopathy in the study eye
  6. Presence of any proliferative diabetic retinopathy in the study eye
  7. Presence of uncontrolled diabetes mellitus (HbA1c > 7) at the time of screening
  8. 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
  9. Presence of any other sight-threatening ocular disease
  10. 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
  11. History of any immunodeficiency
  12. Evidence of herpetic or other viral eye disease
  13. Any current use of immunosuppressive therapy other than intermittent or low dose corticosteroids
  14. Participation within previous 3 months in any clinical trial of a drug by ocular or systemic administration
  15. Axial myopia of greater than -8 diopters in the eye that is to be implanted
  16. Axial length greater than 28 mm in the eye that is to be implanted
  17. History of malignancy (with the exception of successfully treated [excised] basal cell carcinoma [skin cancer] or successfully treated squamous cell carcinoma of the skin)
  18. History of myocardial infarction in previous 12 months
  19. Alanine transaminase/aspartate aminotransferase (ALT/AST) >3.0 times the upper limit of normal or any known liver disease
  20. Renal insufficiency, as defined by estimated creatinine clearance of < 45 ml/min
  21. A positive test for HIV, or Hepatitis B, or Hepatitis C
  22. A hemoglobin concentration of less than 10 gm/dl, a platelet count of less than 100K/mcL or an absolute neutrophil count of less than 1000/mcL at study entry
  23. Ocular lens removal in the study eye within previous 6 weeks
  24. Any other ocular surgery in the study eye in the previous 3 months
  25. If female, pregnancy, the wish to become pregnant, or lactation
  26. Any other medical condition, which, in the Investigator's judgment, will interfere with the patient's ability to comply with the protocol, compromises patient safety, or interferes with the interpretation of the study results
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02590692

United States, California
Retina-Vitreous Associates Medical Group Recruiting
Beverly Hills, California, United States, 90211
Contact: Daniel Bandary    310-289-2478 ext 3   
Principal Investigator: Firas Rahhal, MD         
USC Keck School of Medicine / Eye Institute Recruiting
Los Angeles, California, United States, 90033
Contact: Yoon H Kim, BSc    323-865-6935   
Contact: Nelson J Aguilar, BA    323-442-6490   
Principal Investigator: Amir H Kashani, MD PhD         
Sponsors and Collaborators
Regenerative Patch Technologies, LLC
Study Director: Peter Kondor, Ph.D. Regenerative Patch Technologies
  More Information

No publications provided

Responsible Party: Regenerative Patch Technologies, LLC Identifier: NCT02590692     History of Changes
Other Study ID Numbers: RPT-14-01
Study First Received: October 23, 2015
Last Updated: November 12, 2015
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Geographic Atrophy
Macular Degeneration
Eye Diseases
Retinal Degeneration
Retinal Diseases processed this record on November 25, 2015