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Dry Macular DegenerationPhase 1/2NCT02590692

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

A Phase 1/2 study testing CPCB-RPE1 in people with Dry Macular Degeneration and Geographic Atrophy, run by Regenerative Patch Technologies, LLC at 6 sites in United States.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 55 to 85 years
Conditions the study is for
Dry Macular Degeneration, Geographic Atrophy
What is being tested?
CPCB-RPE1
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
6 sitesUnited States
Study dates
16 Feb 2016to Jun 2023
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 55 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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.

Who cannot join

  • 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
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  • 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)
  • Axial myopia of greater than -8 diopters in the eye that is to be implanted
  • Axial length greater than 28 mm in the eye that is to be implanted
  • History of malignancy within the past 5 years (with the exception of successfully treated [excised] basal cell carcinoma [skin cancer] or successfully treated squamous cell carcinoma of the skin)
  • History of myocardial infarction in previous 12 months
  • Alanine transaminase/aspartate aminotransferase (ALT/AST) >3.0 times the upper limit of normal or any known liver disease
  • Renal insufficiency, as defined by estimated creatinine clearance of < 45 ml/min
  • A positive (or "reactive") test for HIV, or Hepatitis B, or Hepatitis C
  • A hemoglobin concentration of less than 10 gm/dl, a platelet count of less than 100K/µL or an absolute neutrophil count of less than 1000/µL at study entry
  • Ocular lens removal within the previous 6 weeks in either eye
  • Any other ocular surgery in the study eye in the previous 3 months
  • If female, pregnancy, the wish to become pregnant, or lactation
  • 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

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • Retina-Vitreous Associates Medical Group

    Beverly Hills, California, 90211

  • USC Keck School of Medicine / Eye Institute

    Los Angeles, California, 90033

  • Southern California Desert Retina Consultants

    Palm Desert, California, 92211

  • Retinal Consultants of Arizona LTD

    Phoenix, Arizona, 85053

  • Orange County Retina Medical Group

    Santa Ana, California, 92705

  • California Retina Consultants

    Santa Barbara, California, 93103

Other trials in United States

About this study

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)

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:

Read more
  • 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.
Read the 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 of 20/200 or worse. 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 second cohort patients will have significant central vision loss with BCVA of the eye to be implanted of 20/80 or worse, but better than or equal to 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.

Being tested
CPCB-RPE1
Medical subject headings
Geographic Atrophy

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CPCB-RPE1Biological

    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.

Study groups in NCT02590692
Study groupRoleWhat they receive
CPCB-RPE1 treatmentExperimentalSubretinal implantation of CPCB-RPE1 in dry AMD patients
Receives: Biological: CPCB-RPE1

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: 1 year

Secondary outcomes (3)

  1. Visual Acuity

    Comparison of VA changes in the treated eye versus baseline and versus the non-treated eye

    Measured: 1 year

  2. Visual Field

    Comparison of visual field changes in the treated eye versus baseline and versus the non-treated eye

    Measured: 1 year

  3. Photoreceptor Electrical Responses

    Comparison of multifocal electroretinogram changes in the treated eye versus baseline and versus the non-treated eye

    Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Oct 2015 (estimated)
  2. Study start16 Feb 2016
  3. Primary completionJul 2019
  4. Study completionJun 2023 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Other
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02590692
Sponsor's protocol ID
RPT-14-01
Status last verified
May 2020
First submitted
23 Oct 2015
Last updated
29 May 2020
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing CPCB-RPE1 in people with Dry Macular Degeneration and Geographic Atrophy, run by Regenerative Patch Technologies, LLC at 6 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 12 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 29 May 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Frequency and Severity of Treatment-Related Adverse Events [Safety and Tolerability] (time frame: 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02590692.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 May 2020.

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