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Trial record 1 of 1 for:    luxa | Recruiting, Not yet recruiting Studies | Macular Degeneration | United States
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Safety and Tolerability of RPESC-derived RPE Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04627428
Recruitment Status : Recruiting
First Posted : November 13, 2020
Last Update Posted : July 26, 2022
Sponsor:
Collaborators:
National Institutes of Health (NIH)
National Eye Institute (NEI)
Regenerative Research Foundation
Information provided by (Responsible Party):
Luxa Biotechnology, LLC

Study Description
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Brief Summary:
The main objective of the study is evaluation of the safety and tolerability of RPESC-RPE-4W as therapy for dry AMD.

Condition or disease Intervention/treatment Phase
Dry Age-related Macular Degeneration Biological: RPESC-RPE-4W Phase 1 Phase 2

Detailed Description:

RPESC-RPE-4W is Allogeneic RPE stem cell (RPESC)-derived RPE cells (RPESC-RPE) isolated from the RPE layer of human cadaveric eyes are transplanted under the macular.

This first-in-human Phase 1/2a open-label dose-escalation interventional study plans to enroll a total of 18 subjects

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase1/2a, Open-Label Study to Evaluate the Safety and Tolerability of RPESC-derived RPE Transplantation as Therapy for Dry Age-related Macular Degeneration (AMD)
Actual Study Start Date : April 5, 2022
Estimated Primary Completion Date : May 31, 2025
Estimated Study Completion Date : May 31, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 50,000 cells
Six patients will receive single dose of 50,000 RPESC-RPE-4W cells in the eye.
 Biological: RPESC-RPE-4W
RPESC-RPE-4W
Experimental: 150,000 cells
Six patients will receive single dose of 150,000 RPESC-RPE-4W cells in the eye.
 Biological: RPESC-RPE-4W
RPESC-RPE-4W
Experimental: 250,000 cells
Six patients will receive single dose of 250,000 RPESC-RPE-4W cells in the eye.
 Biological: RPESC-RPE-4W
RPESC-RPE-4W


Outcome Measures
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Primary Outcome Measures :
  1. Safety and tolerability of RPESC-RPE-4W transplantation [ Time Frame: 24 months ]

    The transplantation of RPESC-RPE-4W cells will be considered safe and tolerated in the absence of:

    • Decrease in visual acuity (VA) of more than 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (or to worse than counting fingers at three feet) from baseline
    • Any Grade 2 (CTCAE version 5) or greater Adverse Events (AE) related to the cell product and investigational interventions.
    • Any evidence that the cell are contaminated with an infectious agent or serious immune response to the cell product
    • Any evidence that the cells show tumorigenic potential


Secondary Outcome Measures :
  1. Change in the mean of Best Corrected Visual Acuity (BCVA) [ Time Frame: 24 months ]
    Change in visual acuity will be measured by ETDRS chart.

  2. Loss of ≥10 decibels of ten-degree average visual sensitivity microperimetry [ Time Frame: 24 months ]
    Loss of ≥10 decibels of ten-degree average visual sensitivity will by measured by microperimetry.

  3. Change in GA lesion area [ Time Frame: 24 months ]
    Change in GA lesion area will be measured.

  4. Evidence of structural changes [ Time Frame: 24 months ]
    Structural evidence will be measured by OCT imaging, autofluorescence, fluorescein angiography, and fundus photography


Eligibility Criteria
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Ages Eligible for Study:   55 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Clinical diagnosis of dry AMD.
  2. Ability to understand and give informed consent.
  3. Adult male or female >55 years of age.
  4. Medically suitable to undergo vitrectomy and subretinal injection (>60% on Karnofsky scale).
  5. Postmenopausal if female (expected to be common for the age limitation), or the female partner of a male subject unable to father children.
  6. If male, willing to use barrier and spermicidal contraception during the study.

Exclusion Criteria:

  1. Allergy or hypersensitivity to dilation drops or fluorescein.
  2. Active major medical conditions limiting ability to participate in the study.
  3. Active malignancy or treatment with chemotherapy.
  4. Systemic immunosuppressant therapy within past six months.
  5. History of toxoplasmosis, retinal histoplasmosis or tuberculosis.
  6. Receipt of investigational product (IP) in a clinical trial within prior six months.
  7. Any other medical condition, which, in the Investigator's judgment, will interfere with the subject's ability to comply with the protocol, compromises subject safety, or interferes with the interpretation of the study results.
  8. Pregnant or nursing females.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04627428


Contacts
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Contact: Jeffrey H Stern, M.D., Ph.D. 05184371111 jeffreystern@luxabiotech.com

Locations
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United States, Michigan
University of Michigan Kellogg Eye Center Recruiting
Ann Arbor, Michigan, United States, 48105
Contact: Rajesh C Rao, M.D.         
Sponsors and Collaborators
Luxa Biotechnology, LLC
National Institutes of Health (NIH)
National Eye Institute (NEI)
Regenerative Research Foundation
Investigators
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Principal Investigator: Rajesh C Rao, M.D. University of Michigan Kellogg Eye Center
More Information
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Responsible Party: Luxa Biotechnology, LLC
ClinicalTrials.gov Identifier: NCT04627428    
Other Study ID Numbers: RPESC-RPE-01
U01EY030581 ( U.S. NIH Grant/Contract )
UG3EY031810 ( U.S. NIH Grant/Contract )
First Posted: November 13, 2020    Key Record Dates
Last Update Posted: July 26, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases