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Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)

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ClinicalTrials.gov Identifier: NCT05616793
Recruitment Status : Recruiting
First Posted : November 15, 2022
Last Update Posted : June 18, 2023
Sponsor:
Information provided by (Responsible Party):
Opus Genetics, Inc

Study Description
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Brief Summary:
The goal of this clinical trial is to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.

Condition or disease Intervention/treatment Phase
LCA5 Biological: AAV8.hLCA5 Phase 1 Phase 2

Detailed Description:

This is a non-randomized, open-label, phase 1/2 dose-escalation study evaluating two doses of OPGx-001 for the treatment of LCA5-IRD.

Enrollment will begin with a low-dose of OPGx-001 delivered via single, unilateral subretinal injection (Cohort 1) and proceed to an intermediate dose (Cohort 2) and subsequent high dose (Cohort 3). Escalation to each next cohort will proceed only after review of all data and upon recommendation by an independent data monitoring committee (IDMC).

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 9 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label, Dose Exploration, Safety and Tolerability Study of a Subretinal Injection of an OPGx-001 Gene Vector to Participants With LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)
Actual Study Start Date : June 15, 2023
Estimated Primary Completion Date : August 30, 2024
Estimated Study Completion Date : September 30, 2027

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Dose Group 1
Single, unilateral subretinal administration of a low dose of OPGx-001 to adult participants at least 18 years of age
 Biological: AAV8.hLCA5
Adeno-associated virus vector expressing human LCA5 gene
Experimental: Dose Group 2
Single, unilateral subretinal administration of an intermediate dose of OPGx-001 to adult participants at least 18 years of age
 Biological: AAV8.hLCA5
Adeno-associated virus vector expressing human LCA5 gene
Experimental: Dose Group 3
Single, unilateral subretinal administration of a high dose of OPGx-001 to adult participants at least 18 years of age
 Biological: AAV8.hLCA5
Adeno-associated virus vector expressing human LCA5 gene


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of dose limiting toxicities [ Time Frame: 1 year ]
  2. Number of adverse events related to OPGx-001 [ Time Frame: 1 year ]
  3. Number of procedure-related adverse events [ Time Frame: 1 year ]
  4. Change in retinal thickness (as measured by OCT) [ Time Frame: 1 year ]
    Changes from baseline in total retinal thickness and outer retinal thickness (in microns)


Secondary Outcome Measures :
  1. Change from baseline to month 12 in retinal sensitivity as measured by full-field stimulus testing (FST) [ Time Frame: 1 year ]
  2. Change from baseline to month 12 in best corrected visual acuity (BCVA) [ Time Frame: 1 year ]
  3. Change from baseline to month 12 in oculomotor control and fixation stability [ Time Frame: 1 year ]
  4. Change from baseline to month 12 in dark-adapted transient pupillary light reflexes (TPLR) [ Time Frame: 1 year ]
  5. Change from baseline to month 12 in visual functioning questionnaire [ Time Frame: 1 year ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Are willing and able to provide written informed consent (ICF) and, where appropriate, willing to sign an assent prior to any study procedures.
  2. Are willing to adhere to the clinical protocol and able to perform testing procedures.
  3. Participants must be at least 18 years of age at consent.
  4. Carry disease-causing biallelic LCA5 gene mutations determined by a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory.
  5. Visual acuity: BCVA < 20/80 on the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity chart (modified for low vision participants) in the eye to be treated
  6. Show evidence of detectable photoreceptors by Spectral Domain Optical Coherence Tomography (SD-OCT)
  7. Participant is a good candidate for surgery per investigator judgement
  8. Participant agrees to follow direction of investigator regarding restrictions post-surgery.

Exclusion Criteria:

  1. Individuals of childbearing potential (male and female) who are pregnant or unwilling to use effective contraception for the duration of the study, including barrier methods for the first year after investigational product (IP) administration.
  2. Pre-existing eye conditions or complicating systemic diseases that would preclude the planned surgery. This includes individuals who are immunocompromised.
  3. History of intraocular surgery for either eye within 6 months prior to planned IP administration.
  4. Have previously received gene therapy.
  5. Have used any investigational drug or device within 90 days or 5 estimated half-lives of treatment, whichever is longer or plan to participate in another study of drug or device during the study period.
  6. History of disease which may preclude the participant from participation, or which may interfere with outcome measure testing or test results.
  7. Incapable of performing visual function testing (e.g., FST testing) for reasons other than poor vision.
  8. Any absolute contraindication to a course of oral steroids.
  9. Any other condition that would not allow the potential participant to complete follow-up examinations during the study and, in the opinion of the Investigator, makes the potential participant unsuitable for the study.
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): NCT05616793


Contacts
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Contact: Sarah Tuller 8608882718 STuller@OpusGtx.com
Contact: Jasminder Soto

Locations
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United States, Pennsylvania
University of Pennsylvania Perelman School of Medicine Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Mariejel Weber    215-662-6396    mariejel.weber@pennmedicine.upenn.edu   
Principal Investigator: Tomas Aleman, MD         
Sponsors and Collaborators
Opus Genetics, Inc
More Information
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Responsible Party: Opus Genetics, Inc
ClinicalTrials.gov Identifier: NCT05616793    
Other Study ID Numbers: OPGx-LCA5-1001
First Posted: November 15, 2022    Key Record Dates
Last Update Posted: June 18, 2023
Last Verified: June 2023
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
Keywords provided by Opus Genetics, Inc:
LCA
Retinal Degeneration
IRD
 adeno associated virus
gene therapy
LCA5
Additional relevant MeSH terms:
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Retinal Degeneration
Eye Diseases, Hereditary
Eye Diseases
Retinal Diseases