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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

Tracking Information
First Submitted Date  ICMJE November 7, 2022
First Posted Date  ICMJE November 15, 2022
Last Update Posted Date June 18, 2023
Actual Study Start Date  ICMJE June 15, 2023
Estimated Primary Completion Date August 30, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 7, 2022)
  • Incidence of dose limiting toxicities [ Time Frame: 1 year ]
  • Number of adverse events related to OPGx-001 [ Time Frame: 1 year ]
  • Number of procedure-related adverse events [ Time Frame: 1 year ]
  • 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)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 7, 2022)
  • Change from baseline to month 12 in retinal sensitivity as measured by full-field stimulus testing (FST) [ Time Frame: 1 year ]
  • Change from baseline to month 12 in best corrected visual acuity (BCVA) [ Time Frame: 1 year ]
  • Change from baseline to month 12 in oculomotor control and fixation stability [ Time Frame: 1 year ]
  • Change from baseline to month 12 in dark-adapted transient pupillary light reflexes (TPLR) [ Time Frame: 1 year ]
  • Change from baseline to month 12 in visual functioning questionnaire [ Time Frame: 1 year ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)
Official Title  ICMJE 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)
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.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE LCA5
Intervention  ICMJE Biological: AAV8.hLCA5
Adeno-associated virus vector expressing human LCA5 gene
Study Arms  ICMJE
  • Experimental: Dose Group 1
    Single, unilateral subretinal administration of a low dose of OPGx-001 to adult participants at least 18 years of age
    Intervention: Biological: AAV8.hLCA5
  • Experimental: Dose Group 2
    Single, unilateral subretinal administration of an intermediate dose of OPGx-001 to adult participants at least 18 years of age
    Intervention: Biological: AAV8.hLCA5
  • Experimental: Dose Group 3
    Single, unilateral subretinal administration of a high dose of OPGx-001 to adult participants at least 18 years of age
    Intervention: Biological: AAV8.hLCA5
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: November 22, 2022)		 9
Original Estimated Enrollment  ICMJE
 (submitted: November 7, 2022)		 6
Estimated Study Completion Date  ICMJE September 30, 2027
Estimated Primary Completion Date August 30, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Sarah Tuller 8608882718 STuller@OpusGtx.com
Contact: Jasminder Soto
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05616793
Other Study ID Numbers  ICMJE OPGx-LCA5-1001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Opus Genetics, Inc
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Opus Genetics, Inc
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Opus Genetics, Inc
Verification Date June 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP