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Leber Congenital AmaurosisPhase 1/2RDH12NCT07681778

Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)

A Phase 1/2 study testing OPGx-RDH12 in people with Leber Congenital Amaurosis and Leber Congenital Amaurosis (LCA), run by Opus Genetics, Inc at 4 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

This study is an early-stage clinical trial (Phase 1b/2a) testing a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare genetic eye disease that leads to severe vision loss. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
OPGx-RDH12
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
4 sitesUnited States
Study dates
Oct 2026to Jul 2034
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age ≥18 years (adult participants) or 12-17 years (adolescent participants) at the time of consent/assent.
  • Provide written informed consent and/or assent prior to any study procedures.
  • Willing to adhere to the clinical protocol and follow directions of the Investigator regarding post-surgery restrictions.
  • Are a good candidate for surgery, per the Investigator's judgment.
  • Have LCA with autosomal-recessive RDH12 mutation(s), confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory. Historic testing, up to 15 years prior to date of consent, may be considered.
  • Clinical diagnosis of LCA with RDH12 mutation(s), in the judgment of the Investigator.
  • BCVA 20/200 (1.0 logarithm of the minimum angle of resolution [logMAR]) or worse for the sentinel adult in each cohort; BCVA 20/40 (0.5 logMAR) or worse for all subsequent participants in each cohort.

Who cannot join

  • Women of childbearing potential (WOCBP) who are pregnant, lactating, and/or unwilling to use effective contraception from Screening through 1 year after IMP administration.
  • Men who are unwilling to use effective contraception from Screening through 180 days after IMP administration.
  • Have an ocular infection, a pre-existing eye condition, or a complicating systemic disease that could preclude the planned surgery or any future ocular surgery. This includes individuals who are immunocompromised and/or on continuous systemic immunosuppressive therapy.
  • Have a past or current condition that may preclude participation in the study, interfere with outcome measure testing or test results, or otherwise make the potential participant unsuitable for the study.
  • Have previously received gene therapy of any kind.
  • In either eye, have undergone intraocular surgery within 90 days prior to planned IMP administration or have active inflammation at Screening resulting from prior ocular surgery.
Show all 16Show fewer
  • Have used any investigational device or investigational drug within 90 days (or 5 half-lives of the drug, whichever is longer) prior to planned IMP administration or intend to participate in another drug or device study during the same period as the current study.
  • Have received or plan to receive a vaccination within 6 weeks prior to or 6 weeks after IMP administration. Note: For the influenza vaccine, the exclusionary period is shorter: 2 weeks prior to and 5 weeks after IMP administration (i.e., during steroid treatment).
  • Have received anticoagulant therapy within 2 weeks prior to planned IMP administration.
  • Currently use medications that are potentially neuroprotective/beneficial or retinotoxic.
  • Are incapable of performing visual function testing (e.g., FST), with or without assistance, for reason other than poor vision.
  • Have any contraindication to a course of oral steroids, in the opinion of the Investigator.
  • Have a known history of hypersensitivity to constituents or excipients in the pharmaceutical formulation of the IMP.
  • Have a known or active infection of human immunodeficiency virus (HIV) or hepatitis B or C virus.
  • Have a known or active infection of herpes simplex virus with ocular manifestations.
  • Are an employee of the Sponsor or a relative of the Investigator or investigative site staff.

Trial locations and contacts

4 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Study Director contact

984-884-6030

ct.gov_inquiries@opusgtx.com

United States4 sites, 4 recruiting

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231

    Mark Pennesi, MD, Ph.D.

    Not yet recruiting

  • Retina Consultants of Texas & Retina Group Inc.

    Houston, Texas, 77401

    Rebbecca Taing, (800) 833-5921, rebbecca.taing@retinaconsultantstexas.com

    Kenneth Fan, MD

    Recruiting

  • Perelman School of Medicine, University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

    Tomas Aleman, MD

    Not yet recruiting

  • Associated Retina Consultants

    Phoenix, Arizona, 85020

    Kaylee Rosenbusch, (480) 999-5458, kaylee.rosenbusch@doctrials.com

    Mallory Mintert, (480) 999-5458, mallory.mintert@doctrials.com

    Not yet recruiting

Other trials in United States

About this study

Official title: A Phase 1b/2a Open-Label, Dose-Exploration Study to Investigate the Safety and Tolerability of Subretinally Injected OPGx-RDH12 Administered in Participants With Leber Congenital Amaurosis With Autosomal-Recessive Retinol Dehydrogenase 12 Mutations

This study is an early-stage clinical trial (Phase 1b/2a) testing a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare genetic eye disease that leads to severe vision loss. The treatment is delivered as a one-time injection into the retina (subretinal space) of the worse-seeing eye, using a method similar to approved gene therapies like Luxturna. The study is designed to evaluate safety and effectiveness at two dose levels (1E11 and 3E11 viral genomes per eye) in small groups of 5 participants. Each group begins cautiously with 2 adults (age ≥18), treated at least one month apart, followed by FDA review before allowing adolescents (ages 12-17) to participate. An independent monitoring committee (IDMC) oversees safety throughout. After 3 adolescents are treated and followed for 3 months, the committee reviews all data to decide whether to move to a higher dose. However, if the lower dose (1E11 vg/eye) shows strong effectiveness in the first group, the study may expand by treating more adolescents at that same dose instead of increasing it further.

Read the detailed description

This is a Phase 1b/2a multicenter, open-label, and non-randomized dose-escalation safety study of up to two vector doses of OPGx-RDH12 administered via unilateral injection in participants with Leber Congenital Amaurosis (LCA) with autosomal-recessive retinol dehydrogenase 12 (RDH12) gene mutation(s). Administration will occur via a cannula into the subretinal space, using the standard technique for delivery of other adeno-associated virus (AAV) therapies including Luxturna®, the first United States Food and Drug Administration (FDA)-approved ocular gene therapy.

Up to 2 OPGx-RDH12 dose cohorts will be studied: 1E11 vg/eye (Cohort 1) and 3E11 vg/eye (Cohort 2). The treatment eye will be the eye with the worst visual function (as determined by visual acuity, full-field sensitivity testing [FST] and kinetic perimetry) or the non-dominant eye in cases of bilateral symmetric disease. Toxicity related to the administration of OPGx-RDH12 will be monitored in the eye using a comprehensive clinical monitoring plan and an Independent Data Monitoring Committee (IDMC).

Each cohort will enroll 5 participants. The first 2 participants will be adults (≥18 years old), treated at least 1 month apart. One month after the second participant's treatment, safety and efficacy data from the adult participants will be submitted to FDA for review and approval to open the study to adolescent participants (12-17 years old). The IDMC will next determine whether adolescent participants may be enrolled in the same dose cohort.

Three months after the third adolescent participant's treatment, the IDMC will review all safety and efficacy data from the cohort and determine whether to recommend escalation to the next dose cohort. Efficacy signals in Cohort 1 may justify conversion to a larger cohort of adolescent participants at the 1E11 vg/eye dose, with no further dose escalation.

Being tested
OPGx-RDH12
Keywords
Gene therapy, IRD, LCA, Leber Congenital Amaurosis, OPGx-RDH12
Medical subject headings
Leber Congenital Amaurosis

What participants receive

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

  • OPGx-RDH12Drug

    Experimental gene therapy

Study groups in NCT07681778
Study groupRoleWhat they receive
OPGx-RDH12ExperimentalAdministration of OPGx-RDH12 will occur via a cannula into the subretinal space, using the standard technique for delivery of other adeno-associated virus (AAV) therapies including Luxturna®. A dose of 1E11 vg/eye will be injected sub-retinally one time into the treatment eye. The treatment eye will be the eye with the worst visual function (as determined by visual acuity, full-field sensitivity testing [FST] and kinetic perimetry) or the non-dominant eye in cases of bilateral symmetric disease.
Receives: Drug: OPGx-RDH12

What is being measured

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

  1. Number of dose-limiting toxicity (DLT) events at the proposed doses

    Measured: 5 Years

  2. Number and severity of procedure-related AEs

    Measured: 5 Years

  3. Number and severity of AEs related to OPGx-RDH12

    Measured: 5 Years

  4. Qualitative assessment of cross-sectional spectral-domain optical coherence tomography (SD-OCT), fundus photography, and fundus autofluorescence (FAF) images

    Measured: 5 Years

Secondary outcomes (11)

  1. Change from baseline best corrected visual acuity (BCVA) with manifest refraction

    Measured: 5 Years

  2. Change from baseline Low luminance visual acuity (LLVA)

    Measured: 5 Years

  3. Change from baseline Kinetic visual fields

    Measured: 5 Years

  4. Change in baseline Microperimetry

    Measured: 5 Years

  5. Change from baseline Light- and dark-adapted full-field sensitivity testing (FST)

    Measured: 5 Years

  6. Change in baseline Pupillometry

    Measured: 5 Years

  7. Change from baseline Multi-Luminance Orientation and Mobility Test (MLoMT)

    Measured: 5 Years

  8. Change from baseline Participant-reported outcomes (Michigan Retinal Degeneration Questionnaire [MRDQ])

    The Michigan Retinal Degeneration Questionnaire (MRDQ) measures functional vision disability across 7 visual domains (e.g., central vision, color vision, contrast sensitivity). Rather than a simple sum, it uses an item-response theory (IRT) model, resulting in person-ability/disability scores (θ) ranging from -3 to +3. -3: Represents the lowest experienced disability (least difficulty or best visual function). +3: Represents the highest experienced disability (most severe difficulty or worst visual function).

    Measured: 5 Years

  9. Change from baseline Participant-reported outcomes (Patient Global Impressions of Severity [PGI-S])

    The Patient Global Impression of Severity [PGI-S] is a standardized, single-item questionnaire that allows patients to subjectively rate how severe their medical condition feels at a given moment. The Patient Global Impression of Severity [PGI-S] 5-point scale ranges from a minimum of 0 (None) to a maximum of 4 (Very severe).

    Measured: 5 Years

  10. Change from Baseline Participant-reported Outcomes (Patient Global Impressions of Change [PGI-C])

    The Patient Global Impressions of Change (PGIC) is a widely used patient-reported outcome (PRO) questionnaire in clinical trials. It evaluates how a patient's overall health, functioning, or symptoms have changed following a treatment compared to their baseline. A 7-point scale is utilized for the Patient Global Impression of Change (PGI-C), the minimum value is 1 (very much improved) and the maximum value is 7 (very much worse).

    Measured: 5 Years

  11. Change from Baseline Participant-reported outcomes (Patient Global Impressions of Improvement [PGI-I])

    The Patient Global Impression of Improvement (PGI-I) is a standardized, single-item patient-reported outcome (PRO) scale used in clinical trials. It asks participants to rate the change in their overall health or specific condition since beginning a therapy, treatment, or surgery. Patients rate their perceived change on a balanced 7-point scale ranging from 1 to 7: 1 = very much better, 7 = very much worse.

    Measured: 5 Years

Timeline

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

  1. First posted2 Jul 2026
  2. Study startOct 2026 (estimated)
  3. Primary completionDec 2032 (estimated)
  4. Study completionJul 2034 (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
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07681778
Sponsor's protocol ID
OPGx-RDH12-1001
Acronym
RDH12
Status last verified
Oct 2026
First submitted
24 Jun 2026
Last updated
30 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing OPGx-RDH12 in people with Leber Congenital Amaurosis and Leber Congenital Amaurosis (LCA), run by Opus Genetics, Inc at 4 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 7 inclusion and 16 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: looking for participants now, as of the sponsor's last update on 30 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 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?

This study is still under way. Results are not available in the source record yet.

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

Contact Study Director (984-884-6030, ct.gov_inquiries@opusgtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of dose-limiting toxicity (DLT) events at the proposed doses (time frame: 5 Years). The study lists 4 primary outcomes in all. 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 NCT07681778.

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 30 Oct 2026.

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