AzTrials
AzTrials

X-linked RetinoschisisPhase 3LIGHTHOUSENCT05878860

ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

A Phase 3 study testing ATSN-201 in people with X-linked Retinoschisis, run by Atsena Therapeutics Inc. at 21 sites in 3 countries.

Recruiting

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

Sites and contacts
Official record
Search all trials

Trial summary

This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 5 years of age with RS1-associated X-linked retinoschisis (XLRS). Read more about this study

Who is it for?
Ages 5 years and older
Conditions the study is for
X-linked Retinoschisis
What is being tested?
ATSN-201
Could I receive a placebo?
No, everyone receives an active treatment
How many people
97 participants
Where
21 sitesin 3 countries
Study dates
22 Aug 2023to Apr 2033
Phase
Phase 3
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 5 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Age ≥ 18 for Cohorts 1 through 4, and age ≥ 6 years and < 18 years for Cohort 5.
  • Male patients with clinical diagnosis of XLRS caused by mutations in RS1.
  • Best corrected visual acuity (BCVA) in study eye of 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (corresponding to a Snellen acuity of 20/200 to 20/40).
  • Presence of foveal schisis and /or parafoveal/perifoveal schisis in the study eye on SD-OCT per the Principal Investigator.
  • Note: For patients ineligible for bilateral dosing based on the ocular exclusion criteria, the same eye must meet all ocular inclusion criteria, but none of the ocular exclusion criteria, to be eligible for unilateral dosing.
  • General All of the following criteria must be met for unilateral or bilateral dosing.
Show all 11Show fewer
  • Age ≥ 5 years.
  • Genetically male patients with clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1 OR Genetically female patients with clinical diagnosis of XLRS caused by biallelic pathogenic or likely pathogenic mutations in RS1.
  • Ocular At least 1 eye must meet all of the following criteria for both unilateral and bilateral dosing.
  • BCVA of 34 to 73 ETDRS letters (corresponding to a Snellen acuity of 20/200 to 20/40).
  • Presence of foveal schisis on SD-OCT.

Who cannot join

  • Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants).
  • Any intraocular surgery (including laser treatment) in the study eye within 6 months prior to Screening or any intraocular surgery anticipated in the study eye during the first 12 months of the study.
  • Treatment in a prior ocular gene or cell therapy study.
  • Part C:
  • General None of the following criteria can be met for unilateral or bilateral dosing.
  • Treatment with any carbonic anhydrase inhibitor (oral or topical) within 1 month prior to Screening.
Show all 11Show fewer
  • Treatment in a prior ocular gene or cell therapy study.
  • Absence of macular schisis.
  • BCVA better than 75 ETDRS letters (corresponding to a Snellen acuity of 20/32).
  • Pre-existing eye conditions that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants).
  • Any intraocular surgery (including laser treatment) within 6 months prior to Screening or any intraocular surgery anticipated during the first 12 months of the study (laser treatment is allowed as long as it occurs at least 2 weeks before surgery).

Trial locations and contacts

21 sites in 3 countries. Contact the study team or a site near you to ask about taking part.

Atsena Therapeutics Clinical Trials contact

984-261-2001

clinicaltrials@atsenatx.com

21 sites in 3 countries

United States18 sites, 18 recruiting

  • University of Michigan

    Ann Arbor, Michigan, 48105

    Thomas Wubben, MD

    Recruiting

  • Massachusetts Eye and Ear

    Boston, Massachusetts, 02114

    Rachel Huckfeldt, MD

    Recruiting

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

    Anne Fulton, MD

    Not yet recruiting

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231

    Mark Pennesi, MD

    Recruiting

  • Duke University

    Durham, North Carolina, 27710

    Lejla Vajzovic, MD

    Recruiting

  • Shiley Eye Institute

    La Jolla, California, 92093

    Shyamanga Borooah, MD

    Not yet recruiting

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027

    Aaron Nagiel, MD, PhD

    Recruiting

  • University of Wisconsin

    Madison, Wisconsin, 53705

    Melanie Schmitt, MD

    Recruiting

  • Bascom Palmer Eye Institute

    Miami, Florida, 33136

    Byron Lam, MD

    Recruiting

  • Columbia University

    New York, New York, 100032

    Aliaa Abdelhakim, MD

    Recruiting

  • Stanford University

    Palo Alto, California, 94303

    Vinit Mahajan, MD

    Not yet recruiting

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

    Tomas Aleman, MD

    Recruiting

  • Associated Retina Consultants

    Phoenix, Arizona, 85020

    Benjamin Bakall, MD

    Recruiting

  • Oregon Health Sciences University

    Portland, Oregon, 97239

    Lesley Everett, MD, PhD, MPhil

    Recruiting

  • Mayo Clinic

    Rochester, Minnesota, 55905

    Raymond Iezzi, MD

    Recruiting

  • Flaum Eye Institute, University of Rochester

    Rochester, New York, 14642

    Alex Levin, MD

    Not yet recruiting

  • Moran Eye Center

    Salt Lake City, Utah, 84132

    Eileen Hwang, MD

    Recruiting

  • UC San Francisco

    San Francisco, California, 94158

    Jacque Duncan, MD

    Recruiting

Other trials in United States

Canada2 sites, 2 recruiting

  • McGill University (MUHC)

    Montreal, Quebec, H4A 3J1

    Robert Koenekoop, MD

    Not yet recruiting

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 0A4

    Elise Heon, MD

    Not yet recruiting

Other trials in Canada

United Kingdom1 site, 1 recruiting

  • Oxford Eye Hospital

    Oxford, England, OX3 9DU

    Dominik Fischer, MD

    Recruiting

Other trials in United Kingdom

About this study

Official title: A Phase 1/2/3, Open-Label, Dose Escalation, Dose Expansion and Randomized, Controlled Study to Evaluate the Safety and Efficacy of ATSN-201 Gene Therapy in Subjects With RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)

This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 5 years of age with RS1-associated X-linked retinoschisis (XLRS).

Read the detailed description

The study is designed in three parts: a dose escalation phase (Part A), a dose expansion phase (Part B) and a randomized, controlled phase (Part C).

In Part C of the study, eligible patients who enroll in this study will be randomly assigned to be treated with ATSN-201 or to have no treatment; subjects assigned to ATSN-201 will receive the drug as a one-time subretinal injection of ATSN-201 in one eye or both eyes, depending on whether only one or both eyes meet criteria for treatment. Subjects will have regular assessments for 1 year as part of the Main Study Period and additional assessments over the next 4 years as part of the Extension Study Period.

Some subjects may have all their study visits at a surgery site.

Some subjects may go to one study site (a medical site) to determine eligibility and another study site (a surgery site) to have surgery - including pre-operative care and approximately 1-week of post-operative care per treated eye. After the surgery, they will go back to the other study site (the medical site) to complete the follow-up visits.

Subjects who do not receive treatment as part of the control group can choose to receive ATSN-201 in one or both eyes after the 1-year Main Study Period if eligible. If the subject is not eligible at the Month 12 visit, eligibility may be reassessed at the Month 18 visit.

Being tested
ATSN-201
Keywords
XLRS, RS1
Medical subject headings
Retinoschisis

What participants receive

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

  • ATSN-201Biological

    AAV.SPR-hGRK1-hRS1syn

Study groups in NCT05878860
Study groupRoleWhat they receive
Cohort 1, Low DoseExperimentalNot described
Receives: Biological: ATSN-201
Cohort 2, High DoseExperimentalNot described
Receives: Biological: ATSN-201
Cohort 3, Mid DoseExperimentalNot described
Receives: Biological: ATSN-201
Cohort 4, Low VolumeExperimentalNot described
Receives: Biological: ATSN-201
Cohort 4, High VolumeExperimentalNot described
Receives: Biological: ATSN-201
Cohort 4, ControlNo interventionNot described
Cohort 5, PediatricExperimentalNot described
Receives: Biological: ATSN-201
Cohort 6, TreatmentExperimentalNot described
Receives: Biological: ATSN-201
Cohort 6, ControlNo interventionNot described

What is being measured

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

  1. Part A (Dose Escalation) and Part B (Dose Expansion): Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events

    Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs).

    Measured: From baseline to week 52

  2. Part C (Phase 3, Randomized, Controlled) Effect of ATSN-201 on visual function.

    Proportion of subjects with improvement ≥ 7dB from baseline in microperimetry across prespecified loci in the study eye.

    Measured: From baseline to week 52

Secondary outcomes (21)

  1. Part A and Part B: Visual acuity as assessed by best-corrected visual acuity

    Change in best-corrected visual acuity (BCVA).

    Measured: From baseline to week 52

  2. Part A and Part B: Visual acuity as assessed by low-luminance visual acuity

    Change in low-luminance visual acuity (LLVA).

    Measured: From baseline to week 52

  3. Part A and Part B: Visual function as assessed by contrast sensitivity

    Change in contrast sensitivity.

    Measured: From baseline to week 52

  4. Part A and Part B: Visual function as assessed by microperimetry

    Change in microperimetry.

    Measured: From baseline to week 52

  5. Part A and Part B: Macular structure as assessed by spectral domain optical coherence tomography

    Change in spectral domain optical coherence tomography (SD-OCT).

    Measured: From baseline to week 52

  6. Part A and Part B: Macular structure as assessed by fundus autofluorescence

    Change in fundus autofluorescence (FAF).

    Measured: From baseline to week 52

  7. Part A and Part B: Subject-reported visual function as assessed by the NEI VFQ-25 in adult subjects

    Change in the National Eye Institute's Visual Function Questionnaire 25 (NEI VFQ-25) score for adult subjects with scores from 0 to 100 where a higher score indicates a better outcome.

    Measured: From baseline to week 52

  8. Part A and Part B: Subject-reported visual function as assessed by the CVAQC in pediatric subjects

    Change in the Cardiff Visual Ability Questionnaire for Children (CVAQC) score for pediatric subjects with scores from -3.00 to +2.80 where a higher score indicates a worse outcome.

    Measured: From baseline to week 52

  9. Part A and Part B: Subject-reported visual function as assessed by the MRDQ in subjects 13 years of age or greater.

    Description: Change in the Michigan Retinal Degeneration Questionnaire (MRDQ) score for subjects 13 years of age or greater.

    Measured: From baseline to week 52

  10. Part C: Macular structure as assessed by spectral domain optical coherence tomography

    Change in spectral domain optical coherence tomography (SD-OCT).

    Measured: From baseline to week 52

  11. Part C: Visual acuity as assessed by best-corrected visual acuity or low-luminance visual acuity

    Change in best-corrected visual acuity (BCVA) or low luminance visual acuity (LLVA).

    Measured: From baseline to week 52

  12. Part C: Visual acuity as assessed by best-corrected visual acuity as measured by Early Treatment Diabetic Retinopathy Study chart

    Change in best-corrected visual acuity (BCVA) as assessed by ETDRS.

    Measured: From baseline to week 52

  13. Part C: Visual acuity as assessed by low luminance visual acuity as measured by Early Treatment Diabetic Retinopathy Study chart

    Description: Change in low luminance visual acuity (LLVA) as assessed by ETDRS.

    Measured: From baseline to week 52

  14. Part C: Visual function as assessed by microperimetry

    Change in microperimetry for subjects.

    Measured: From baseline to week 52

  15. Part C: Functional vision as assessed by reading speed

    Change from baseline in reading speed as measured by MNREAD for subjects 8 years of age and older.

    Measured: From baseline to week 52

  16. Part C: Subject-reported visual function as assessed by the MRDQ in subjects 13 years of age or greater

    Change in the Michigan Retinal Degeneration Questionnaire (MRDQ) score for subjects 13 years of age or greater.

    Measured: From baseline to week 52

  17. Part C: Subject-reported visual function as assessed by the CVAQC in pediatric subjects

    Change in the Cardiff Visual Ability Questionnaire for Children (CVAQC) score for pediatric subjects ages 5-13 years of age.

    Measured: From baseline to week 52

  18. Part C: Subject perception of change and severity (PGIC/PGIS)

    Patient global impression of change and severity (PGIC/PGIS).

    Measured: From baseline to week 52

  19. Part C: Evaluate the safety of ATSN-201

    Incidence of treatment emergent adverse events (TEAEs).

    Measured: From baseline to week 52

  20. Part C: To evaluate the quality of life in subjects treated with ATSN-201

    Change from Baseline in European Quality of Life 5-Dimension 5-Level (EQ-5D-5L) Health Index score and Visual Analogue Scale (VAS) score at Month 12 for subjects ≥ 12 years of age in the ATSN-201 group vs. controls. Scale 0 (worst health you can imagine) to 100 (best health you can imagine).

    Measured: From baseline to week 52

  21. Part C: To evaluate the quality of life in pediatric patients treated with ATSN-201

    Change from Baseline in European Quality of Life 5-Dimension Youth 5-Level (EQ-5D-Y-5L) Health Index score and Visual analogue Scale (VAS) score at Month 12 for subjects ≥ 8 to < 12 years of age in the ATSN201 group vs. controls. Scale 0 (worst health you can imagine) to 100 (best health you can imagine).

    Measured: From baseline to week 52

Timeline

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

  1. First posted26 May 2023
  2. Study start22 Aug 2023
  3. Primary completionApr 2028 (estimated)
  4. Study completionApr 2033 (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 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
97 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05878860
Sponsor's protocol ID
ATSN-201-1
Acronym
LIGHTHOUSE
Status last verified
Sep 2026
First submitted
12 May 2023
Last updated
29 Sep 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 3 study testing ATSN-201 in people with X-linked Retinoschisis, run by Atsena Therapeutics Inc. at 21 sites in 3 countries.

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 11 inclusion and 11 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 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 21 sites in United States, Canada, United Kingdom. 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 phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 Atsena Therapeutics Clinical Trials (984-261-2001, clinicaltrials@atsenatx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part A (Dose Escalation) and Part B (Dose Expansion): Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events (time frame: From baseline to week 52). The study lists 2 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 NCT05878860.

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 Sep 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in