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ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)

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: NCT05878860
Recruitment Status : Recruiting
First Posted : May 26, 2023
Last Update Posted : February 20, 2024
Sponsor:
Information provided by (Responsible Party):
Atsena Therapeutics Inc.

Brief Summary:
This study will evaluate the safety and tolerability of ATSN-201 in male subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).

Condition or disease Intervention/treatment Phase
X-linked Retinoschisis Biological: ATSN-201 Phase 1 Phase 2

Detailed Description:
Eligible patients who enroll in this study will receive a one-time subretinal injection of ATSN-201 in one eye. Safety and tolerability will be evaluated for 5 years.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Masking Description: Cohort 3 will be partially masked.
Primary Purpose: Treatment
Official Title: A Phase 1/2, Open-Label, Dose Escalation and Dose Expansion Study to Evaluate the Safety and Tolerability of ATSN-201 Gene Therapy in Male Subjects With RS1-Associated X-linked Retinoschisis
Actual Study Start Date : August 22, 2023
Estimated Primary Completion Date : October 2025
Estimated Study Completion Date : October 2029


Arm Intervention/treatment
Experimental: Cohort 1
ATSN-201 at Low Dose
Biological: ATSN-201
AAV.SPR-hGRK1-hRS1syn

Experimental: Cohort 2
ATSN-201 at High Dose
Biological: ATSN-201
AAV.SPR-hGRK1-hRS1syn

Experimental: Cohort 3, High Dose
ATSN-201 at High Volume
Biological: ATSN-201
AAV.SPR-hGRK1-hRS1syn

Experimental: Cohort 3, Low Dose
ATSN-201 at Low Volume
Biological: ATSN-201
AAV.SPR-hGRK1-hRS1syn

No Intervention: Cohort 3, Control
Experimental: Cohort 4, Pediatric
ATSN-201 at High Dose
Biological: ATSN-201
AAV.SPR-hGRK1-hRS1syn




Primary Outcome Measures :
  1. Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events [ Time Frame: From baseline to week 52 ]
    Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs).


Secondary Outcome Measures :
  1. Visual acuity as assessed by best-corrected visual acuity [ Time Frame: From baseline to week 52 ]
    Change in best-corrected visual acuity (BCVA).

  2. Visual acuity as assessed by low-luminance visual acuity [ Time Frame: From baseline to week 52 ]
    Change in low-luminance visual acuity (LLVA).

  3. Visual function as assessed by contrast sensitivity [ Time Frame: From baseline to week 52 ]
    Change in contrast sensitivity.

  4. Visual function as assessed by full-field electroretinogram parameters [ Time Frame: From baseline to week 52 ]
    Change in full-field electroretinogram (ffERG) parameters.

  5. Visual function as assessed by microperimetry [ Time Frame: From baseline to week 52 ]
    Change in microperimetry.

  6. Visual function as assessed by static perimetry [ Time Frame: From baseline to week 52 ]
    Change in static perimetry.

  7. Macular structure as assessed by spectral domain optical coherence tomography [ Time Frame: From baseline to week 52 ]
    Change in spectral domain optical coherence tomography (SD-OCT).

  8. Macular structure as assessed by fundus autofluorescence [ Time Frame: From baseline to week 52 ]
    Change in fundus autofluorescence (FAF).

  9. Subject-reported visual function as assessed by the NEI VFQ-25 in adult subjects [ Time Frame: From baseline to week 52 ]
    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.

  10. Subject-reported visual function as assessed by the CVAQC in pediatric subjects [ Time Frame: From baseline to week 52 ]
    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.



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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Age ≥ 18 for Cohorts 1 through 3, and age ≥ 6 years and < 18 years for Cohort 4.
  2. Male patients with clinical diagnosis of XLRS caused by mutations in RS1.
  3. 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).

Exclusion Criteria:

  1. Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection.
  2. Any intraocular surgery (including laser treatment) in the study eye within 6 months prior or any intraocular surgery anticipated in the study eye during the first 12 months of the study.
  3. Treatment in a prior ocular gene or cell therapy study.

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): NCT05878860


Contacts
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Contact: Atsena Therapeutics Clinical Trials 984-261-2001 clinicaltrials@atsenatx.com

Locations
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United States, California
Children's Hospital of Los Angeles Recruiting
Los Angeles, California, United States, 90027
Contact: Hillary Schwartz, MS    323-361-1004    hschwartz@chla.usc.edu   
Contact: Dilshad Contractor    323-361-7194    dcontractor@chla.usc.edu   
Principal Investigator: Aaron Nagiel, MD, PhD         
United States, Florida
Bascom Palmer Eye Institute Recruiting
Miami, Florida, United States, 33136
Contact: Tamara Riesgo    305-482-4584    txj362@miami.edu   
Principal Investigator: Byron Lam, MD         
United States, Oregon
Oregon Health Sciences University Recruiting
Portland, Oregon, United States, 97239
Contact: Stephanie Timko    503-494-9743    spences@ohsu.edu   
Principal Investigator: Lesley Everett, MD, PhD, MPhil         
Sponsors and Collaborators
Atsena Therapeutics Inc.
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Responsible Party: Atsena Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT05878860    
Other Study ID Numbers: ATSN-201-1
First Posted: May 26, 2023    Key Record Dates
Last Update Posted: February 20, 2024
Last Verified: February 2024
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 Atsena Therapeutics Inc.:
XLRS, RS1
Additional relevant MeSH terms:
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Retinoschisis
Retinal Degeneration
Retinal Diseases
Eye Diseases