Clinical trial NCT05878860: ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: X-linked Retinoschisis
Keywords: XLRS, RS1
Lead sponsor: Atsena Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2023-05-26
Enrollment: 21
Sex: MALE
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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).

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.

Eligibility 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.

Primary outcomes:
measure: Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events
timeFrame: From baseline to week 52
Secondary outcomes:
measure: Visual acuity as assessed by best-corrected visual acuity
timeFrame: From baseline to week 52
measure: Visual acuity as assessed by low-luminance visual acuity
timeFrame: From baseline to week 52
measure: Visual function as assessed by contrast sensitivity
timeFrame: From baseline to week 52
measure: Visual function as assessed by full-field electroretinogram parameters
timeFrame: From baseline to week 52
measure: Visual function as assessed by microperimetry
timeFrame: From baseline to week 52
measure: Visual function as assessed by static perimetry
timeFrame: From baseline to week 52
measure: Macular structure as assessed by spectral domain optical coherence tomography
timeFrame: From baseline to week 52
measure: Macular structure as assessed by fundus autofluorescence
timeFrame: From baseline to week 52
measure: Subject-reported visual function as assessed by the NEI VFQ-25 in adult subjects
timeFrame: From baseline to week 52
measure: Subject-reported visual function as assessed by the CVAQC in pediatric subjects
timeFrame: From baseline to week 52
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: Miami
country: United States
facility: Bascom Palmer Eye Institute
state: Florida
city: Portland
country: United States
facility: Oregon Health Sciences University
state: Oregon
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
Study officials:
References: