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ATSN-201

Phase 3

X-linked Retinoschisis | Monoclonal antibody | Other |cbdMD, Inc.|Last Updated: Aug 21, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedNO_TREATMENT_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment97

FDA Designations

No designations recorded

Clinical trial landscape

ATSN-201 · 1 trial · 1 indication

Phase 3 1
NCT05878860ATSN-201 Gene Therapy in RS1-Associated X-linked RetinoschisisX-linked Retinoschisis
RECRUITING97 Analytics
PHASE3RECRUITING
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
X-linked Retinoschisis
NCT0587886097
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Study Endpoints

Primary Endpoints

Part A (Dose Escalation) and Part B (Dose Expansion): Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events
From baseline to week 52

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

Part C (Phase 3, Randomized, Controlled) Effect of ATSN-201 on visual function.
From baseline to week 52

Proportion of subjects ≥12 years of age with improvement ≥ 7dB from baseline in microperimetry across prespecified loci in the study eye.

Secondary Endpoints

Part A and Part B: Visual acuity as assessed by best-corrected visual acuity
From baseline to week 52
Part A and Part B: Visual acuity as assessed by low-luminance visual acuity
From baseline to week 52
Part A and Part B: Visual function as assessed by contrast sensitivity
From baseline to week 52
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1, Low DoseEXPERIMENTAL -
Cohort 2, High DoseEXPERIMENTAL -
Cohort 3, Mid DoseEXPERIMENTAL -
Cohort 4, Low VolumeEXPERIMENTAL -
Cohort 4, High VolumeEXPERIMENTAL -
Cohort 4, ControlNO_INTERVENTION -
Cohort 5, PediatricEXPERIMENTAL -
Cohort 6, TreatmentEXPERIMENTAL -
Cohort 6, ControlNO_INTERVENTION -

Interventions

NameTypeDescription
ATSN-201BIOLOGICALAAV.SPR-hGRK1-hRS1syn
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Eligibility Criteria

Age Range6 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites19

Part A and B: Inclusion Criteria: 1. Age ≥ 18 for Cohorts 1 through 4, and age ≥ 6 years and \< 18 years for Cohort 5. 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 S...

Countries:United StatesUnited Kingdom
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