Health TrialsbyHealthPanel

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

ConditionX-linked Retinoschisis
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age5+
SponsorAtsena Therapeutics Inc.

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Age ≥ 5 years.

Trial design

Design model

Sequential

Treatments tested in this trial

  • ATSN-201

    Biological/Vaccine

    AAV.SPR-hGRK1-hRS1syn

Treatment groups

97 Participants
are divided into 9 treatment groups

9

Treatment groups

See each treatment group below.

Group A: Cohort 1, Low DoseExperimental treatment 1 intervention
Group B: Cohort 2, High DoseExperimental treatment 1 intervention
Group C: Cohort 3, Mid DoseExperimental treatment 1 intervention
Group D: Cohort 4, Low VolumeExperimental treatment 1 intervention
Group E: Cohort 4, High VolumeExperimental treatment 1 intervention
Group F: Cohort 4, ControlNo intervention 0 interventions
Group G: Cohort 5, PediatricExperimental treatment 1 intervention
Group H: Cohort 6, TreatmentExperimental treatment 1 intervention
Group I: Cohort 6, ControlNo intervention 0 interventions

Trial outcomes

Primary outcomes

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).

Time frame
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.

Time frame
From baseline to week 52

Secondary outcomes

1

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

Change in best-corrected visual acuity (BCVA).

Time frame
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).

Time frame
From baseline to week 52
3

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

Change in contrast sensitivity.

Time frame
From baseline to week 52
4

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

Change in microperimetry.

Time frame
From baseline to week 52

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Atsena Therapeutics Inc.

Lead sponsor

Source ClinicalTrials.gov
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