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Gene Therapy

Gene Therapy for Retinoschisis (LIGHTHOUSE Trial)

Phase 1 & 2
Recruiting
Research Sponsored by Atsena Therapeutics Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline to week 52
Awards & highlights

Summary

This trial will test a new drug to treat XLRS, a rare eye disorder, in males 6-64 years old, to check safety and tolerability.

Who is the study for?
This trial is for male patients with X-linked retinoschisis (XLRS) due to RS1 mutations. Adults must be between 18 and 64 years old, while children should be aged 6 to under 18. Participants need a specific level of vision clarity. Those who've had previous eye gene therapy or certain eye conditions/surgeries are excluded.Check my eligibility
What is being tested?
The study tests the safety and effects of ATSN-201, a gene therapy for males with XLRS. It will involve different age groups (cohorts) to assess how well they tolerate this potential new treatment.See study design
What are the potential side effects?
While not explicitly listed, side effects may include typical risks associated with gene therapies such as immune reactions, discomfort at injection site, and potential worsening of vision or other ocular issues.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline to week 52
This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to week 52 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events
Secondary outcome measures
Macular structure as assessed by fundus autofluorescence
Macular structure as assessed by spectral domain optical coherence tomography
Subject-reported visual function as assessed by the CVAQC in pediatric subjects
+7 more

Trial Design

6Treatment groups
Experimental Treatment
Active Control
Group I: Cohort 4, PediatricExperimental Treatment1 Intervention
ATSN-201 at High Dose
Group II: Cohort 3, Low DoseExperimental Treatment1 Intervention
ATSN-201 at Low Volume
Group III: Cohort 3, High DoseExperimental Treatment1 Intervention
ATSN-201 at High Volume
Group IV: Cohort 2Experimental Treatment1 Intervention
ATSN-201 at High Dose
Group V: Cohort 1Experimental Treatment1 Intervention
ATSN-201 at Low Dose
Group VI: Cohort 3, ControlActive Control1 Intervention

Find a Location

Who is running the clinical trial?

Atsena Therapeutics Inc.Lead Sponsor
1 Previous Clinical Trials
15 Total Patients Enrolled

Media Library

ATSN-201 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05878860 — Phase 1 & 2
Retinoschisis Research Study Groups: Cohort 1, Cohort 3, Control, Cohort 3, High Dose, Cohort 2, Cohort 3, Low Dose, Cohort 4, Pediatric
Retinoschisis Clinical Trial 2023: ATSN-201 Highlights & Side Effects. Trial Name: NCT05878860 — Phase 1 & 2
ATSN-201 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05878860 — Phase 1 & 2
~10 spots leftby Oct 2025