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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

LIGHTHOUSE Trial 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.

LIGHTHOUSE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to week 52

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

LIGHTHOUSE 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

Do I qualify?Apply below to find out

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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Am I a suitable participant for this experiment?

"This clinical trial is accepting 18 participants with retinoschisis, aged between 6 and 64. Additionally, they must meet the following conditions: Cohort 1 to 3 applicants have to be over 18 years of age but under 65; for cohort 4, patients should be younger than 18; males only affected by mutations in RS1 gene; BCVA of 34-73 ETDRS letters (equivalent to a Snellen acuity range from 20/200 to 20/40)."

Answered by AI

Can people under eighty years of age participate in this research?

"As outlined in the inclusion criteria, individuals between 6 and 64 years old are eligible to enroll in this trial. There is also 2 trials that cater exclusively for patients under 18 and those over 65 respectively."

Answered by AI

Are there any unfilled positions in this research project?

"Confirming the data hosted on clinicaltrials.gov, this experiment is still recruiting patients after its first posting date of July 1st 2023 and latest update on July 7th 2023."

Answered by AI

How many subjects are currently engaged in this research project?

"Affirmative. According to clinicaltrials.gov, this medical research project is still seeking enrolment which began on July 1st 2023 and was most recently updated on the 7th of July that same year. 18 subjects are required from one site only."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2023. "ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05878860.

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