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Virus Therapy

AGTC-501 for Retinitis Pigmentosa

Phase 2 & 3

Recruiting

Research Sponsored by Beacon Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up month 12, 18 and 24

Awards & highlights

No Placebo-Only Group

Summary

This trial will test two different doses of a gene therapy called AGTC-501 to see how safe and effective it is in treating a certain type of eye disease in men.

Who is the study for?

This trial is for males aged 8-50 with X-linked retinitis pigmentosa (XLRP) due to RPGR gene mutations. Participants must have vision that falls within a specific range, not too good and not too poor, as measured by an eye chart test.

What is being tested?

The study tests two doses of AGTC-501, a gene therapy delivered via injection into the eye, against no treatment in patients with XLRP. It aims to assess the safety and effectiveness of this potential new treatment.

What are the potential side effects?

While specific side effects are not listed here, similar treatments may cause eye irritation or inflammation, changes in vision, discomfort at the injection site, and possible immune reactions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ month 12, 18 and 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~month 12, 18 and 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and month 12, 18 and 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

The proportion of participants with a ≥15 letter increase from baseline in LLVA

Secondary study objectives

Change from baseline in BCVA over time as assessed by an Early Treatment of Diabetic Retinopathy (ETDRS) or Tumbling E chart at Month 12

Change from baseline in BCVA over time as assessed by an Early Treatment of Diabetic Retinopathy (ETDRS) or Tumbling E chart at Month 18 and 24

Change from baseline in full-field stimulus threshold (FST)

+15 more

Other study objectives

Number and proportion of treatment-emergent ocular/non-ocular adverse events

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

3Treatment groups

Experimental Treatment

Active Control

Group I: Group 3: ControlExperimental Treatment2 Interventions

Male participants 12-50 years of age in the untreated control group. Participants in the control group will be followed for a minimum of 24 months. After all participants have reached Month 12, participants in the control group will be given the option to receive the study drug in the fellow eye, if eligible.

Group II: Group 1: DoseActive Control1 Intervention

Male participants 12-50 years of age treated by subretinal injection with the of AGTC-501

Group III: Group 2: DoseActive Control1 Intervention

Male participants 12-50 years of age treated by subretinal injection with the dose of AGTC-501

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Control

2011

Completed Phase 4

~15780