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Virus Therapy

Group 3: Control 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

Study 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.Check my eligibility

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.See study design

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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

Secondary outcome measures

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 outcome measures

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

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

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Beacon TherapeuticsLead Sponsor

3 Previous Clinical Trials

61 Total Patients Enrolled

3 Trials studying Retinitis Pigmentosa

61 Patients Enrolled for Retinitis Pigmentosa

Applied Genetic Technologies CorpLead Sponsor

10 Previous Clinical Trials

278 Total Patients Enrolled

2 Trials studying Retinitis Pigmentosa

43 Patients Enrolled for Retinitis Pigmentosa

Carrie ReichleyStudy DirectorBeacon Therapeutics

1 Previous Clinical Trials

1,219 Total Patients Enrolled

Frequently Asked Questions

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

Are prospective participants currently able to enroll in this medical study?

"Yes, the details on clinicaltrials.gov indicate that this particular research study is actively seeking suitable participants. The trial was initially listed on March 14th, 2024, and its most recent update was on March 27th, 2024. This investigation aims to enroll a total of 75 patients from two distinct locations."

Answered by AI

What is the upper limit for patient recruitment in this particular research endeavor?

"Yes, information found on clinicaltrials.gov indicates that this research study is currently seeking eligible participants. Initially shared on March 14, 2024 and last modified on March 27, 2024, the trial aims to enroll a total of 75 individuals distributed across two designated locations."

Answered by AI

Are individuals older than 60 years old being sought for enrollment in this study?

"To partake in this research, individuals must be at least 12 years old but no older than 50."

Answered by AI

Is this a study that has never been conducted before?

"Group 3, known as the Control group, has been under scrutiny since 2018. The inaugural trial in this series was funded by Beacon Therapeutics and encompassed a cohort of 29 participants. Subsequently, Group 3: Control gained authorization for both Phase 1 and Phase 2 trials. Presently, there are two ongoing clinical investigations for Group 3: Control spanning seven urban centers within one nation."

Answered by AI

Which individuals meet the eligibility criteria to participate in this experimental study?

"The research is enrolling 75 individuals diagnosed with retinitis pigmentosa aged between 12 and 50 years. Specifically, male candidates in the age range of 8-50 who have a verified RPGR mutation leading to X-linked retinitis pigmentosa are sought after for this trial if they also fulfill all other stipulated entry criteria. Furthermore, eligible participants must possess at least one confirmed harmful or probably harmful alteration within exons 1-14 and/or ORF15 of the RPGR gene, alongside a Best Corrected Visual Acuity (BCVA) falling between no more than 75 letters (20/32)"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP

2024. "A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04850118.

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