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Gene Therapy

Low Dose Group for Retinitis Pigmentosa

Phase 2

Waitlist Available

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 day 0 - month 12

Awards & highlights

Study Summary

This trial will test a new treatment for a genetic eye disorder called X-linked retinitis pigmentosa.

Who is the study for?

This trial is for male subjects with a specific eye condition called X-linked Retinitis Pigmentosa, which must be caused by mutations in the RPGR gene. The eligibility criteria are not fully listed here, so additional requirements may apply.Check my eligibility

What is being tested?

The study is testing the safety and effectiveness of a gene therapy named rAAV2tYF-GRK1-RPGR on patients with X-linked Retinitis Pigmentosa due to RPGR mutations. It will compare two different doses of this treatment.See study design

What are the potential side effects?

Potential side effects are not detailed here but could include typical reactions to gene therapies such as immune responses, inflammation at injection sites, or changes in vision.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 0 - month 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 0 - month 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 0 - month 12 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 difference in the proportion of responding eyes between treated and control eyes in the low dose group and high dose group at 12 months, as measured by MAIA microperimetry, where response is defined as a 7dB or more improvement in at least 5 loci.

Secondary outcome measures

Difference in mean change from baseline in "within bleb" mean sensitivity, as measured by MAIA microperimetry, in treated eyes versus control eyes in the low dose group and high dose group at Month 12.

Difference in mean change from baseline in BCVA, as measured by ETDRS or tumbling "E" chart, in treated eyes versus control eyes in the low dose group and high dose group at Month 12.

Difference in mean change from baseline in central 10 degrees of vision on light adapted static perimetry, as measured by Octopus 900, in treated eyes versus control eyes in the low dose group and high dose group at Month 12.

+8 more

Trial Design

2Treatment groups

Experimental Treatment

Group I: Low Dose GroupExperimental Treatment1 Intervention

Male subjects at least 8 y/o treated with a lower dose (Dose 2 in RPGR-001 Horizon Phase 1/2 study) of rAAV2tYF-GRK1-RPGR study drug.

Group II: High Dose GroupExperimental Treatment1 Intervention

Male subjects at least 8 y/o treated with a higher dose (Dose 5 in RPGR-001 Horizon Phase 1/2 study) of rAAV2tYF-GRK1-RPGR study drug.

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Beacon TherapeuticsLead Sponsor

3 Previous Clinical Trials

122 Total Patients Enrolled

3 Trials studying Retinitis Pigmentosa

122 Patients Enrolled for Retinitis Pigmentosa

Frequently Asked Questions

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

Which individuals meet the criteria for participating in this medical study?

"Individuals aged between 8 and 50 diagnosed with retinitis pigmentosa are sought to fill approximately 14 openings in this research study."

Answered by AI

Are individuals currently eligible to participate in this research study?

"As per clinicaltrials.gov, patient enrollment for this particular trial is not active. Even though the study was initially made public on April 13th, 2021 and last revised on March 20th, 2024, recruitment has ceased. Nevertheless, there are currently 53 alternative trials actively seeking participants."

Answered by AI

How prevalent is the participation of medical centers in this study within the United States?

"This research is being conducted at the Cincinnati Eye Institute in Ohio, Cleveland Clinic in Oregon, and Casey Eye Institute in Texas. Additionally, there are six supplementary locations where this study will take place."

Answered by AI

Is the research trial open to participants who have reached adulthood, typically considered as 18 years or older?

"Individuals aged between 8 and 50 years old are eligible for participation based on the trial's entry criteria."

Answered by AI

What is the safety profile of the low-dose treatment arm for participants?

"Based on our evaluation at Power, the safety rating for the Low Dose Group is a 2. This assessment arises from Phase 2 trial results indicating some level of safety but limited efficacy data available."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)

2021. "A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06333249.

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