Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

• Register: ClinicalTrials.gov
• Last refreshed on: 27 February 2024
• Main ID: NCT03316560
• Date of registration: 10/10/2017
• Prospective Registration: Yes
• Primary sponsor: Beacon Therapeutics
• Public title: Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations HORIZON
• Scientific title: A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene
• Date of first enrolment: April 16, 2018
• Target sample size: 29
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03316560
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Phase 1/2 Dose Escalation Inclusion Criteria:

- Male subjects with a documented RPGR mutation

- Clinical diagnosis of X-linked retinitis pigmentosa (XLRP)

- Best-corrected visual acuity not better than 78 ETDRS letters (20/32) in the study
eye;

- Ability to perform tests of visual and retinal function and structure and ability to
comply with other research procedures;

- Detectable baseline mean macular sensitivity, as measured by microperimetry.

- Have detectable Ellipsoid Zone (EZ) line during the pre-treatment period as assessed
by OCT and confirmed by the CRC.

Phase 1/2 Dose Escalation Exclusion Criteria:

- Pre-existing eye conditions that would preclude the planned surgery or interfere with
the interpretation of study endpoints or increase the risk of surgical complications
(for example, glaucoma, corneal or lenticular opacities, diabetic retinopathy, retinal
vasculitis);

- Use of anti-coagulant agents that may alter coagulation within 7 days prior to study
agent administration;

- Use of systemic corticosteroids or other immunosuppressive medications within 3 months
prior to enrollment;

- Any other condition that would prevent a subject from completing follow-up
examinations during the course of the study;

- Any other condition or reason that, in the opinion of the investigator, makes the
subject unsuitable for the study;

- Previous receipt of any AAV gene therapy product;

- Monocular or having BCVA less than 20/800 in the fellow eye



Age minimum: 6 Years
Age maximum: 50 Years
Gender: Male

Health Condition(s) or Problem(s) studied

X-Linked Retinitis Pigmentosa

Intervention(s)

Biological: rAAV2tYF-GRK1-RPGR

Primary Outcome(s)

Number and proportion of participants experiencing abnormal clinically relevant hematology or clinical chemistry parameters. [Time Frame: Day 0 - Month 36]

Number and proportion of Adverse Events [Time Frame: Day 0 - Month 36]

Secondary Outcome(s)

Changes from baseline in visual function as measured by mesopic microperimetry in the treated eye compared to the untreated eye [Time Frame: Day 0 - Month 36]

Change from baseline in visual function by light-adapted perimetry [Time Frame: Day 0 - Month 36]

Changes from baseline in visual function by dark-adapted full field perimetry (for subjects treated peripherally) [Time Frame: Day 0 - Month 36]

Changes from baseline in visual acuity [Time Frame: Day 0 - Month 36]

Change from baseline in fundus imaging [Time Frame: Day 0 - Month 36]

Changes from baseline in retinal structure as assessed by spectral-domain optical coherence tomography (SD-OCT) [Time Frame: Day 0 - Month 36]

Change from baseline in full-field light sensitivity threshold (FST) [Time Frame: Day 0 - Month 36]

Changes from baseline in quality of life questionnaire responses [Time Frame: Day 0 - Month 36]

Secondary ID(s)

AGTC-RPGR-001 HORIZON

Source(s) of Monetary Support

Please refer to primary and secondary sponsors