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

Conditions

• X-Linked Retinitis Pigmentosa

Eligibility Criteria

Sex

MALE

Age

12 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• AGTC-501 (high dose and standard corticosteroid regimen) — BIOLOGICAL
  Adeno-associated virus vector expressing a human RPGR gene
• AGTC-501 (low dose and standard corticosteroid regimen) — BIOLOGICAL
  Adeno-associated virus vector expressing a human RPGR gene
• AGTC-501 (high dose and modified corticosteroid regimen) — BIOLOGICAL
  Adeno-associated virus vector expressing a human RPGR gene

Study Details

This Phase 2 study is a non-randomized, open-label, study of the safety of AGTC-501 in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein.

Key Dates

Start date

Nov 14, 2023

Status verified

Oct 2024

Primary completion

Nov 30, 2025

Completion

Dec 31, 2029

Study Design

Enrollment

24 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Group 1 (High Dose, Standard Corticosteroid)
  Following a pars plana vitrectomy, the previously untreated eye of participants (n=up to 12) will receive a central subretinal injection at the high dose in the study eye at the Baseline visit; no treatment will be administered in the previously treated fellow eye. The corticosteroid taper for all treated participants in Groups 1 and 2 (high and low doses with standard corticosteroid regimen) will be a standard taper over the course of several weeks.
• Experimental: Group 2 (Low Dose, Standard Corticosteroid)
  Following a pars plana vitrectomy, the previously untreated eye of participants (n=6) will receive a central subretinal injection at the low dose in the study eye at the Baseline visit; no treatment will be administered in the previously treated fellow eye. The corticosteroid taper for all treated participants in Groups 1 and 2 (high and low doses with standard corticosteroid regimen) will be a standard taper over the course of several weeks.
• Experimental: Group 3 (High Dose, Modified Corticosteroid)
  Following a pars plana vitrectomy, the previously untreated eye of participants (n=approximately 3-6) will receive a central subretinal injection at the high dose in the study eye at the Baseline visit; no treatment will be administered in the previously treated fellow eye. Participants in Group 3 (high dose, modified corticosteroid) will have a more rapid corticosteroid taper.

Primary Outcome Measure

The primary safety outcome is the number of participants experiencing Grade 3 or higher local (ocular) or non-ocular treatment-emergent adverse events, including treatment-emergent serious adverse events (SAEs). [ Time Frame: Day 0 - Month 12 ]

Locations (7)

Find similar trials in Jacksonville, FL

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