NCT06275620

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

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 Participants With X-linked Retinitis Pigmentosa

• Status: Enrolling By Invitation
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Beacon Therapeutics · Industry
• Sex: Male
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This is a Phase 2, open-label, multicenter study to evaluate the safety of 2 doses of AGTC-501 administered as a single subretinal injection in participants with XLRP who have previously been treated with a full-length AAV vector-based gene therapy targeting RPGR protein. The trial includes a screening period of up to 60 days and a 5 year study period. Each participant will receive a single subretinal injection of one of two dose levels of AGTC-501 in their previously untreated eye. There will be 3 groups. Group 1 will receive the high dose and include up to 12 participants, Group 2 will receive the low dose and will include 6 participants, and Group 3 will include \~3-6 participants. Participants in Groups 1 and 2 will receive the standard corticosteroid regimen. A single subretinal injection of the high dose AGTC-501 will be administered to participants in Group 1 (n = 12), while participants in Group 2 (n = 6) will receive a single subretinal injection of low dose AGTC-501. Group 2 (low dose AGTC-501, Standard Steroid) will be dosed before moving to Group 3. After 6 Group 1 (high dose) study participants reach post-operative Month 1, all data will be reviewed by the DSMC. If no safety signals arise, additional participants, Group 3 (n \~ 3-6), will receive a single subretinal injection of the high dose with a modified course of corticosteroids.

Conditions

• X-Linked Retinitis Pigmentosa

Interventions

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

Timeline

• Start date: 2023-11-14
• Primary completion: 2025-11-01
• Completion: 2029-12-01
• First posted: 2024-02-23
• Last updated: 2024-10-30

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study