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Clinical Trial Details — Status: Enrolling by invitation

Administrative data

NCT number NCT06275620
Other study ID # AGTC-RPGR-001 DAWN
Secondary ID
Status Enrolling by invitation
Phase Phase 2
First received
Last updated
Start date November 14, 2023
Est. completion date August 2029

Study information

Verified date March 2024
Source Beacon Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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 in their previously untreated eye. There will be 2 groups of 6 participants, each receiving one of two doses of the study treatment along with the standard corticosteroid regimen, followed by a third group of ~3-6 participants receiving the high dose but with a modified corticosteroid regimen. The first 6 participants will be enrolled in Group 1, followed by Group 2. Group 3 will open for enrollment after data has been reviewed by the DSMC. DSMC review will occur after all 6 Group 1 (high dose) study participants reach post-operative Month 1 and periodically throughout the study.


Recruitment information / eligibility

Status Enrolling by invitation
Enrollment 18
Est. completion date August 2029
Est. primary completion date July 2025
Accepts healthy volunteers No
Gender Male
Age group 12 Years and older
Eligibility Inclusion Criteria: - Be at least 12 years of age - Have one eye previously treated with an AAV vector-based gene therapy designed to provide full-length functioning RPGR protein. - Have a BCVA no better than 78 letters and no worse than 34 letters - Be able to perform all tests of visual and retinal function and structure in both eyes based on the participant's reliability and fixation, per the Investigator's discretion. - Have detectable baseline mean macular sensitivity measured by MAIA microperimetry, as determined by the Investigator and confirmed by the Central Reading Center (CRC). - Have detectable EZ line in the study eye as assessed by SD-OCT and confirmed by the CRC. Exclusion Criteria: - Have other known disease-causing mutations documented in the participant's medical history or identified through a retinal dystrophy gene panel that, in the opinion of the Investigator, would interfere with the potential therapeutic effect of the study agent or the quality of the assessments. - Have pre-existing eye conditions that would preclude the planned surgery, interfere with the interpretation of study endpoints, or increase the risk of surgical complications - Had intraocular surgery within 90 days of study treatment administration. - Have any active ocular/intraocular infection or inflammation - Have a history of steroid-induced raised IOP of >25 mmHg following corticosteroid exposure, despite topical IOP-lowering pharmacologic therapy.

Study Design


Intervention

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

Locations

Country Name City State
United States Boston Children's Hospital Boston Massachusetts
United States Cincinnati Eye Institute Cincinnati Ohio
United States Cleveland Clinic Cleveland Ohio
United States Retina Foundation of the Southwest Dallas Texas
United States University of Florida Jacksonville Florida
United States Bascom Palmer Eye Institute Miami Florida
United States Casey Eye Institute Portland Oregon

Sponsors (1)

Lead Sponsor Collaborator
Beacon Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary The primary safety outcome is the number and proportion of participants experiencing Grade 3 or higher local (ocular) or non-ocular treatment-emergent adverse events, including treatment-emergent serious adverse events (SAEs). Day 0 - Month 12
Secondary Change from baseline in mean sensitivity across the whole grid, as measured by MAIA microperimetry Day 0 - Month 12
Secondary Response, as measured by MAIA microperimetry, where response is defined as a greater than or equal to 7 dB visual sensitivity improvement from baseline in at least 7 loci. Day 0 - Month 12
Secondary Change from baseline in BCVA using Early-Treatment Diabetic Retinopathy Study (ETDRS) visual acuity Day 0 - Month 12
Secondary Change from baseline in LLVA using ETDRS visual acuity Day 0 - Month 12
Secondary Change from baseline in Ora-VNC mobility test score Day 0 - Month 12
See also
  Status Clinical Trial Phase
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Recruiting NCT05926583 - A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa Phase 3
Completed NCT03116113 - A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112 Phase 1/Phase 2
Recruiting NCT04850118 - A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XLRP Phase 2/Phase 3
Active, not recruiting NCT04517149 - 4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP) Phase 1/Phase 2
Enrolling by invitation NCT03584165 - Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa Phase 3
Active, not recruiting NCT04312672 - Long-term Follow-up Gene Therapy Study for RPGR- XLRP
Completed NCT04868916 - An Observational Study of Japanese Participants With X-linked Retinitis Pigmentosa
Completed NCT03252847 - Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR) Phase 1/Phase 2
Recruiting NCT05874310 - Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa Early Phase 1
Active, not recruiting NCT04671433 - Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Phase 3
Active, not recruiting NCT04794101 - Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Phase 3
Completed NCT03314207 - Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
Active, not recruiting NCT03316560 - Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations Phase 1/Phase 2
Active, not recruiting NCT06333249 - A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE) Phase 2