A Phase 2 interventional study of AGTC-501 (high dose and standard corticosteroid regimen) and AGTC-501 (low dose and standard corticosteroid regimen) in X-Linked Retinitis Pigmentosa, sponsored by Beacon Therapeutics. Enrolling by invitation at 7 sites in United States. Open to male participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2024-10-30.
Sponsored by Beacon Therapeutics · Phase 2, Interventional, and Treatment
Among 84 studies in Retinal Degeneration with a site in United States — 11 open to participants now, 29 under way, 3 of them phase 3. Retinal Degeneration · United States.
Among the 84 studies in Retinal Degeneration with a site in United States, this one is the 45th largest by planned enrollment of the 62 interventional ones that give a count, is one of 11 that started in 2023, is one of 16 in phase 2, reaches primary completion later than 3 of the 26 other live ones, and was the 77th registered.
132 studies on the registry are indexed under Retinal Degeneration; 23 are open to participants now.
This study's planned enrollment of 24 is below the median of 40 across 98 interventional studies indexed under Retinal Degeneration.
Browse Retinal Degeneration studies →11 of the 84 studies in Retinal Degeneration with a site in United States are open now, in 6 countries. Each count lists the studies.
Of the 15 Retinal Degeneration studies recruiting now, 10 (67%) set no upper age limit and 3 (20%) stop at 75 or younger; 7 take children under 18; 5 take healthy volunteers.
This study: 12 Years and older · male · no healthy volunteers.
The full table on the hub →84 studies; the count open now beside each. This study's row is marked.
First in 1978; this study’s year is marked. 11 started in 2023.
Median enrollment 51 across the 62 interventional studies, 83 across the 21 observational. Of the 50 that completed or were terminated, 27 (54%) have results posted.
58 of 84 have an industry lead sponsor. The 10 most frequent:
Beacon Therapeutics is the lead sponsor of 13 studies on the registry; 1 is open to participants now.
Beacon Therapeutics has 7 studies filed under Retinal Degeneration — fewer than ten, so there is no comparison to draw.
7 of the studies in Retinal Degeneration with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04704921Phase 3AbbViePivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMD | 2026-12 |
| NCT05748873SparingVisionPromising ROd-cone DYstrophy Gene therapY | 2027-02 |
| NCT06167642Johns Hopkins UniversityRetinal Atrophy and Neurofilament Light Chain in People With Multiple Sclerosis Taking Ofatumumab | 2027-03 |
| NCT06048185Nanoscope Therapeutics Inc.Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study | 2027-05 |
| NCT04765345Jaeb Center for Health ResearchRate of Progression of PCDH15-Related Retinal Degeneration in Usher Syndrome 1F | 2027-06-30 |
Among the 84 studies in Retinal Degeneration with a site in United States, from their registry updates:
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.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Biological: AGTC-501 (high dose and standard corticosteroid regimen)
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.
Biological: AGTC-501 (low dose and standard corticosteroid regimen)
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.
Biological: AGTC-501 (high dose and modified corticosteroid regimen)
Adeno-associated virus vector expressing a human RPGR gene
Adeno-associated virus vector expressing a human RPGR gene
Adeno-associated virus vector expressing a human RPGR gene
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
The primary safety outcome is the proportion 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
The number of participants experiencing treatment-emergent AEs of ocular/non-ocular adverse events, including treatment-emergent serious AEs.
Time frame: Day 0 - Month 12
The proportion of participants experiencing treatment-emergent AEs of ocular/non-ocular adverse events, including treatment-emergent serious AEs.
Time frame: Day 0 - Month 12
Change from baseline in mean sensitivity across the whole grid, as measured by MAIA (Macular Integrity Assessment) microperimetry, assess photoreceptor function under low light
Time frame: Day 0 - Month 12
Response, as measured by MAIA (Macular Integrity Assessment) microperimetry, where response is defined as a greater than or equal to 7 decibel (dB) visual sensitivity improvement from baseline in at least 5 loci.
Time frame: Day 0 - Month 12
Change from baseline in full-field stimulus threshold (FST)
As assessed by full-field stimulus threshold (FST); FST measures the sensitivity of the visual field by testing for the lowest luminance flash which elicits a visual sensation perceived
Time frame: Day 0 - Month 12
Change from baseline in Best Corrected Visual Acuity (BCVA) using Early-Treatment Diabetic Retinopathy Study (ETDRS) visual acuity
Time frame: Day 0 - Month 12
Change from baseline in Low Luminance Visual Acuity (LLVA) using Early-Treatment Diabetic Retinopathy Study (ETDRS) visual acuity
Time frame: Day 0 - Month 12
Proportion of responding eyes in treated versus control eyes at Month 12 where responder is defined as an improvement of at least 15-letters on low-luminance visual acuity (LLVA)
Time frame: Day 0 - Month 12
Change from baseline in ellipsoid zone (EZ) area measured by spectral domain optical coherence tomography (SD OCT)
Time frame: Day 0 - Month 12
Change from baseline in seven domain scores from a Michigan Retinal Degeneration Questionnaire (MRDQ)
Time frame: Day 0 - Month 12
Change from baseline in Ora-VNC (visual navigation course) mobility test score
As assessed by functional assessment Ora-VNC (visual navigation course) mobility course
Time frame: Day 0 - Month 12
Change from baseline in the MObility Standardized Test-Virtual Reality (MOST-VR) mobility course test score
As measured by the MObility Standardized Test-Virtual Reality (MOST-VR) mobility course
Time frame: Day 0 - Month 12
Started Nov 2023 (actual). Primary completion is estimated for Nov 2025, 24 months after the start.
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Beacon Therapeutics