A Phase 1/2 interventional study of rAAV2tYF-GRK1-RPGR in X-Linked Retinitis Pigmentosa, sponsored by Beacon Therapeutics. Active, not recruiting at 4 sites in United States. Open to male participants aged 6 Years to 50 Years. Per ClinicalTrials.gov, last updated 2024-05-20.
Sponsored by Beacon Therapeutics · Phase 1/2, 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 42nd largest by enrollment of the 62 interventional ones that give a count, is one of 6 that started in 2018, is one of 14 in phase 1/2, reaches primary completion later than 2 of the 26 other live ones, and was the 25th registered.
132 studies on the registry are indexed under Retinal Degeneration; 23 are open to participants now.
This study's enrollment of 29 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.
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
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: 6 Years to 50 Years · male · no healthy volunteers.
The full table on the hub →84 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 24 · Phase 2 33 · Phase 3 13 · Phase 4 2
First in 1978; this study’s year is marked. 6 started in 2018.
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 study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
This protocol includes a non-randomized, open-label, Phase 1/2 study (HORIZON). Approximately 30 participants will be enrolled into the dose escalation study (HORIZON). Each participant will receive the study agent by subretinal injection in one eye on a single occasion. Enrollment will begin with the lowest dose and will proceed to higher doses only after review of safety data by a Data and Safety Monitoring Committee (DSMC). There are a total of 15 visits over approximately 36 months, and long-term follow-up evaluations annually at years 4 and 5.
Phase 1/2 Dose Escalation Inclusion Criteria:
Phase 1/2 Dose Escalation 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.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
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.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Male subjects at least 18 y/o treated with Dose 1 of rAAV2tYF-GRK1-RPGR study drug.
Biological: rAAV2tYF-GRK1-RPGR
Male subjects at least 18 y/o treated with Dose 2 of rAAV2tYF-GRK1-RPGR study drug.
Biological: rAAV2tYF-GRK1-RPGR
Group 3 male subjects at least 18 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.
Biological: rAAV2tYF-GRK1-RPGR
Group 4 male subjects at least 6 y/o treated with Dose 3 of rAAV2tYF-GRK1-RPGR study drug.
Biological: rAAV2tYF-GRK1-RPGR
Male subjects at least 18 y/o treated with Dose 5 of rAAV2tYF-GRK1-RPGR study drug.
Biological: rAAV2tYF-GRK1-RPGR
Male subjects at least 18 y/o treated with Dose 6 of rAAV2tYF-GRK1-RPGR study drug.
Biological: rAAV2tYF-GRK1-RPGR
Adeno-associated virus vector expressing a human RPGR gene
Number and proportion of Adverse Events
Number and proportion of participants experiencing Grade 3 or higher local (ocular) or systemic treatment-emergent adverse events that occur during the 36 months after study agent administration; number and proportion of participants experiencing treatment-emergent AEs, including treatment-emergent serious AEs;
Time frame: Day 0 - Month 36
Number and proportion of participants experiencing abnormal clinically relevant hematology or clinical chemistry parameters.
Time frame: Day 0 - Month 36
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
Changes from baseline in visual acuity
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
Changes from baseline in quality of life questionnaire responses
Time frame: Day 0 - Month 36
Change from baseline in visual function by light-adapted perimetry
Time frame: Day 0 - Month 36
Change from baseline in fundus imaging
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 visual function by dark-adapted full field perimetry (for subjects treated peripherally)
Time frame: Day 0 - Month 36
Plan to share: No
No publications or documents are linked to this record.
Ran from Apr 2018 to primary completion in Nov 2023, 66 months.
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This study is active, not recruiting, as verified in May 2024. You cannot join it, but the record below documents what was studied.
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Beacon Therapeutics