A Phase 1/2 interventional study of AAV RPE65 in Leber Congenital Amaurosis, sponsored by MeiraGTx UK II Ltd. Completed at 2 sites in 2 countries. Open to participants aged 3 Years and older. Per ClinicalTrials.gov, last updated 2021-07-12.
Sponsored by MeiraGTx UK II Ltd · Phase 1/2, Interventional, and Treatment
A clinical trial of AAV2/5 vector for patients with Defects in RPE65
A dose escalation and dose expansion (Phase I/II) trial of adults and children with retinal dystrophy associated with defects in RPE65. ATIMP will be administered to one eye only in a single sub-retinal procedure
161 studies on the registry are indexed under Blindness; 33 are open to participants now.
This study's enrollment of 15 is below the median of 30 across 105 interventional studies indexed under Blindness.
Browse Blindness studies →MeiraGTx UK II Ltd is the lead sponsor of 9 studies on the registry; none are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 4 (80%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Subretinal administration of a single low dose of range AAV-RPE65
Biological: AAV RPE65
Subretinal administration of a single intermediate dose of range AAV-RPE65
Biological: AAV RPE65
Subretinal administration of a single high dose of range AAV-RPE65
Biological: AAV RPE65
Comparison of different dosages of AAV RPE65
Overall Safety of Adeno-Associated Virus Vector (AAV-OPTIRPE65) - Number of Participants With a Safety Event
Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more. * Severe unresponsive inflammation. * Infective endophthalmitis. * Ocular malignancy. * Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).
Time frame: 6 months
| Milestone | Low Dose AAV-RPE65 | Intermediate Dose AAV-RPE65 | High Dose AAV-RPE65 |
|---|---|---|---|
| Started | 9 | 3 | 3 |
| Completed | 9 | 3 | 3 |
| Not completed | 0 | 0 | 0 |
Safety was defined as an advanced therapy investigational medicinal product (ATIMP) related: * Reduction in visual acuity by 15 Early Treatment Diabetic Retinopathy Study (ETDRS) letters or more. * Severe unresponsive inflammation. * Infective endophthalmitis. * Ocular malignancy. * Grade III or above non-ocular suspected unexpected serious adverse reaction (SUSAR).
| Participants | Low Dose AAV-RPE65 | Intermediate Dose AAV-RPE65 | High Dose AAV-RPE65 |
|---|---|---|---|
| Number of subjects with dose limiting events. | 0 | 0 | 1 |
| Reduction in visual acuity by 15 ETDRS | 0 | 0 | 1 |
| Severe unresponsive inflammation | 0 | 0 | 0 |
| Infective endophthalmitis | 0 | 0 | 0 |
| Ocular malignancy | 0 | 0 | 0 |
| Grade III or above non-ocular SUSAR | 0 | 0 | 0 |
Collected over Within 6 months of ATIMP administration.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Low Dose AAV-RPE65 | 0/9 (0%) | 0/9 (0%) | 5/9 (55.6%) |
| Intermediate Dose AAV-RPE65 | 0/3 (0%) | 2/3 (66.7%) | 2/3 (66.7%) |
| High Dose AAV-RPE65 | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| Event | Low Dose AAV-RPE65 | Intermediate Dose AAV-RPE65 | High Dose AAV-RPE65 |
|---|---|---|---|
| UveitisEye disorders | 0/9 | 2/3 | 1/3 |
| Visual acuity reducedEye disorders | 0/9 | 0/3 | 1/3 |
| Event | Low Dose AAV-RPE65 | Intermediate Dose AAV-RPE65 | High Dose AAV-RPE65 |
|---|---|---|---|
| UveitisEye disorders | 3/9 | 1/3 | 1/3 |
| Visual acuity reducedEye disorders | 2/9 | 0/3 | 1/3 |
| Anterior chamber inflammationEye disorders | 0/9 | 0/3 | 1/3 |
| Blurred visionEye disorders | 0/9 | 1/3 | 0/3 |
| Visual field defectNervous system disorders | 0/9 | 1/3 | 0/3 |
| Hypotony of eyeEye disorders | 1/9 | 0/3 | 0/3 |
| Intraocular pressure increasedInvestigations | 1/9 | 0/3 | 0/3 |
| Age, Continuous(years) | Low Dose AAV-RPE65 | Intermediate Dose AAV-RPE65 | High Dose AAV-RPE65 | Total |
|---|---|---|---|---|
| Mean | 12.8 ± 5.52 | 19.0 ± 2.65 | 21.0 ± 3.00 | 15.7 ± 5.79 |
| Sex: Female, Male(Participants) | Low Dose AAV-RPE65 | Intermediate Dose AAV-RPE65 | High Dose AAV-RPE65 | Total |
|---|---|---|---|---|
| Female | 7 | 2 | 0 | 9 |
| Male | 2 | 1 | 3 | 6 |
| Race/Ethnicity, Customized(Participants) | Low Dose AAV-RPE65 | Intermediate Dose AAV-RPE65 | High Dose AAV-RPE65 | Total |
|---|---|---|---|---|
| Hispanic or Latino | 0 | 1 | 0 | 1 |
| Not Hispanic or Latino | 9 | 2 | 3 | 14 |
| Black or African American | 0 | 0 | 1 | 1 |
| Asian | 2 | 0 | 0 | 2 |
| White | 7 | 3 | 2 | 12 |
| Region of Enrollment(participants) | Low Dose AAV-RPE65 | Intermediate Dose AAV-RPE65 | High Dose AAV-RPE65 | Total |
|---|---|---|---|---|
| United States | 2 | 0 | 0 | 2 |
| United Kingdom | 7 | 3 | 3 | 13 |
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MeiraGTx UK II Ltd