Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2016-000898-20 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This study is a longer-term follow-up study for patients who have been administered AAV2/5-OPTIRPE65 in the Phase I/II, open label, non-randomised, two-centre, dose escalation trial in adults and children with retinal dystrophy associated with defects in RPE65.
The follow up study is designed to collect data on longer-term safety and efficacy of AAV2/5-OPTIRPE65 administration in the OPTIRPE65 trial.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Low dose AAV OPTIRPE65 | subretinal administration of a single low dose of AAV RPE65 |
| |
| Intermediate dose AAV OPTIRPE65 | subretinal administration of a single intermediate dose of AAV RPE65 |
| |
| High dose AAV OPTIRPE65 | subretinal administration of a single highdose of AAV RPE65 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAV OPTIRPE65 | Biological | comparison of different doses of AAV RPE65 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of Adverse Events related to the treatment | Safety is defined as the absence of ATIMP-related safety events | 5 years |
| Measure | Description | Time Frame |
|---|---|---|
| Improvement in the retinal function | Improvements in visual function as assessed by visual assessment | 5 years |
| Improvement in the visual function | Improvements in retinal function as assessed by visual assessment |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
The patient population are those with Leber Congenital Amaurosis (LCA) with gene mutation RPE65 who have participated in the OPTIRPE65 trial.
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kellogg Eye Center, University of Michigan Health | Ann Arbor | Michigan | 48105 | United States | ||
| Moorfields Eye Hospital NHS Foundation Trust |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| 5 years |
| Improvement in quality of life | Improvement in the participant's quality of life which is measurable by QoL questionnaire | 5 years |
| London |
| United Kingdom |
| ID | Term |
|---|---|
| D057130 | Leber Congenital Amaurosis |
| D005128 | Eye Diseases |
| D015785 | Eye Diseases, Hereditary |
| D012164 | Retinal Diseases |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided