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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Low dose AAV-RPE65 | Experimental | Subretinal administration of a single low dose of range AAV-RPE65 |
|
| Intermediate dose AAV-RPE65 | Experimental | Subretinal administration of a single intermediate dose of range AAV-RPE65 |
|
| High dose AAV-RPE65 | Experimental | Subretinal administration of a single high dose of range AAV-RPE65 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAV RPE65 | Biological | Comparison of different dosages of AAV RPE65 |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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:
| 6 months |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kellogg Eye Centre, University of Michigan | Ann Arbor | Michigan | 48105 | United States | ||
| Moorfields Eye Hospital NHS Foundation Trust |
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| ID | Title | Description |
|---|---|---|
| FG000 | Low Dose AAV-RPE65 | Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at low dose (1 x 10^11 vg/mL) |
| FG001 | Intermediate Dose AAV-RPE65 | Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at intermediate dose (3 x 10^11 vg/mL) |
| FG002 | High Dose AAV-RPE65 | Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at high dose (1 x 10^12 vg/mL) |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Low Dose AAV-RPE65 | Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at low dose (1 x 10^11 vg/mL) |
| BG001 | Intermediate Dose AAV-RPE65 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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:
| Posted | Count of Participants | Participants | 6 months |
|
Within 6 months of ATIMP administration.
A serious adverse event that started on or after the start of the administration of ATIMP. Any serious adverse events that occurred after the last administration of ATIMP + 6 months were not considered treatment emergent.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Low Dose AAV-RPE65 | Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at low dose (1 x 10^11 vg/mL) |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Uveitis | Eye disorders | Systematic Assessment | ATIMP Related |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Uveitis | Eye disorders | Systematic Assessment | ATIMP Related |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Medical Officer | MeiraGTx | +1 973 609-2156 | robert.zeldin@meiragtx.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jun 11, 2018 | Mar 9, 2021 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D057130 | Leber Congenital Amaurosis |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D012164 | Retinal Diseases |
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AAV2/5-OPTIRPE65
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| London |
| EC1V 2PD |
| United Kingdom |
Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at intermediate dose (3 x 10^11 vg/mL)
| BG002 | High Dose AAV-RPE65 | Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at high dose (1 x 10^12 vg/mL) |
| BG003 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at intermediate dose (3 x 10^11 vg/mL)
| OG002 | High Dose AAV-RPE65 | Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at high dose (1 x 10^12 vg/mL) |
|
|
| 0 |
| 9 |
| 0 |
| 9 |
| 5 |
| 9 |
| EG001 | Intermediate Dose AAV-RPE65 | Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at intermediate dose (3 x 10^11 vg/mL) | 0 | 3 | 2 | 3 | 2 | 3 |
| EG002 | High Dose AAV-RPE65 | Unilateral sub-retinal injection of AAV2/5-OPTIRPE65 in a total volume of no more than 1mL at high dose (1 x 10^12 vg/mL) | 0 | 3 | 1 | 3 | 3 | 3 |
| Visual acuity reduced | Eye disorders | Systematic Assessment | ATIMP Related |
|
| Visual acuity reduced | Eye disorders | Systematic Assessment | ATIMP Related |
|
| Anterior chamber inflammation | Eye disorders | Systematic Assessment | ATIMP Related |
|
| Hypotony of eye | Eye disorders | Systematic Assessment | ATIMP Related |
|
| Blurred vision | Eye disorders | Systematic Assessment | ATIMP Related |
|
| Intraocular pressure increased | Investigations | Systematic Assessment | ATIMP Related |
|
| Visual field defect | Nervous system disorders | Systematic Assessment | ATIMP Related |
|
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