Ignite Creation Date:
2024-05-06 @ 3:33 AM
Last Modification Date:
2024-10-26 @ 11:35 AM
Study NCT ID:
NCT02317887
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-04-26
First Post:
2014-12-16
Brief Title:
Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
Sponsor:
VegaVect Inc
Organization:
VegaVect Inc
Study Overview
Official Title:
A Phase IIIa Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-04-13
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Background
- X-linked juvenile retinoschisis XLRS is caused by changes in the RS1 gene These changes cause abnormal function of the eye protein retinoschisin Without normal retinoschisin the layers of the retina split and vision is lost Researchers want to try to introduce a healthy RS1 gene into eye cells to see if this helps retinal cells make healthy retinoschisin They will put the gene in a virus The gene and virus package is known as a gene transfer vector AAV-RS1 vector
Objectives
- To see if the AAV-RS1 vector is safe to use in people
Eligibility
- Adults 18 and older with a mutation of the RS1 gene 2063 vision or worse in one eye and XLRS
Design
Participants will be screened with genetic tests to confirm XLRS They will have a medical history and physical and eye exams
At visits 1-2 participants will have some or all of the following
Medical history
Physical exam
Blood and urine tests
Tuberculosis skin test
Eye exam
Vision tests for one test an intravenous line will be placed in the arm A dye will be injected that will travel to the blood vessels in the eye
At visit 3 the AAV-RS1 vector will be injected with a needle in the study eye Participants pupils will be dilated They will get numbing eye drops
Visits 4-13 will occur in the 18 months after gene transfer Many of the above tests will be repeated Participants will discuss any side effects
Visits 14-17 will occur yearly between years 2 and 5
After year 5 participants will be contacted yearly by phone for up to 15 years
- X-linked juvenile retinoschisis XLRS is caused by changes in the RS1 gene These changes cause abnormal function of the eye protein retinoschisin Without normal retinoschisin the layers of the retina split and vision is lost Researchers want to try to introduce a healthy RS1 gene into eye cells to see if this helps retinal cells make healthy retinoschisin They will put the gene in a virus The gene and virus package is known as a gene transfer vector AAV-RS1 vector
Objectives
- To see if the AAV-RS1 vector is safe to use in people
Eligibility
- Adults 18 and older with a mutation of the RS1 gene 2063 vision or worse in one eye and XLRS
Design
Participants will be screened with genetic tests to confirm XLRS They will have a medical history and physical and eye exams
At visits 1-2 participants will have some or all of the following
Medical history
Physical exam
Blood and urine tests
Tuberculosis skin test
Eye exam
Vision tests for one test an intravenous line will be placed in the arm A dye will be injected that will travel to the blood vessels in the eye
At visit 3 the AAV-RS1 vector will be injected with a needle in the study eye Participants pupils will be dilated They will get numbing eye drops
Visits 4-13 will occur in the 18 months after gene transfer Many of the above tests will be repeated Participants will discuss any side effects
Visits 14-17 will occur yearly between years 2 and 5
After year 5 participants will be contacted yearly by phone for up to 15 years
Detailed Description:
Objective To evaluate the safety and tolerability of ocular AAV-RS1 vector AAV8-scRSIRBPhRS gene transfer to the retina of participants affected with X-linked juvenile retinoschisis XLRS
Study Population Male participants affected with XLRS will receive ocular gene transfer A maximum of up to 24 participants may be enrolled
Design This is a Phase IIIa prospective dose escalation single-center study One eye of each participant will receive the AAV-RS1 gene vector application by intravitreal injection Participants will be closely monitored in conjunction with DSMC oversight Participants will be followed for 18 months after which they will continue to be followed for up to 5 years after enrollment or per FDA requirements for further safety analysis
Outcome Measures The primary outcome is the safety of ocular AAV-RS1 vector as determined from assessment of retinal function ocular structure and occurrence of adverse events and laboratory tests Secondary outcomes include changes in visual function electroretinogram ERG responses visual field measurements retinal imaging with optical coherence tomography OCT and the formation of anti-AAV and anti-RS1 antibodies
Statistics No formal sample size calculations are used in this Phase IIIa dose-escalation study
Study Population Male participants affected with XLRS will receive ocular gene transfer A maximum of up to 24 participants may be enrolled
Design This is a Phase IIIa prospective dose escalation single-center study One eye of each participant will receive the AAV-RS1 gene vector application by intravitreal injection Participants will be closely monitored in conjunction with DSMC oversight Participants will be followed for 18 months after which they will continue to be followed for up to 5 years after enrollment or per FDA requirements for further safety analysis
Outcome Measures The primary outcome is the safety of ocular AAV-RS1 vector as determined from assessment of retinal function ocular structure and occurrence of adverse events and laboratory tests Secondary outcomes include changes in visual function electroretinogram ERG responses visual field measurements retinal imaging with optical coherence tomography OCT and the formation of anti-AAV and anti-RS1 antibodies
Statistics No formal sample size calculations are used in this Phase IIIa dose-escalation study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 15-EI-0038 | None | None | None |