Ignite Creation Date:
2024-05-06 @ 1:03 PM
Last Modification Date:
2024-10-26 @ 1:07 PM
Study NCT ID:
NCT03913130
Status:
TERMINATED
Last Update Posted:
2023-01-04
First Post:
2019-02-21
Brief Title:
Extension Study to Study PQ-110-001 NCT03140969
Sponsor:
ProQR Therapeutics
Organization:
ProQR Therapeutics
Study Overview
Official Title:
An Open-Label Extension Study to Evaluate the Safety Tolerability Efficacy and Pharmacokinetics of QR-110 in Subjects With Lebers Congenital Amaurosis LCA Due to the c29911655AG Mutation pCys998X in the CEP290 Gene
Status:
TERMINATED
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Terminated Study prematurely terminated due to sponsor decision for reasons unrelated to safety
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
INSIGHT
Brief Summary:
Subjects completing participation in study PQ-110-001 EudraCT 2017-000813-22 NCT03140969 will be given the opportunity to enroll into the extension study for continued dosing if available data support current andor future benefits for the subject Study PQ-110-002 will provide long-term safety tolerability pharmacokinetic PK and efficacy data of QR-110
Detailed Description:
Subjects completing participation in study PQ-110-001 EudraCT 2017-000813-22 NCT03140969 will be given the opportunity to enroll into the extension study for continued dosing if available data support current andor future benefits for the subject
Subjects will be given the opportunity to enroll into this extension study for continued dosing if available data support current andor future benefits for the subject The Investigator in consultation and agreement with the Medical Monitor will decide on enrollment of each individual subject as well as on dosing of the first treated eye and treatment initiation of the contralateral eye Continued subject treatment in this study is desirable but cannot be guaranteed since it will depend on the risks and benefit of further treatment on a case-by-case basis as discussed and agreed upon with the Medical Monitor
The contralateral eye and the first treated eye will be injected 3 months apart The injection interval of 3 months between both eyes will limit burden for the subjects with a 3 month-visit frequency during the course of the study This between-eye interval could be adapted if safety data are supportive and for logistic reasons and in agreement with the Medical Monitor
The same safety monitoring protocol and efficacy assessments will apply to both eyes
QR-110 will be administered via intravitreal IVT injection
Subjects will be given the opportunity to enroll into this extension study for continued dosing if available data support current andor future benefits for the subject The Investigator in consultation and agreement with the Medical Monitor will decide on enrollment of each individual subject as well as on dosing of the first treated eye and treatment initiation of the contralateral eye Continued subject treatment in this study is desirable but cannot be guaranteed since it will depend on the risks and benefit of further treatment on a case-by-case basis as discussed and agreed upon with the Medical Monitor
The contralateral eye and the first treated eye will be injected 3 months apart The injection interval of 3 months between both eyes will limit burden for the subjects with a 3 month-visit frequency during the course of the study This between-eye interval could be adapted if safety data are supportive and for logistic reasons and in agreement with the Medical Monitor
The same safety monitoring protocol and efficacy assessments will apply to both eyes
QR-110 will be administered via intravitreal IVT injection
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 |
|---|---|---|---|
| 2018-003500-40 | EUDRACT_NUMBER | None | None |