Ignite Creation Date:
2024-05-06 @ 4:04 PM
Last Modification Date:
2024-10-26 @ 2:02 PM
Study NCT ID:
NCT04855045
Status:
UNKNOWN
Last Update Posted:
2022-03-25
First Post:
2021-04-13
Brief Title:
An Open-label Dose Escalation and Double-masked Randomized Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children 8 Years of Age With LCA10 Caused by Mutations in the CEP290 Gene
Sponsor:
ProQR Therapeutics
Organization:
ProQR Therapeutics
Study Overview
Official Title:
An Open-Label Dose Escalation and Double-Masked Randomized Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects 8 Years of Age With Leber Congenital Amaurosis Type 10 LCA10 Due to the c2991 1655AG pCys998X Mutation
Status:
UNKNOWN
Status Verified Date:
2022-03
Last Known Status:
RECRUITING
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:
BRIGHTEN
Brief Summary:
PQ-110-005 BRIGHTEN is an open-label dose escalation and double-masked randomized controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal IVT injection in pediatric subjects 8 years of age with LCA10 due to the c29911655AG mutation over 24 months of treatment
Detailed Description:
This is an open-label dose escalation and double-masked randomized controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal IVT injection in pediatric subjects 8 years of age with LCA10 due to the c29911655AG mutation The study consists of two parts an open-label dose escalation part followed by a double-masked randomized part
In the open label part subjects will be assigned to one of 3 planned dose groups using a staggered dose escalation design After at least 1 patient is dosed in each group the Data Monitoring Committee DMC will review at least 4 weeks of safety data post dosing and may recommend initiation of the next dose group The DMC may recommend initiation of the double-masked randomized part of the study after completion of the last dose group in the dose escalation part of the study
In the double-masked randomized controlled part of the study subjects will be randomized to one of 2 planned dose groups
Subjects will receive a unilateral IVT injection of sepofarsen on Day 1 Thereafter a 6-monthly dosing schedule is planned
After each dosing subjects will be assessed for safety and tolerability at follow up visits
In the open label part subjects will be assigned to one of 3 planned dose groups using a staggered dose escalation design After at least 1 patient is dosed in each group the Data Monitoring Committee DMC will review at least 4 weeks of safety data post dosing and may recommend initiation of the next dose group The DMC may recommend initiation of the double-masked randomized part of the study after completion of the last dose group in the dose escalation part of the study
In the double-masked randomized controlled part of the study subjects will be randomized to one of 2 planned dose groups
Subjects will receive a unilateral IVT injection of sepofarsen on Day 1 Thereafter a 6-monthly dosing schedule is planned
After each dosing subjects will be assessed for safety and tolerability at follow up visits
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None