Ignite Creation Date:
2024-10-26 @ 3:41 PM
Last Modification Date:
2024-10-26 @ 3:41 PM
Study NCT ID:
NCT06609889
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-08-10
Brief Title:
A Safety and Efficacy of Intrathecally Administered ION283 in Patients with Lafora Disease
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Phase 12 Open Label Study to Evaluate the Safety and Efficacy of Intrathecally Administered ION283 in Patients with Lafora Disease
Status:
RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
Lafora
Brief Summary:
This study will test the safety and efficacy of multiple doses of ION283 administered as intrathecal IT injections by lumbar puncture LP All subjects will receive ION283 The dose level of 15 mg will be studied in all subjects
Detailed Description:
A Phase 12 Open Label Study to Evaluate the Safety and Efficacy of Intrathecally Administered ION283 in Patients with Lafora Disease
A single cohort will be evaluated in the study
N10
Initial dose of 15 mg ION283 intrathecal bolus ITB injection every 12 weeks
The study consists of 2 periods
Screening Period 4 weeks
Open label Treatment Period 24 months
A single cohort will be evaluated in the study
N10
Initial dose of 15 mg ION283 intrathecal bolus ITB injection every 12 weeks
The study consists of 2 periods
Screening Period 4 weeks
Open label Treatment Period 24 months
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None