Ignite Creation Date:
2024-05-06 @ 7:55 PM
Last Modification Date:
2024-10-26 @ 3:16 PM
Study NCT ID:
NCT06181136
Status:
RECRUITING
Last Update Posted:
2024-04-03
First Post:
2023-12-04
Brief Title:
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA Sanfilippo Syndrome Type A
Sponsor:
Denali Therapeutics Inc
Organization:
Denali Therapeutics Inc
Study Overview
Official Title:
A Phase 12 Multicenter Open-Label Study to Evaluate the Safety Tolerability Pharmacokinetics and Pharmacodynamics of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA Sanfilippo Syndrome Type A
Status:
RECRUITING
Status Verified Date:
2024-08
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:
This is a multicenter open-label Phase 12 study to assess the safety tolerability pharmacokinetics PK pharmacodynamics PD and exploratory clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A MPS IIIA The core study period is 25 weeks approximately 6 months and is followed by a 72-week approximately 18 month open-label extension OLE Participants with MPS IIIA will be enrolled in two planned cohorts and additional participants with MPS IIIA may be enrolled in three optional cohorts
Detailed Description:
None
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