Ignite Creation Date:
2025-12-24 @ 1:42 PM
Ignite Modification Date:
2025-12-27 @ 10:11 PM
Study NCT ID:
NCT04143295
Status:
AVAILABLE
Last Update Posted:
2025-12-11
First Post:
2018-04-24
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Rescue of Infants With MCT8 Deficiency
Sponsor:
Roy E. Weiss, M.D.
Organization:
Study Overview
Official Title:
Rescue of Infants With MCT8 Deficiency
Status:
AVAILABLE
Status Verified Date:
2025-12
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:
DITPA
Brief Summary:
Monocarboxylate Transporter 8 (MCT8) deficiency (that is also known as Allan-Herndon-Dudley syndrome) is a rare X-linked inherited disorder of brain development that causes severe intellectual disability and problems with movement. This condition, which occurs almost exclusively in males, disrupts development from before birth.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: