Ignite Creation Date:
2024-05-06 @ 8:20 AM
Last Modification Date:
2024-10-26 @ 11:58 AM
Study NCT ID:
NCT02714764
Status:
RECRUITING
Last Update Posted:
2023-07-28
First Post:
2016-02-19
Brief Title:
Evaluation of Outcome Metrics in Alexander Disease
Sponsor:
Childrens Hospital of Philadelphia
Organization:
Childrens Hospital of Philadelphia
Study Overview
Official Title:
Evaluation of Outcome Metrics in Alexander Disease
Status:
RECRUITING
Status Verified Date:
2023-07
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:
AxD Outcomes
Brief Summary:
The purpose of this study is to define the natural history of Alexander Disease a leukodystrophy that causes neurological dysfunction Investigators will obtain clinical outcome assessments to measure how the disease affects a patients gross motor fine motor speech and language function swallowing and quality of life Specimens are collected to measure glial fibrillary acidic protein GFAP levels in cerebrospinal fluid CSF and blood The data obtained from this study will be used for the design of future treatment trials
Detailed Description:
Participants will be asked to complete physical examinations including physical therapy occupational therapy speech and language therapy neurocognitive and swallowing assessments Patients or caretakers may be asked to complete questionnaires as well Specimen collection is an optional procedure The study asks for participants to return at least once yearly to repeat assessments
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?:
None
Is an FDA AA801 Violation?:
None