Ignite Creation Date:
2024-05-06 @ 7:53 PM
Last Modification Date:
2024-10-26 @ 3:16 PM
Study NCT ID:
NCT06178120
Status:
RECRUITING
Last Update Posted:
2024-01-31
First Post:
2023-11-28
Brief Title:
Disease Progression in Women With X-linked Adrenoleukodystrophy
Sponsor:
Minoryx Therapeutics SL
Organization:
Minoryx Therapeutics SL
Study Overview
Official Title:
An Observational Study to Assess Disease Progression in Women With X-linked Adrenoleukodystrophy
Status:
RECRUITING
Status Verified Date:
2023-12
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:
None
Brief Summary:
Observational single-site prospective and minimally interventional study in women with X-linked adrenoleukodystrophy ALD conducted in France
Detailed Description:
Patients accepting participation after signing ICF will be followed up to at least 2 years or until they started treatment for X-linked adrenoleukodystrophy ALD or withdraw consent whichever occurs first Follow-up will be extended beyond 2 years if deemed appropriate after an interim report Tests and questionnaires will be assessed at baseline and yearly If the study is extended beyond 2 years patients will be assessed at 1-year intervals
At baseline visit and follow-up visits patients will undergo an MRI of the brain and the spinal cord and assessments of body sway EDSS ADL pain VAS and SF-36 questionnaire Plasma biomarkers will be assessed from samples obtained through routine blood draw and a monthly falls diary will be provided each visit to be completed once a month
This study will not assess any specific medicinal product or intervention and the study will not interfere with that prescribed in clinical practice
At baseline visit and follow-up visits patients will undergo an MRI of the brain and the spinal cord and assessments of body sway EDSS ADL pain VAS and SF-36 questionnaire Plasma biomarkers will be assessed from samples obtained through routine blood draw and a monthly falls diary will be provided each visit to be completed once a month
This study will not assess any specific medicinal product or intervention and the study will not interfere with that prescribed in clinical practice
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