Ignite Creation Date:
2024-05-06 @ 3:48 PM
Last Modification Date:
2024-10-26 @ 1:57 PM
Study NCT ID:
NCT04768348
Status:
TERMINATED
Last Update Posted:
2023-08-25
First Post:
2021-02-18
Brief Title:
Natural History Clinical Study in Adult PKU
Sponsor:
Homology Medicines Inc
Organization:
Homology Medicines Inc
Study Overview
Official Title:
A Prospective Study Investigating the Natural History of Adults With Phenylketonuria PKU Due to Phenylalanine Hydroxylase Deficiency
Status:
TERMINATED
Status Verified Date:
2023-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Homology Medicines has discontinued the development of this program
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The objective of this study is to characterize the natural history of phenylketonuria PKU due to phenylalanine hydroxylase PAH deficiency in adults through prospective collection of clinical cognitive and quality of life assessments
Detailed Description:
Phenylalanine hydroxylase PAH deficiency is a rare disease caused by an inborn error of metabolism If left untreated PAH deficiency results in progressive irreversible neurological impairment during infancy and early childhood
This study is designed to collect information about important PKU-related symptoms and tests to characterize the natural history of PKU due to PAH deficiency in a selected sample of adults No new investigational treatment will be administered to participating patients
This study is designed to collect information about important PKU-related symptoms and tests to characterize the natural history of PKU due to PAH deficiency in a selected sample of adults No new investigational treatment will be administered to participating patients
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