Ignite Creation Date:
2025-12-25 @ 2:09 AM
Ignite Modification Date:
2025-12-26 @ 5:53 AM
Study NCT ID:
NCT04246060
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2021-11-29
First Post:
2020-01-27
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients
Sponsor:
Chiesi SA/NV
Organization:
Study Overview
Official Title:
Multicentre, Ambispective, Observational, Real Life Study to Assess the Quality of Life Effectiveness of Extended Release (ER) Oral Cysteamine Therapy (Procysbi) in Belgian Patients Suffering From Nephropathic Cystinosis
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2021-11
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:
PROREAL
Brief Summary:
Most of the real world evidence data related to efficacy of cysteamine therapy is retrospective. This study is a ambispective study to investigate the impact of cystine depletion therapy on the quality of life of patients and their parents.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
False
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?:
False
Is an FDA AA801 Violation?: