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Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients
Sponsor: Chiesi SA/NV
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.
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
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
31
Start Date
2020-07-31
Completion Date
2026-07-01
Last Updated
2021-11-29
Healthy Volunteers
No
Conditions
Interventions
Cysteamine Bitartrate
ERT
Locations (7)
UZA
Antwerp, Belgium
HUDERF
Brussels, Belgium
UCL Saint-Luc
Brussels, Belgium
UZ Gent
Ghent, Belgium
UZ Leuven
Leuven, Belgium
CHC
Liège, Belgium
CHU
Liège, Belgium