Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients
NCT04246060 · Status: ENROLLING_BY_INVITATION · Type: OBSERVATIONAL · Enrollment: 31
Last updated 2021-11-29
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.
Conditions
- Nephropathic Cystinosis
Interventions
- DRUG
-
Cysteamine Bitartrate
ERT
Sponsors & Collaborators
-
Chiesi SA/NV
lead OTHER
Eligibility
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2020-07-31
- Primary Completion
- 2022-07-01
- Completion
- 2026-07-01
- FDA Drug
- Yes
Countries
- Belgium
Study Locations
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