Observational Study to Assess the Quality of Life in Nephropathic Cystinosis Patients

• Conditions: Nephropathic Cystinosis
• Interventions: Drug: Cysteamine Bitartrate

• Registration Number: NCT04246060
• Lead Sponsor: Chiesi SA/NV

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

Not available

Study Timeline

• Study First Submitted: 2020-01-27
• Study First Submitted QC: 2020-01-27
• Study First Posted: 2020-01-29
• Study Start: 2020-07-31
• Status Verified: 2021-11
• Last Update Submitted: 2021-11-25
• Last Update Posted: 2021-11-29
• Primary Completion: 2022-07-01
• Study Completion: 2026-07-01

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 31

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Cohort 3	Cysteamine Bitartrate	Patients remaining on immediate release cysteamine treatment
Cohort 1	Cysteamine Bitartrate	Patients on extended release cysteamine treatment at study enrollment
Cohort 2	Cysteamine Bitartrate	Patients switching from immediate release cysteamine to extended release cysteamine during the study

Primary Outcome Measures

Name	Time	Method
to descriptively assess the change from baseline and percent change from baseline in the quality of life overall summary sores at 12 months in switch cohort	12 months

Trial Locations

Locations (7)

UZA; 🇧🇪 Antwerp, Belgium

HUDERF; 🇧🇪 Brussels, Belgium

UCL Saint-Luc; 🇧🇪 Brussel, Belgium

UZ Gent; 🇧🇪 Gent, Belgium

UZ Leuven; 🇧🇪 Leuven, Belgium

CHC; 🇧🇪 Liège, Belgium

CHU; 🇧🇪 Liège, Belgium