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

N/AEnrolling By InvitationNCT04246060

Chiesi SA/NV31 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Nephropathic Cystinosis

Interventions

Cysteamine BitartrateDRUG

ERT

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion criteria: * Confirmed diagnosis of nephropathic cystinosis * Undergoing cystine depletion therapy with oral cysteamine * Signature of informed concent.

Locations (7)

UZA

Antwerp, Belgium

HUDERF

Brussels, Belgium

UCL Saint-Luc

Brussels, Belgium

UZ Gent

Ghent, Belgium

UZ Leuven

Leuven, Belgium

Outcomes

Primary Outcomes

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

Time frame: 12 months

Data from ClinicalTrials.gov

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