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

NCT ID: NCT04246060

Last Updated: 2021-11-29

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

31 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-07-31

Study Completion Date

2026-07-01

Brief Summary

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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

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Conditions

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Nephropathic Cystinosis

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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Cohort 1

Patients on extended release cysteamine treatment at study enrollment

Cysteamine Bitartrate

Intervention Type DRUG

ERT

Cohort 2

Patients switching from immediate release cysteamine to extended release cysteamine during the study

Cysteamine Bitartrate

Intervention Type DRUG

ERT

Cohort 3

Patients remaining on immediate release cysteamine treatment

Cysteamine Bitartrate

Intervention Type DRUG

ERT

Interventions

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Cysteamine Bitartrate

ERT

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Confirmed diagnosis of nephropathic cystinosis
* Undergoing cystine depletion therapy with oral cysteamine
* Signature of informed concent.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Chiesi SA/NV

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UZA

Antwerp, , Belgium

Site Status

HUDERF

Brussels, , Belgium

Site Status

UCL Saint-Luc

Brussels, , Belgium

Site Status

UZ Gent

Ghent, , Belgium

Site Status

UZ Leuven

Leuven, , Belgium

Site Status

CHC

Liège, , Belgium

Site Status

CHU

Liège, , Belgium

Site Status

Countries

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Belgium

Other Identifiers

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CHIESI-NIS-003

Identifier Type: -

Identifier Source: org_study_id