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

November 25, 2021 updated by: Chiesi SA/NV

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

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 Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Anticipated)

31

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Antwerp, Belgium
        • UZA
      • Brussel, Belgium
        • UCL Saint-Luc
      • Brussels, Belgium
        • Huderf
      • Gent, Belgium
        • UZ Gent
      • Leuven, Belgium
        • UZ Leuven
      • Liège, Belgium
        • CHC
      • Liège, Belgium
        • CHU

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Probability Sample

Study Population

All Belgian nephropathic cystinosis patients.

Description

Inclusion criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Other

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Chiesi SA/NV

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 31, 2020

Primary Completion (Anticipated)

July 1, 2022

Study Completion (Anticipated)

July 1, 2026

Study Registration Dates

First Submitted

January 27, 2020

First Submitted That Met QC Criteria

January 27, 2020

First Posted (Actual)

January 29, 2020

Study Record Updates

Last Update Posted (Actual)

November 29, 2021

Last Update Submitted That Met QC Criteria

November 25, 2021

Last Verified

November 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CHIESI-NIS-003

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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