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

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

Status

Enrolling by invitation

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT04246060

Acronym

PROREAL

Enrollment

Registered

2020-01-29

Start date

2020-07-31

Completion date

2026-07-01

Last updated

2021-11-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Nephropathic Cystinosis

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.

Interventions

DRUGCysteamine Bitartrate

ERT

Study design

Observational model

COHORT

Time perspective

OTHER

Eligibility

Sex/Gender

ALL

Healthy volunteers

Inclusion criteria

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

Design outcomes

Primary

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	12 months

Countries

Belgium

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026