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

Nephropathic Cystinosis Clinical Trial

— PROREAL  

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04246060
• Other study ID #: CHIESI-NIS-003
• Status: Enrolling by invitation
• Start date: July 31, 2020
• Est. completion date: July 1, 2026

Study information

• Verified date: November 2021
• Source: Chiesi SA/NV
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 31
• Est. completion date: July 1, 2026
• Est. primary completion date: July 1, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion criteria:

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

Related Conditions & MeSH terms

• Cystinosis
• Fanconi Syndrome
• Nephropathic Cystinosis

Intervention

Drug:
• Cysteamine Bitartrate
ERT

Locations

Country	Name	City	State
Belgium	UZA	Antwerp
Belgium	UCL Saint-Luc	Brussel
Belgium	HUDERF	Brussels
Belgium	UZ Gent	Gent
Belgium	UZ Leuven	Leuven
Belgium	CHC	Liège
Belgium	CHU	Liège

Sponsors (1)

Lead Sponsor	Collaborator
Chiesi SA/NV

Country where clinical trial is conducted

Belgium, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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