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

Chiesi SA/NV

Status: Invitation-only

Conditions: Nephropathic Cystinosis

Treatments: Drug: Cysteamine Bitartrate

Study type: Observational

Funder types: Other

Identifiers

NCT04246060

CHIESI-NIS-003

Details and patient eligibility

About

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.

Enrollment: 31 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion criteria:

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

Trial design

31 participants in 3 patient groups

Cohort 1

Description:

Patients on extended release cysteamine treatment at study enrollment

Treatment:

Drug: Cysteamine Bitartrate

Cohort 2

Description:

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

Treatment:

Drug: Cysteamine Bitartrate

Cohort 3

Description:

Patients remaining on immediate release cysteamine treatment

Treatment:

Drug: Cysteamine Bitartrate