Trials / Enrolling By Invitation
Enrolling By InvitationNCT04246060
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
- Phase
- —
- Study type
- Observational
- Enrollment
- 31 (estimated)
- Sponsor
- Chiesi SA/NV · Academic / Other
- Sex
- All
- Age
- —
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Cysteamine Bitartrate | ERT |
Timeline
- Start date
- 2020-07-31
- Primary completion
- 2022-07-01
- Completion
- 2026-07-01
- First posted
- 2020-01-29
- Last updated
- 2021-11-29
Locations
7 sites across 1 country: Belgium
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04246060. Inclusion in this directory is not an endorsement.