A MULTICENTRE, NON-INTERVENTIONAL, RETROSPECTIVE STUDY TO EXPLORE THE EFFECTS OF TRANSITIONING FROM IMMEDIATE RELEASE TO EXTENDED RELEASE ORAL CYSTEAMINE THERAPY IN NORWEGIAN PATIENTS WITH NEPHROPATHIC CYSTINOSIS (CYSTRANSFER)

Study identification

• EU PAS number:
  EUPAS35491
• Study ID:
  40977
• Official title and acronym:
  A MULTICENTRE, NON-INTERVENTIONAL, RETROSPECTIVE STUDY TO EXPLORE THE EFFECTS OF TRANSITIONING FROM IMMEDIATE RELEASE TO EXTENDED RELEASE ORAL CYSTEAMINE THERAPY IN NORWEGIAN PATIENTS WITH NEPHROPATHIC CYSTINOSIS (CYSTRANSFER)
• DARWIN EU® study:
  No
• Study countries:
  Norway
• Study description:
  The aim of this study is to evaluate the implementation of the Extended-Release(ER)-cysteamine therapy in patients already treated with Immidiate-Release(IR)-cysteamine in Norway and to assess the outcomes of this option in routine care for patients with nephropathic cystinosis. This retrospective non-interventional multi-centre study will assess the efficacy and safety of oral IR- and ER-cysteamine treatment in the Norwegian patient population. The study will be based on retrospective patient journal data.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Oslo University Hospital

Rikshospitalet

Study timelines

Date when funding contract was signed

Planned:10/02/2020

Actual:02/04/2020

Study start date

Planned:01/06/2020

Actual:09/06/2020

Data analysis start date

Planned:01/07/2021

Date of final study report

Planned:31/12/2021

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Chiesi Pharma AB

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable