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 type

• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)

Study drug and medical condition

• Name of medicine:
  PROCYSBI

  CYSTAGON
• Anatomical Therapeutic Chemical (ATC) code:
  (A16AA04) mercaptamine

  mercaptamine
• Medical condition to be studied:
  Cystinosis

Population studied

• Age groups:
  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Estimated number of subjects:
  10

Study design details

• Main study objective:
  To evaluate the long-term efficacy and safety of IR-cysteamine (Cystagon®) and ER-cysteamine (Procysbi®) therapy under routine clinical practice.
• Outcomes:
  Primary efficacy endpoints• WBCs cystine levels• eGFR:, Secondary efficacy endpoints• Growth• Total prescribed daily cysteamine dose
• Data analysis plan:
  Data analysis includes four periods: Screening, IR-treatment period and ER-treatment periods 1 and 2. Efficacy, safety, prescribed dose and other patient characteristics are collected.