A non-interventional, post authorization safety study (PASS) to evaluate the safety of Kineret in the treatment of Cryopyrin Associated Periodic Syndromes (CAPS) in routine clinical care with regard to serious infections, malignancies, injection site reactions, allergic reactions and medication errors, including re-use of syringe. Sobi.Anakin-201 (PASS Kineret CAPS)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Effectiveness study (incl. comparative)

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  100000096873

  anakinra
• Medical condition to be studied:
  Cryopyrin associated periodic syndrome

Population studied

• Age groups:
  Infants and toddlers (28 days – 23 months)

  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:
  20

Study design details

• Main study objective:
  The primary objective of the study is to evaluate the safety of Kineret treatment in CAPS patients in routine clinical care with focus on serious infections, malignancies, injection site reactions, allergic reactions and medication errors, including re-use of the syringe. The study is designed to address the effectiveness of the risk minimization measures for medication.
• Outcomes:
  The primary endpoints of the study are: • Rate of serious infections • Rate of new malignancies • Rate of ISRs • Rate of allergic reactions • Rate of medication errors including re-use of syringe. Medication errors will be further classified as infections of the injection site, re-use of syringe, over- or underdosing, or other medication errors. The secondary endpoints of the study are: • Kineret dose • Proportion of patients who discontinue Kineret treatment permanently (including reason for dicontinuation) • Proportion of patients who discontinue Kineret treatment temporarily (including reason for dicontinuation) • Proportion of patients who are transferred to another IL 1 blocking treatment
• Data analysis plan:
  The presence of serious infections, new malignancies, ISRs, allergic reactions and medication errors will be presented as rates, calculated as the number of events divided by the total cumulative exposure to Kineret treatment in the study (patient years). 95% confidence intervals will be calculated for the rate of each of the five event types. In addition to the rate, the distribution of the severity, relationship to the Kineret treatment and seriousness will be presented. All enrolled patients will be included in the analysis. The analyses will be conducted primarily for the total study population. In addition, the subgroup of patients who are already using Kineret at baseline and the subgroup who initiate Kineret treatment at baseline will be analyzed separately.

Documents

Study results

Sobi.ANAKIN-201_CSR Synopsis