NN7008-3553 A Multi-centre Non-interventional Study of Safety and Efficacy of turoctocog alfa (rFVIII) during Long-Term Treatment of Severe and Moderately Severe Haemophilia A (FVIII ≤2%) (guardian™ 5)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Prospective, multinational, non-randomised, non-interventional post-authorisation safety study (PASS)

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  TUROCTOCOG ALFA
• Medical condition to be studied:
  Haemophilia A with anti factor VIII

Population studied

• Short description of the study population:
  Previously FVIII treated (>150 EDs) patients with severe and moderately severe haemophilia A with FVIII ≤2%. Only patients for whom it has already been decided to start treatment or have already started treatment with commercially available turoctocog alfa and who has not previously participated in any guardian™ clinical trials will be eligible for the study.; For an eligible patient, all inclusion criteria must be answered “yes”.; 1. Informed consent obtained before any study-related activities. (Study-related activities are any procedure related to recording of data according to the protocol).; 2. Previously FVIII treated (>150 EDs at the time of first dosing with turoctocog alfa) male patients with the diagnosis of congenital severe and moderately severe haemophilia A (FVIII ≤2%).; 3. The decision to initiate treatment with commercially available turoctocog alfa has been made by the patient/parent and the patient’s treating physician before and independently from the decision to include the patient in this study.; 4. Availability of a detailed and reliable patient documentation (patient records, diary, logbook etc.) covering either the last 50 EDs or the last 2 years per patient to confirm treatment modality (i.e. prophylaxis, on-demand or recent surgery) prior to enrolment.; 5. A negative FVIII inhibitor test obtained not more than four weeks prior to first dosing with turoctocog alfa.; 6. Patients with a history of FVIII inhibitors and who have been immune-tolerized to FVIII through Immune Tolerance Induction treatment must have FVIII plasma recovery level ≥66 % of expected level and a FVIII half- life (T½) of ≥6 h after a 72 h wash-out period (as demonstrated by available medical records).; 7. No clinical suspicion of HIV-1 or, if HIV-1 seropositive, viral load <400.000 copies/mL and immunocompetent with CD4+ lymphocyte count ≥200/µL, as assessed during the last 6 months prior to the Baseline visit.; For an eligible patient, all exclusion criteria must be answered “no”.; 1. Contraindications for use according to the approved product information text (US Package Insert (PI), European Summary of Product Characteristics (SmPC) or corresponding local prescribing information). This includes known or suspected allergy to turoctocog alfa or related products.; 2. Previous participation and/or withdrawal from this study. Participation is defined as having given informed consent in this study.; 3. Treatment with any investigational drug within 30 days prior to enrolment into the study; 4. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.; 5. Previous participation in any clinical trial with turoctocog alfa.; 6. Treatment with other FVIII products after initiation of treatment with turoctocog alfa.</400.000></400.000>
• 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)
• Special population of interest:
  Other
• Special population of interest, other:
  Haemophilia A patients
• Estimated number of subjects:
  63

Study design details

• Main study objective:
  To provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal clinical practise in patients previously treated with a factor VIII agent (FVIII).
• Outcomes:
  Incidence rate of FVIII inhibitors (at least 0.6 Bethesda Units (BU) for central laboratory analyses, or above the specific local laboratory reference range) represented as the percentage of patients developing inhibitors, •Number of adverse reactions reported•Number of serious adverse reactions reported•Haemostatic effect of turoctocog alfa in the treatment of bleeds•Haemostatic effect of turoctocog alfa during surgical procedures•Annualised bleeding rate for patients using turoctocog alfa for preventive treatment•Annualised bleeding rate for patients using turoctocog alfa for on-demand treatment
• Data analysis plan:
  No formal testing of statistical hypotheses will be performed. Evaluation of data will be based upon descriptive statistics, i.e. summary tables, listings, and figures. Categorical data will be summarised by frequency tables while continuous data will be summarised by mean, standard deviation,minimum and maximum value.

Documents

Study results

3553 nsr version 2 eu-pas-reg redacted

Study report

3553-eu-pass-progress-report-20160519