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Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients (Protect-NOW)

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ClinicalTrials.gov Identifier: NCT03695978
Recruitment Status : Recruiting
First Posted : October 4, 2018
Last Update Posted : March 15, 2019
Sponsor:
Information provided by (Responsible Party):
Octapharma

Tracking Information
First Submitted Date October 3, 2018
First Posted Date October 4, 2018
Last Update Posted Date March 15, 2019
Actual Study Start Date February 13, 2018
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 3, 2018)
  • Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment [ Time Frame: 100 exposure days ]
    Annualised rate of all bleeding events (BEs), including all spontaneous, traumatic and joint BEs
  • Incidence of Adverse Drug Reactions (ADRs) [ Time Frame: 100 exposure days ]
    Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded in by patients in treatment diaries, which will be reviewed at each Follow-up Visit.
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT03695978 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures
 (submitted: October 3, 2018)
  • Dosage of FVIII concentrates [ Time Frame: 100 exposure days ]
    For each individual FVIII injection the dose will be recorded.
  • Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians [ Time Frame: 100 exposure days ]
    At the end of the postoperative period, treating physicians will assess the effectiveness of surgical prophylaxis using a scale including the four items: 'excellent,' 'good,' moderate,' and 'none'.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients
Official Title Practical Utilisation of Octapharma FVIII Concentrates in Previously Untreated & Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment With Nuwiq, Octanate or Wilate - Efficacy & Safety Observational Study-Protect-NOW
Brief Summary International, post-authorisation non-interventional study to evaluate real-life effectiveness, safety and utilisation patterns of Octapharma's FVIII concentrates Nuwiq, Octanate, and Wilate in previously untreated and minimally treated severe haemophilia A patients in routine clinical practice.
Detailed Description Octapharma's FVIII concentrates have been tested in clinical trials and registered for treatment of haemophilia A; however, as haemophilia A is a rare disease, the numbers of patients treated in studies so far are limited. For previously untreated patients (PUPs), who are typically young children, and for minimally treated patients (MTPs), who have been exposed to only minimal FVIII dosages, there is a general interest to increase the body of data on treatment effectiveness and safety, particularly related to inhibitor development. Also, specifically for PUPs, treatment algorithms are not standardized, e.g. with respect to utilisation, dosage, frequency or optimal start age of FVIII prophylaxis. Real world evidence derived from a non-interventional study (NIS) can describe product utilisation and demonstrate value over a product's life cycle and facilitate benefit-risk assessments. The purpose of this study is thus to evaluate product utilisation, effectiveness and safety, including inhibitor development information, in severe haemophilia A PUPs and MTPs, who have been prescribed Octapharma's FVIII concentrates.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population A total of 140 patients, either previously untreated patients (PUPs) or minimally treated patients (MTPs), are planned to be documented in the study
Condition Haemophilia A
Intervention Not Provided
Study Groups/Cohorts
  • Nuwiq
    All patients receiving Nuwiq (recombinant FVIII)
  • Octanate
    All patients receiving Octanate (plasma derived FVIII)
  • Wilate
    All patients receiving Wilate (plasma derived FVIII/von Willebrand factor [VWF])
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: October 3, 2018)
140
Original Estimated Enrollment Same as current
Estimated Study Completion Date June 2022
Estimated Primary Completion Date June 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Male and female patients of any age and ethnicity
  • Severe haemophilia A (FVIII:C<1%)
  • Decision to prescribe Octapharma's FVIII concentrate before enrollment into the study
  • Either
  • No previous treatment with FVIII concentrates or other blood products containing FVIII (PUPs) OR
  • Less than 5 Exposure Days (EDs) to FVIII concentrates or other blood products containing FVIII (MTPs), if
  • their first ED occurred after 1st January 2015, AND
  • they did not develop an inhibitor at any time point, OR
  • they developed an inhibitor during treatment with an Octapharma FVIII concentrate AND continue treatment with THIS Octapharma FVIII concentrate
  • Voluntarily given, fully informed written and signed consent obtained before any study-related data documentation is conducted (obtained from the patient's parent/legal guardian)

Exclusion Criteria:

  • Diagnosis with a coagulation disorder other than haemophilia A
  • Concomitant treatment with any systemic immunosuppressive drug
  • Participation in an interventional clinical trial during the time period evaluated
  • Participation in another non-interventional study of Octapharma
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Sigurd Knaub, PhD +41 554512141 Sigurd.Knaub@octapharma.ch
Listed Location Countries Azerbaijan,   Belarus,   Belgium,   Canada,   Estonia,   France,   Germany,   Italy,   Lithuania,   Russian Federation,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03695978
Other Study ID Numbers GENA-25
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Octapharma
Study Sponsor Octapharma
Collaborators Not Provided
Investigators
Study Director: Sigurd Knaub, PhD Octapharma
PRS Account Octapharma
Verification Date March 2019