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

• ClinicalTrials.gov Identifier: NCT03695978
• Recruitment Status: Recruiting
• First Posted: October 4, 2018
• Last Update Posted: April 23, 2024
• Sponsor: Octapharma
• Information provided by (Responsible Party): Octapharma

Tracking Information

• First Submitted Date: October 3, 2018
• First Posted Date: October 4, 2018
• Last Update Posted Date: April 23, 2024
• Actual Study Start Date: February 13, 2018
• Estimated Primary Completion Date: June 2030 (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

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 200 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

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: November 1, 2023): 200
• Original Estimated Enrollment (submitted: October 3, 2018): 140
• Estimated Study Completion Date: June 2030
• Estimated Primary Completion Date: June 2030 (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
• data are available on all previous treatment, 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 (in the presence or absence of emicizumab).
• 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, Hungary, Italy, Lithuania, Mexico, Russian Federation, Spain, Turkey, United Kingdom, United States

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
• Current Responsible Party: Octapharma
• Original Responsible Party: Same as current
• Current Study Sponsor: Octapharma
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Sigurd Knaub, PhD; Octapharma

• PRS Account: Octapharma
• Verification Date: April 2024