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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03695978
Recruitment Status : Recruiting
First Posted : October 4, 2018
Last Update Posted : July 8, 2022
Sponsor:
Information provided by (Responsible Party):
Octapharma

Study Description
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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.

Condition or disease
Haemophilia A

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 Design
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Study Type : Observational
Estimated Enrollment : 140 participants
Observational Model: Cohort
Time Perspective: Other
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
Actual Study Start Date : February 13, 2018
Estimated Primary Completion Date : June 2030
Estimated Study Completion Date : June 2030

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Groups and Cohorts
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Group/Cohort
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])


Outcome Measures
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Primary Outcome Measures :
  1. 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

  2. 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.


Secondary Outcome Measures :
  1. Dosage of FVIII concentrates [ Time Frame: 100 exposure days ]
    For each individual FVIII injection the dose will be recorded.

  2. 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'.


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03695978


Contacts
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Contact: Sigurd Knaub, PhD +41 554512141 Sigurd.Knaub@octapharma.ch

Locations
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Sponsors and Collaborators
Octapharma
Investigators
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Study Director: Sigurd Knaub, PhD Octapharma
More Information
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Responsible Party: Octapharma
ClinicalTrials.gov Identifier: NCT03695978    
Other Study ID Numbers: GENA-25
First Posted: October 4, 2018    Key Record Dates
Last Update Posted: July 8, 2022
Last Verified: July 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn