Brief Title
Efficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10)
Official Title
A Multi-centre, Open-label Trial Evaluating Efficacy, Safety and Pharmacokinetics of Turoctocog Alfa Pegol (N8-GP) When Used for Treatment and Prophylaxis of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A
Brief Summary
The study investigates how well the medicine called turoctocog alfa pegol (N8-GP) works in
previously treated Chinese patients with severe haemophilia A.
Participants will be treated with N8-GP. This is a medicine that doctors can already
prescribe in other countries.
The medicine will be injected into a vein (intravenous injections) and blood samples will be
collected.
The study will last for about 7-8 months. Participants will have between 8 and 15 visits to
the clinic and possibly a number of phone calls with the study doctor.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Number of bleeding episodes
Secondary Outcome
Haemostatic effect of N8-GP when used for treatment of bleeding episodes, assessed on a four-point scale for haemostatic response (excellent, good, moderate and none)
Condition
Haemophilia A
Intervention
turoctocog alfa pegol (N8-GP)
Study Arms / Comparison Groups
N8-GP prophylaxis
Description: All patients will receive prophylaxis with 50 IU/kg N8-GP every 4 days for a treatment period of at least 28 weeks (with the possibility of switching to twice-weekly dosing during the treatment period at the discretion of the investigator).
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
36
Start Date
September 27, 2021
Completion Date
December 28, 2022
Primary Completion Date
December 28, 2022
Eligibility Criteria
Inclusion Criteria:
- Informed consent obtained before any trial-related activities. Trial-related
activities are any procedures that are carried out as part of the trial, including
activities to determine suitability for the trial.
- Male Chinese patient with severe congenital haemophilia A with a FVIII activity below
1% according to medical records.
- Aged greater than or equal to 12 years at the time of signing informed consent.
- History of at least 150 exposure days (EDs) to other FVIII products.
- The patient and/or caregiver is capable of assessing a bleeding episode, keeping a
diary, performing home treatment of bleeding episodes and otherwise following the
trial procedures at the discretion of the investigator.
Exclusion Criteria:
- Known or suspected hypersensitivity to trial product or related products.
- Previous participation in this trial. Participation is defined as signed informed
consent.
- Participation in any clinical trial of an approved or non-approved investigational
medicinal product within 5 half-lives or 30 days from screening, whichever is longer.
- Known history of FVIII inhibitors based on existing medical records, laboratory report
reviews and patient and/or caregiver interviews.
- Current FVIII inhibitors greater than or equal to 0.6 BU.
- Congenital or acquired coagulation disorder other than haemophilia According to
medical records.
- HIV positive, defined by medical records, with CD4+ count less than or equal 200/L and
a viral load greater than 200 particles/μl or greater than 400000 copies/mL within 6
months of the trial entry. If the data are not available in medical records within
last 6 months, then the test must be performed at screening visit.
- Previous significant thromboembolic events (e.g. myocardial infarction,
cerebrovascular disease or deep venous thrombosis) as defined by available medical
records.
- Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine
aminotransferase (ALT) greater than 3 times limit of normal combined with total
bilirubin greater than 1.5 times the upper limit of normal at screening, as defined by
central laboratory
- Renal impairment defined as estimated glomerular filtration rate (eGFR) below or equal
to 30 mL/min/1.73 m^2 for serum creatinine measured at screening, as defined by
central laboratory.
- Platelet count below 50×109/L at screening based on central laboratory values at
screening.
- Ongoing immune modulating or chemotherapeutic medication.
- Any disorder, except for conditions associated with haemophilia A, which in the
investigator's opinion might jeopardise the patient's safety or compliance with the
protocol.
- Mental incapacity, unwillingness or language barriers precluding adequate
understanding or cooperation.
Gender
Male
Ages
12 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Clinical Transparency (dept. 1452), ,
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT05082116
Organization ID
NN7088-4595
Secondary IDs
U1111-1235-5905
Responsible Party
Sponsor
Study Sponsor
Novo Nordisk A/S
Study Sponsor
Clinical Transparency (dept. 1452), Study Director, Novo Nordisk A/S
Verification Date
March 2023