Pathfinder10: 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)
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: N8-GP prophylaxis 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). |
Drug: turoctocog alfa pegol (N8-GP)
N8-GP will be injected into a vein (intravenous injections) every 4 days in at least 28 weeks
|
Outcome Measures
Primary Outcome Measures
- Number of bleeding episodes [From start of treatment (week 0) until visit 7 (week 28)]
Count
Secondary Outcome Measures
- 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) [From start of treatment (week 0) until visit 7 (week 28)]
Count
- Consumption of N8-GP for treatment of bleeding episodes [From start of treatment (week 0) until visit 7 (week 28)]
IU/kg/bleed
- Consumption of N8-GP for prophylaxis [From start of treatment (week 0) until visit 7 (week 28)]
IU/kg/year
- FVIII trough activity during prophylaxis [From start of treatment (week 0) (excluding the first exposure) until visit 7 (week 28)]
IU/mL
- Incidence rate of confirmed FVIII inhibitors greater than or equal to 0.6 BU (Bethesda Units) [From start of treatment (week 0) until visit 7 (week 28)]
Rate
- Number of adverse events (AEs) [From start of treatment (week 0) to end of trial (week 28 + 30 days)]
Count
- Number of serious adverse events (SAEs) [From start of treatment (week 0) to end of trial (week 28 + 30 days)]
Count
- FVIII activity 30 minutes post-injection (C30min) [Single-dose: 30 minutes plus/minus 5 minutes post-injection at visit 2a (week 0)]
IU/mL
- FVIII activity 30 minutes post-injection (C30min) [Steady state: 30 minutes plus/minus 5 min post-injection at visit 7 (week 28)]
IU/mL
- Incremental recovery (IR) [Single-dose: 30 minutes plus/minus 5 minutes post-injection at visit 2a (week 0)]
(IU/mL)/(IU/kg)
- Incremental recovery (IR) [Steady state: 30 minutes plus/minus 5 minutes post-injection at visit 7 (week 28)]
(IU/mL)/(IU/kg)
- Area under the curve (AUC) [Single-dose: 0-inf post-injection at visit 2a (week 0)]
hours*(IU/mL)
- Area under the curve (AUC) [Steady state: 0-inf post-injection at visit 7 (week 28)]
hours*(IU/mL)
- Terminal half-life (t½) [Single-dose: 0-96 hours post-injection at visit 2a (week 0)]
Hours
- Terminal half-life (t½) [Steady state: 0-96 hours post-injection at visit 7 (week 28)]
Hours
- Clearance (CL) [Single-dose: 0-96 hours post-injection at visit 2a (week 0)]
mL/h/kg
- Clearance (CL) [Steady state: 0-96 hours post-injection at visit 7 (week 28)]
mL/h/kg
- FVIII trough activity 96 hours post-injection (C96h) [Single-dose: 96 hours plus/minus 8 hours post-injection at visit 2a (week 0)]
IU/mL
- FVIII trough activity 96 hours post-injection (C96h) [Steady state: 96 hours plus/minus 8 hours post-injection at visit 7 (week 28)]
IU/mL
Eligibility Criteria
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.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Novo Nordisk Investigational Site | Beijing | Beijing | China | 100045 |
| 2 | Novo Nordisk Investigational Site | Fuzhou | Fujian | China | 350001 |
| 3 | Novo Nordisk Investigational Site | Guangzhou | Guangdong | China | 510515 |
| 4 | Novo Nordisk Investigational Site | Guiyang | Guizhou | China | 550004 |
| 5 | Novo Nordisk Investigational Site | Changsha | Hunan | China | 410008 |
| 6 | Novo Nordisk Investigational Site | Suzhou | Jiangsu | China | 215006 |
| 7 | Novo Nordisk Investigational Site | Xining | Qinghai | China | 810007 |
| 8 | Novo Nordisk Investigational Site | Jinan | Shandong | China | 250013 |
| 9 | Novo Nordisk Investigational Site | Tianjing | Tianjin | China | 300020 |
| 10 | Novo Nordisk Investigational Site | Kunming | Yunnan | China | 650101 |
Sponsors and Collaborators
- Novo Nordisk A/S
Investigators
- Study Director: Clinical Transparency (dept. 1452), Novo Nordisk A/S
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- NN7088-4595
- U1111-1235-5905
- 2020-003001-58