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)

NCT05082116 | Completed Phase 3 Results FDA Drug

• 3 other identifiers: NN7088-4595U1111-1235-59052020-003001-58
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 11

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.

Conditions

Haemophilia A

Outcome Measures

Primary Outcomes (1)

• Number of Bleeding Episodes Per Year (Annualised Bleeding Rate)

  Number of bleeding episodes per year (Annualised Bleeding Rate) data is reported. Annualised bleeding rate (ABR) is the number of bleeding episodes per year.

  From start of treatment (Week 0) until Week 28

Secondary Outcomes (21)

• 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 Week 28

• Number of Injections Needed to Treat Bleeding Episodes

  From start of treatment (Week 0) until Week 28

• Consumption of N8-GP for Prophylaxis

  From start of treatment (Week 0) until Week 28

• FVIII Trough Activity During Prophylaxis

  From start of treatment (Week 0) (excluding the first exposure) until Week 28

• Percentage of Participants With Incidence Rate of Confirmed FVIII Inhibitors ≥0.6 BU

  From start of treatment (Week 0) until Week 28

Study Arms (1)

N8-GP prophylaxis

EXPERIMENTAL

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)

Interventions

turoctocog alfa pegol (N8-GP) DRUG

N8-GP will be injected into a vein (intravenous injections) every 4 days in at least 28 weeks

N8-GP prophylaxis

Eligibility Criteria

• Age: 12 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (11)

Beijing Children's Hospital, Capital Medical University

Beijing, Beijing Municipality, 100045, China

Location

Fujian Medical University Union Hospital-Hematology

Fuzhou, Fujian, 350001, China

Location

Nanfang Hospital, Southern Medical University-Haematology

Guangzhou, Guangdong, 510515, China

Location

The Affiliated Hospital of Guizhou Medical University-Hematology

Guiyang, Guizhou, 550004, China

Location

Xiangya Hospital Central-South University

Changsha, Hunan, 410008, China

Location

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

Location

Qinghai Provincial People's Hospital

Xining, Qinghai, 810007, China

Location

Jinan Central Hospital

Jinan, Shandong, 250013, China

Location

Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology

Tianjin, Tianjin Municipality, 300020, China

Location

Institute of hematology and Blood Diseases Hospital, Tianjin

Tianjin, Tianjin Municipality, 300020, China

Location

The Second Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, 650101, China

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Clinical Reporting Office (2834)
• Organization: Novo Nordisk A/S

clinicaltrials@novonordisk.com

(+1) 866-867-7178

Study Officials

• Clinical Transparency (dept. 1452)

  Novo Nordisk A/S

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 21, 2021
• First Posted: October 18, 2021
• Study Start: September 27, 2021
• Primary Completion: December 28, 2022
• Study Completion: December 28, 2022
• Last Updated: January 6, 2026
• Results First Posted: January 18, 2024

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

CN (11)