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)

NCT ID: NCT05082116

Last Updated: 2024-12-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 36 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-09-27
• Study Completion Date: 2022-12-28

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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).

Group Type: EXPERIMENTAL

turoctocog alfa pegol (N8-GP)

Intervention Type: DRUG

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

Interventions

turoctocog alfa pegol (N8-GP)

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

Intervention Type: DRUG

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.

• Minimum Eligible Age: 12 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Novo Nordisk A/S

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Transparency (dept. 1452)

Role: STUDY_DIRECTOR

Novo Nordisk A/S

Locations

Beijing Children's Hospital，Capital Medical University

Beijing, Beijing Municipality, China

Fujian Medical University Union Hospital-Hematology

Fuzhou, Fujian, China

Haemotology, Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, China

The Affiliated hospital of Guizhou Medical University-Hemato

Guiyang, Guizhou, China

Qinghai Provincial People's Hospital

Xining, Qinghai, China

Jinan Central Hospital

Ji'nan, Shandong, China

Institute of hematology and Blood Diseases Hospital, Tianjin

Tianjin, Tianjin Municipality, China

The Second Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, China

Countries

China

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

U1111-1235-5905

Identifier Type: OTHER

Identifier Source: secondary_id

2020-003001-58

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

NN7088-4595

Identifier Type: -

Identifier Source: org_study_id