A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B (Paradigm9)

Novo Nordisk

Status and phase: Completed; Phase 3

Conditions: Haemophilia B

Treatments: Drug: Nonacog beta pegol

Study type: Interventional

Funder types: Industry

Identifiers

NCT05365217

U1111-1260-0438 (Other Identifier)

NN7999-4670

CTR20220763 (Registry Identifier)

2021-004947-25 (EudraCT Number)

Details and patient eligibility

About

The study investigates how well the medicine called nonacog beta pegol (N9-GP) works in Chinese people with haemophilia B. Participants will be treated with N9-GP. This is a medicine that doctors can already prescribe in other countries. The medicine will be injected into a vein (intravenous injection). At the visits to the clinic, the medicine will be injected by the study doctor. When treating themselves at home, participants inject the medicine using a needle and vial set. The study will last for about 12-16 months. The participants will have between 9 and 19 visits to the clinic and possibly also some phone calls with the study doctor. At all visits to the clinic, the participants will have blood samples taken.

Enrollment: 30 patients

Sex: Male

Ages: 12 to 70 years old

Volunteers: No Healthy Volunteers

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 moderate to severe congenital haemophilia B with a factor IX (FIX) activity less than or equal to 2 percent according to medical records.
• Aged 12-70 years (both inclusive) at the time of signing informed consent.
• History of at least 100 exposure days (EDs) to products containing FIX.1.
• Patients currently on prophylaxis or patients currently treated on-demand with at least 6 bleeding episodes during the last 12 months or at least 3 bleeding episodes during the last 6 months.
• The patient, legally authorised representative (LAR) and/or caregiver are capable of assessing a bleeding episode, keeping a diary, performing home treatment of bleeding episodes and otherwise following the trial procedures.

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 FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR interviews.
• Current FIX inhibitors greater than or equal to 0.6 Bethesda unit (BU).
• HIV positive, defined by medical records, with CD4+ count less than or equal 200 per microlitre (μL) and a viral load greater than 200 particles per microlitre or greater than 400000 copies per millilitre (mL) within 6 months of the trial entry. If the data are not available in the medical records within the last 6 months, then the test must be performed at the screening visit.
• Congenital or acquired coagulation disorder other than haemophilia B.
• Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records).
• Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) greater than 3 times the upper limit of normal combined with total bilirubin greater than 1.5 times the upper limit of normal at screening.
• Renal impairment defined as estimated glomerular filtration rate (eGFR) less than or equal to 30 mL/min/1.73 m^2 for serum creatinine measured at screening.
• Any disorder, except for conditions associated with haemophilia B, which in the investigator's opinion might jeopardise the patient's safety or compliance with the protocol.
• Platelet count less than 50×10^9/L at screening.
• Immune modulating or chemotherapeutic medication.
• Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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30 participants in 2 patient groups

Arm A - Nonacog beta pegol (On-demand/Prophylaxis)

Experimental group

Description:

Participants on on-demand treatment for 28 weeks, thereafter prophylactic treatment

Treatment:

Drug: Nonacog beta pegol

Arm B - Nonacog beta pegol (Prophylaxis)

Experimental group

Description:

Participants on prophylactic treatment only

Treatment:

Drug: Nonacog beta pegol