Clinical trial NCT05365217: A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B
Official title: A Multi-centre, Open-label Trial Evaluating Efficacy, Safety and Pharmacokinetics of Nonacog Beta Pegol When Used for Treatment and Prophylaxis of Bleeding Episodes in Chinese Patients With Haemophilia B
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Haemophilia B
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2022-05-09
Enrollment: 30
Sex: MALE
Minimum age (years): 12
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


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 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.

Primary outcomes:
measure: Haemostatic Effect of Nonacog Beta Pegol When Used for Treatment of Bleeding Episodes During on Demand and Prophylaxis (PPX)
timeFrame: From start of treatment (week 0) until end of treatment (up to week 50)
Secondary outcomes:
measure: Number of Treated Bleeding Episodes During Prophylaxis (PPX) Treatment (Arm B Only)
timeFrame: From start of treatment (week 0) until end of treatment (week 50)
measure: Consumption of Nonacog Beta Pegol for Treatment of Bleeding Episodes
timeFrame: From start of treatment (week 0) until end of treatment (up to week 50)
measure: Consumption of Nonacog Beta Pegol for Prophylaxis (PPX) Treatment (Arm B Only)
timeFrame: From start of treatment (week 0) until end of treatment (week 50)
measure: FIX Trough Levels During Prophylaxis (PPX) Treatment (Arm B Only)
timeFrame: From start of treatment (week 0) until end of treatment (week 50)
measure: Number of Participants With Inhibitory Antibodies Against FIX Defined as Titre ≥0.6 Bethesda Units (BU)
timeFrame: From start of treatment (week 0) until end of treatment (week 50)
measure: Number of Adverse Events (AEs)
timeFrame: From start of treatment (week 0) until end of treatment (week 50)
measure: Number of Serious Adverse Events (SAEs)
timeFrame: From start of treatment (week 0) until end of treatment (week 50)
measure: Incremental Recovery (IR) (Arm B Only)
timeFrame: Single-dose: 30±10 minutes post injection at week 0, Steady-state: 30±10 minutes post injection at week 12
measure: Terminal Half-life (t½) (Arm B Only)
timeFrame: Single-dose: 30±10 minutes post injection at week 0, Steady-state: 30±10 minutes post injection at week 12
measure: Clearance (CL) (Arm B Only)
timeFrame: Single-dose: 0-168 hours post injection at week 0, Steady-state: 0-168 hours post injection at week 12
measure: Area Under the Curve (AUC) (Arm B Only)
timeFrame: Single-dose: 0-168 hours post injection at week 0, Steady-state: 0-168 hours post injection at week 12
Other outcomes:
Locations:
city: Beijing
country: China
facility: Beijing Children's Hospital，Capital Medical University
state: Beijing
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
city: Fuzhou
country: China
facility: Fujian Medical University Union Hospital-Hematology
state: Fujian
city: Guangzhou
country: China
facility: Haemotology, Nanfang Hospital, Southern Medical University
state: Guangdong
city: Guiyang
country: China
facility: The Affiliated hospital of Guizhou Medical University-Hemato
state: Guizhou
city: Zhengzhou
country: China
facility: Henan Cancer Hospital-Hematology
state: Henan
city: Wuhan
country: China
facility: Tongji Hospital, Tongji Medical College of HUST-Hematology
state: Hubei
city: Changsha
country: China
facility: Xiangya Hospital Central-South University
state: Hunan
city: Suzhou
country: China
facility: The First Affiliated Hospital of Soochow University
state: Jiangsu
city: Ji'nan
country: China
facility: Jinan Central Hospital
state: Shandong
city: Qingdao
country: China
facility: The Affiliated Hospital of Qingdao University
state: Shandong
city: Tianjin
country: China
facility: Institute of hematology and Blood Diseases Hospital, Tianjin-Hematology
state: Tianjin
city: Kunming
country: China
facility: The Second Affiliated Hospital of Kunming Medical University
state: Yunnan
city: Hangzhou
country: China
facility: Children's Hospital, Zhejiang University school of medicine
state: Zhejiang
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Transparency dept. 2834
role: STUDY_DIRECTOR
References: