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

NCT05365217 | Completed Phase 3 Results FDA Drug

• 4 other identifiers: NN7999-46702021-004947-25U1111-1260-0438CTR20220763
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 15

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.

Conditions

Haemophilia B

Outcome Measures

Primary Outcomes (1)

• Haemostatic Effect of Nonacog Beta Pegol When Used for Treatment of Bleeding Episodes During on Demand and Prophylaxis (PPX)

  Haemostatic effect of N9-GP for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Evaluation during trial was done by participant and/or parent(s)/caregiver within approximately 8 hours after a single injection as follows: Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hrs after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hrs after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms.

  From start of treatment (week 0) until end of treatment (up to week 50)

Secondary Outcomes (11)

• Number of Treated Bleeding Episodes During Prophylaxis (PPX) Treatment (Arm B Only)

  From start of treatment (week 0) until end of treatment (week 50)

• Consumption of Nonacog Beta Pegol for Treatment of Bleeding Episodes

  From start of treatment (week 0) until end of treatment (up to week 50)

• Consumption of Nonacog Beta Pegol for Prophylaxis (PPX) Treatment (Arm B Only)

  From start of treatment (week 0) until end of treatment (week 50)

• FIX Trough Levels During Prophylaxis (PPX) Treatment (Arm B Only)

  From start of treatment (week 0) until end of treatment (week 50)

• Number of Participants With Inhibitory Antibodies Against FIX Defined as Titre ≥0.6 Bethesda Units (BU)

  From start of treatment (week 0) until end of treatment (week 50)

Study Arms (2)

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

EXPERIMENTAL

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

Drug: Nonacog beta pegol

Arm B - Nonacog beta pegol (Prophylaxis)

EXPERIMENTAL

Participants on prophylactic treatment only

Drug: Nonacog beta pegol

Interventions

Nonacog beta pegol DRUG

Nonacog beta pegol is administered as intravenous injections. Participants will receive nonacog beta pegol as prophylaxis, as on-demand for treatment of bleeding episodes and in relation to surgery.

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

Eligibility Criteria

• Age: 12 Years - 70 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 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.

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 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.

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (15)

Beijing Children's Hospital, Capital Medical University

Beijing, Beijing Municipality, 100045, China

Location

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, 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

Henan Cancer Hospital-Hematology

Zhengzhou, Henan, 450003, China

Location

Tongji Hospital, Tongji Medical College of HUST-Hematology

Wuhan, Hubei, 430030, China

Location

Xiangya Hospital Central-South University

Changsha, Hunan, 410008, China

Location

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

Location

Jinan Central Hospital

Jinan, Shandong, 250013, China

Location

The Affiliated Hospital of Qingdao University

Qingdao, Shandong, 266000, China

Location

The Affiliated Hospital of Qingdao University

Qingdao, Shandong, 266555, China

Location

Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology

Tianjin, Tianjin Municipality, 300020, China

Location

The Second Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, 650101, China

Location

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310052, China

Location

MeSH Terms

Conditions

Hemophilia B

Interventions

nonacog beta pegol

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; Genetic Diseases, X-Linked

Results Point of Contact

• Title: Clinical Reporting Anchor and Disclosure (1452)
• Organization: Novo Nordisk A/S

clinicaltrials@novonordisk.com

(+1) 866-867-7178

Study Officials

• Clinical Transparency dept. 2834

  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: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 4, 2022
• First Posted: May 9, 2022
• Study Start: May 18, 2022
• Primary Completion: April 11, 2024
• Study Completion: May 11, 2024
• Last Updated: January 12, 2026
• Results First Posted: May 31, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

CN (15)