New hope for hemophilia b: trial tests Long-Acting clotting factor in chinese patients
NCT ID NCT05365217
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a medicine called nonacog beta pegol (N9-GP) in 30 Chinese men with moderate to severe hemophilia B. The medicine is injected into a vein to help blood clot and prevent or treat bleeding episodes. The trial lasted about 12-16 months and checked how well the medicine worked and how safe it was.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- nonacog beta pegol (a clotting factor IX medicine)
- What this could lead to
- If successful, this could provide a safe and effective option for Chinese patients with hemophilia B to control and prevent bleeding episodes.
- What could go wrong
- This is a small, completed phase 3 trial with only 30 participants, so results may not apply to all patients. The medicine is already approved elsewhere, but its long-term safety and effectiveness in this specific population need confirmation.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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30 people
The number who actually took part.
- Started
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May 2022
- Finished
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May 2024
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 to 70 years
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, 100045, China
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Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310052, China
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Fujian Medical University Union Hospital-Hematology
Fuzhou, Fujian, 350001, China
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Henan Cancer Hospital-Hematology
Zhengzhou, Henan, 450003, China
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Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology
Tianjin, Tianjin Municipality, 300020, China
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Jinan Central Hospital
Jinan, Shandong, 250013, China
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Nanfang Hospital, Southern Medical University-Haematology
Guangzhou, Guangdong, 510515, China
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Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730, China
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The Affiliated Hospital of Guizhou Medical University-Hematology
Guiyang, Guizhou, 550004, China
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The Affiliated Hospital of Qingdao University
Qingdao, Shandong, 266000, China
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The Affiliated Hospital of Qingdao University
Qingdao, Shandong, 266555, China
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The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006, China
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The Second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, 650101, China
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Tongji Hospital, Tongji Medical College of HUST-Hematology
Wuhan, Hubei, 430030, China
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Xiangya Hospital Central-South University
Changsha, Hunan, 410008, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Japanese hemophilia patients monitored on alhemo in new safety study
- Researchers track real-world safety of haemophilia b drug over nearly a decade
- Weekly shot could help kids with hemophilia b avoid bleeding
- New hope for kids with hemophilia b: Long-Acting drug shows promise in preventing bleeds
- Japanese hemophilia b patients monitored on refixia – no surprises found