Haemophilia BPhase 3Paradigm9NCT05365217
A Research Study Investigating Nonacog Beta Pegol (N9-GP) for Treatment and Prevention of Bleedings in Chinese People With Haemophilia B
A Phase 3 study testing Nonacog beta pegol in people with Haemophilia B, run by Novo Nordisk A/S at 15 sites in China.
Trial 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). Read more about this study
- Who is it for?
- Ages 12 to 70 years, men only
- Conditions the study is for
- Haemophilia B
- What is being tested?
- Nonacog beta pegol
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 15 sitesChina
- Study dates
- 18 May 2022to 11 May 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 70 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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 locations and contacts
15 sites in 1 country.
15 sites in 1 country
China15 sites
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, 100045
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730
Xiangya Hospital Central-South University
Changsha, Hunan, 410008
Fujian Medical University Union Hospital-Hematology
Fuzhou, Fujian, 350001
Nanfang Hospital, Southern Medical University-Haematology
Guangzhou, Guangdong, 510515
The Affiliated Hospital of Guizhou Medical University-Hematology
Guiyang, Guizhou, 550004
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310052
Jinan Central Hospital
Jinan, Shandong, 250013
The Second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, 650101
The Affiliated Hospital of Qingdao University
Qingdao, Shandong, 266000
The Affiliated Hospital of Qingdao University
Qingdao, Shandong, 266555
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
Institute of Hematology and Blood Diseases Hospital, Tianjin-Hematology
Tianjin, Tianjin Municipality, 300020
Tongji Hospital, Tongji Medical College of HUST-Hematology
Wuhan, Hubei, 430030
Henan Cancer Hospital-Hematology
Zhengzhou, Henan, 450003
About this study
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
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
- Being tested
- Nonacog beta pegol
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nonacog beta pegolDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Arm A - Nonacog beta pegol (On-demand/Prophylaxis) | Experimental | Participants on on-demand treatment for 28 weeks, thereafter prophylactic treatment Receives: Drug: Nonacog beta pegol |
| Arm B - Nonacog beta pegol (Prophylaxis) | Experimental | Participants on prophylactic treatment only Receives: Drug: Nonacog beta pegol |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
Number of bleeding episodes per year data is reported. Annualised bleeding rate (ABR) is the number of bleeding episodes per year.
Measured: From start of treatment (week 0) until end of treatment (week 50)
Consumption of Nonacog Beta Pegol for Treatment of Bleeding Episodes
The mean number of injections of N9-GP used for treatment of a bleed from start to stop of a bleed was reported and it was measured in international units per kilogram per bleed (IU/kg/bleed).
Measured: 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)
The mean consumption of N9-GP for prophylaxis per year per participant was reported and it was measured in international units per kilogram per year (IU/kg/year).
Measured: From start of treatment (week 0) until end of treatment (week 50)
FIX Trough Levels During Prophylaxis (PPX) Treatment (Arm B Only)
Trough levels of FVIII was reported for all participants who received prophylaxis treatment. Chromogenic assay was performed with N9-GP product specific standard (PSS) as a calibrator. The analysis is based on a mixed model on the log transformed plasma FVIII activity with age group as fixed effect and participants as a random effect. The mean trough is presented back-transformed to the natural scale. The estimated mean/average steady state trough level of FVIII over time (all visits from start of treatment (week 0) until end of treatment) was presented. Data is reported for specific treatment in which participants were a part of at any time from week 0 to end of the treatment (EOT) Week 50, not at specific time points assessed from week 0 to EOT.
Measured: 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)
Number of participants who developed inhibitory antibodies (IA) against FVIII was presented. A participant was said to have FVIII-inhibitors if two consecutive tests, preferably within 2 weeks, were positive (greater than or equal to (≥) 0.6 bethesda unit (BU)). For the calculation of the inhibitor rate the numerator was included for all participants with neutralising antibodies while the denominator was included for all participants with a minimum of 50 exposures plus any participants with less than 50 exposures but with neutralising inhibitor.
Measured: From start of treatment (week 0) until end of treatment (week 50)
Number of Adverse Events (AEs)
An adverse event (AE) was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. All presented AEs are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N9-GP administration.
Measured: From start of treatment (week 0) until end of treatment (week 50)
Number of Serious Adverse Events (SAEs)
A serious adverse event (SAE) is defined as any untoward medical occurrence that at any dose results in death, or is life-threatening, or requires inpatient hospitalization or causes prolongation of existing hospitalization results in persistent or significant disability/incapacity, or may have caused a congenital anomaly/birth defect, or requires intervention to prevent permanent impairment or damage. All presented SAEs are treatment-emergent (any serious adverse events which occurred after trial product administration).
Measured: From start of treatment (week 0) until end of treatment (week 50)
Incremental Recovery (IR) (Arm B Only)
The incremental recovery was calculated by subtracting the FVIII activity (IU/mL) measured in plasma at time 0 from that measured at time 30 min after dosing and dividing this difference by the dose injected at time 0 expressed as international units per kilogram (IU/kg) body weight. FVIII activity was measured with a chromogenic assay.
Measured: Single-dose: 30±10 minutes post injection at week 0, Steady-state: 30±10 minutes post injection at week 12
Terminal Half-life (t½) (Arm B Only)
Terminal half life was calculated as ln(2)/λz; where λz is the terminal elimination rate constant. The terminal elimination rate constant was estimated using linear regression on the terminal part of the log (activity) versus time profile.
Measured: Single-dose: 30±10 minutes post injection at week 0, Steady-state: 30±10 minutes post injection at week 12
Clearance (CL) (Arm B Only)
Clearance (CL) of drug after intravenous administration was reported. Clearance was calculated using the formula CL= Dose / AUC(0-inf) for single dose and CL= Dose / AUC(0-96) h for steady state.
Measured: Single-dose: 0-168 hours post injection at week 0, Steady-state: 0-168 hours post injection at week 12
Area Under the Curve (AUC) (Arm B Only)
Area under the plasma activity versus time profile from time zero to 168 hours (AUC0-168h) was measured.
Measured: Single-dose: 0-168 hours post injection at week 0, Steady-state: 0-168 hours post injection at week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2022
- Study start18 May 2022
- Primary completion11 Apr 2024
- Study completion11 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Haemophilia BStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Transparency dept. 2834, Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05365217
- Sponsor's protocol ID
- NN7999-4670
- Other IDs
- 2021-004947-25, U1111-1260-0438, CTR20220763
- Acronym
- Paradigm9
- Status last verified
- Dec 2025
- First submitted
- 4 May 2022
- Last updated
- 12 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05365217
- Acronym / sponsor ID
- Paradigm9 / NN7999-4670
- Conditions
- Haemophilia B
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 18 May 2022 (actual)
- Primary completion
- 11 Apr 2024 (actual)
- Study completion
- 11 May 2024 (actual)
- First posted
- 9 May 2022 (actual)
- Results
- Posted 31 May 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm A - Nonacog beta pegol (On-demand/Prophylaxis) | Experimental | Drug: Nonacog beta pegolParticipants on on-demand treatment for 28 weeks, thereafter prophylactic treatment |
| Arm B - Nonacog beta pegol (Prophylaxis) | Experimental | Drug: Nonacog beta pegolParticipants on prophylactic treatment only |
Who may qualify
- Ages
- 12 to 70 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (14 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nonacog beta pegolNonacog 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. | Drug | Arm A - Nonacog beta pegol (On-demand/Prophylaxis), Arm B - Nonacog beta pegol (Prophylaxis) |
Primary outcome
Haemostatic Effect of Nonacog Beta Pegol When Used for Treatment of Bleeding Episodes During on Demand and Prophylaxis (PPX)
Time frame: From start of treatment (week 0) until end of treatment (up to week 50)
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.
11 secondary outcomes
Number of Treated Bleeding Episodes During Prophylaxis (PPX) Treatment (Arm B Only)
Time frame: From start of treatment (week 0) until end of treatment (week 50)
Consumption of Nonacog Beta Pegol for Treatment of Bleeding Episodes
Time frame: 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)
Time frame: From start of treatment (week 0) until end of treatment (week 50)
FIX Trough Levels During Prophylaxis (PPX) Treatment (Arm B Only)
Time frame: 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)
Time frame: From start of treatment (week 0) until end of treatment (week 50)
Number of Adverse Events (AEs)
Time frame: From start of treatment (week 0) until end of treatment (week 50)
Number of Serious Adverse Events (SAEs)
Time frame: From start of treatment (week 0) until end of treatment (week 50)
Incremental Recovery (IR) (Arm B Only)
Time frame: Single-dose: 30±10 minutes post injection at week 0, Steady-state: 30±10 minutes post injection at week 12
Terminal Half-life (t½) (Arm B Only)
Time frame: Single-dose: 30±10 minutes post injection at week 0, Steady-state: 30±10 minutes post injection at week 12
Clearance (CL) (Arm B Only)
Time frame: Single-dose: 0-168 hours post injection at week 0, Steady-state: 0-168 hours post injection at week 12
Area Under the Curve (AUC) (Arm B Only)
Time frame: Single-dose: 0-168 hours post injection at week 0, Steady-state: 0-168 hours post injection at week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jan 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Transparency dept. 2834 study director
Novo Nordisk A/S
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Nonacog beta pegol in people with Haemophilia B, run by Novo Nordisk A/S at 15 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Haemostatic Effect of Nonacog Beta Pegol When Used for Treatment of Bleeding Episodes During on Demand and Prophylaxis (PPX) (time frame: From start of treatment (week 0) until end of treatment (up to week 50)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jan 2026; AzTrials last refreshed its copy on 1 Nov 2026.