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 Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
30
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.
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, China
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
Fujian Medical University Union Hospital-Hematology
Fuzhou, Fujian, China
Nanfang Hospital, Southern Medical University-Haematology
Guangzhou, Guangdong, China
The Affiliated Hospital of Guizhou Medical University-Hematology
Guiyang, Guizhou, China
Henan Cancer Hospital-Hematology
Zhengzhou, Henan, China
Tongji Hospital, Tongji Medical College of HUST-Hematology
Wuhan, Hubei, China
Xiangya Hospital Central-South University
Changsha, Hunan, China
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
Jinan Central Hospital
Jinan, Shandong, China
...and 5 more locations
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.
Time frame: From start of treatment (week 0) until end of treatment (up to week 50)
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.
Time frame: 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).
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)
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).
Time frame: 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.
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)
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.
Time frame: 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.
Time frame: 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).
Time frame: 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.
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)
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.
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)
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.
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)
Area under the plasma activity versus time profile from time zero to 168 hours (AUC0-168h) was measured.
Time frame: Single-dose: 0-168 hours post injection at week 0, Steady-state: 0-168 hours post injection at week 12
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.