Clinical Trial Results:
A Multi-centre, Open-label Trial Evaluating Efficacy, Safety and Pharmacokinetics of Turoctocog Alfa Pegol (N8-GP) When used for Treatment and Prophylaxis of Bleeding Episodes in Previously Treated Chinese Patients with Haemophilia A
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Summary
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EudraCT number |
2020-003001-58 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
28 Dec 2022
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Results information
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Results version number |
v1(current) |
This version publication date |
13 Jul 2023
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First version publication date |
13 Jul 2023
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
NN7088-4595
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT05082116 | ||
WHO universal trial number (UTN) |
U1111-1235-5905 | ||
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Sponsors
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Sponsor organisation name |
Novo Nordisk A/S
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Sponsor organisation address |
Novo Allé, Bagsvaerd, Denmark, 2880
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Public contact |
Clinical Reporting Office (2834), Novo Nordisk A/S, clinicaltrials@novonordisk.com
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Scientific contact |
Clinical Reporting Office (2834), Novo Nordisk A/, clinicaltrials@novonordisk.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
27 Feb 2023
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
28 Dec 2022
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
To evaluate the clinical efficacy of turoctocog alfa pegol (N8-GP) in bleeding prophylaxis (number of bleeding episodes during prophylaxis) in Chinese adolescent and adult patients with severe haemophilia A previously treated with other antihemophilic factor (FVIII) products
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Protection of trial subjects |
The trial was conducted in accordance with the Declaration of Helsinki [64th World Medical Association (WMA) 2013] and International Council for Harmonisation Good Clinical Practice, including archiving of essential documents, (2016) and 21 Code of Federal Regulations (CFR) 312.120.
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Background therapy |
Not applicable | ||
Evidence for comparator |
Not applicable | ||
Actual start date of recruitment |
27 Sep 2021
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
China: 36
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Worldwide total number of subjects |
36
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
13
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Adults (18-64 years) |
23
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
A total of 36 Chinese subjects with a severe haemophilia A were recruited in this trial. The trial was conducted at 8 sites in China mainland. | ||||||||||||||||||
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Pre-assignment
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Screening details |
A total of 38 subjects were screened for the trial, of which 2 were screen failures and 36 subjects completed the study treatment. | ||||||||||||||||||
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Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||||||||
Blinding implementation details |
Not Applicable
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Arms
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Are arms mutually exclusive |
Yes
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Arm title
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Adolescents (12-17 years) | ||||||||||||||||||
Arm description |
Subjects with haemophilia A aged 12-17 years with greater than or equal to (≥) 150 exposure days to other FVIII product received one single bolus dose of 50 international unit per Kilogram (IU/kg) of turoctocog alfa pegol (N8-GP), administered intravenously (IV) every 4th day (96 hours) or twice weekly (investigator's discretion). All bleeds were to be treated with doses between 20-75 IU/kg according to the severity and location of the bleeding episode. | ||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||
Investigational medicinal product name |
N8-GP rFVIII
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Investigational medicinal product code |
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Other name |
Turoctocog alfa pegol
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Pharmaceutical forms |
Powder and solvent for solution for injection
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Routes of administration |
Intravenous use
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Dosage and administration details |
Subjects with haemophilia A aged 12-17 years with ≥150 exposure days to other FVIII product received one single bolus dose of 50 IU/kg of N8-GP, administered IV every 4th day (96 hours) or twice weekly (investigator's discretion). All bleeds were to be treated with doses between 20-75 IU/kg according to the severity and location of the bleeding episode.
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Arm title
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Adults (18-70 years) | ||||||||||||||||||
Arm description |
Subjects with haemophilia A aged 18-70 years with ≥150 exposure days to other FVIII product received one single bolus dose of 50 IU/kg of turoctocog alfa pegol (N8-GP), administered IV every 4th day (96 hours) or twice weekly (investigator's discretion). All bleeds were to be treated with doses between 20-75 IU/kg according to the severity and location of the bleeding episode. | ||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||
Investigational medicinal product name |
N8-GP rFVIII
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Investigational medicinal product code |
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Other name |
Turoctocog alfa pegol
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Pharmaceutical forms |
Powder and solvent for solution for injection
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Routes of administration |
Intravenous use
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Dosage and administration details |
Subjects with haemophilia A aged 18-70 years with ≥150 exposure days to other FVIII product received one single bolus dose of 50 IU/kg of N8-GP, administered IV every 4th day (96 hours) or twice weekly (investigator's discretion). All bleeds were to be treated with doses between 20-75 IU/kg according to the severity and location of the bleeding episode.
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| Notes [1] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left. Justification: The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left. Either resolve this issue or provide a justification. [2] - The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left. Justification: The number of subjects at this milestone seems inconsistent with the number of subjects in the arm. It is expected that the number of subjects will be greater than, or equal to the number that completed, minus those who left. Either resolve this issue or provide a justification. |
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Baseline characteristics reporting groups
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Reporting group title |
Adolescents (12-17 years)
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Reporting group description |
Subjects with haemophilia A aged 12-17 years with greater than or equal to (≥) 150 exposure days to other FVIII product received one single bolus dose of 50 international unit per Kilogram (IU/kg) of turoctocog alfa pegol (N8-GP), administered intravenously (IV) every 4th day (96 hours) or twice weekly (investigator's discretion). All bleeds were to be treated with doses between 20-75 IU/kg according to the severity and location of the bleeding episode. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Adults (18-70 years)
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Reporting group description |
Subjects with haemophilia A aged 18-70 years with ≥150 exposure days to other FVIII product received one single bolus dose of 50 IU/kg of turoctocog alfa pegol (N8-GP), administered IV every 4th day (96 hours) or twice weekly (investigator's discretion). All bleeds were to be treated with doses between 20-75 IU/kg according to the severity and location of the bleeding episode. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Adolescents (12-17 years)
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Reporting group description |
Subjects with haemophilia A aged 12-17 years with greater than or equal to (≥) 150 exposure days to other FVIII product received one single bolus dose of 50 international unit per Kilogram (IU/kg) of turoctocog alfa pegol (N8-GP), administered intravenously (IV) every 4th day (96 hours) or twice weekly (investigator's discretion). All bleeds were to be treated with doses between 20-75 IU/kg according to the severity and location of the bleeding episode. | ||
Reporting group title |
Adults (18-70 years)
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Reporting group description |
Subjects with haemophilia A aged 18-70 years with ≥150 exposure days to other FVIII product received one single bolus dose of 50 IU/kg of turoctocog alfa pegol (N8-GP), administered IV every 4th day (96 hours) or twice weekly (investigator's discretion). All bleeds were to be treated with doses between 20-75 IU/kg according to the severity and location of the bleeding episode. | ||
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End point title |
Number of Bleeding Episodes | ||||||||||||
End point description |
Number of bleeding episodes per year data is reported. Annualised bleeding rate (ABR) is the number of bleeding episodes per year. Results were based on the full analysis set (FAS) which included all participants exposed to N8-GP in this trial.
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End point type |
Primary
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End point timeframe |
From start of treatment until visit 7
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Statistical analysis title |
N8-GP Prophylaxis | ||||||||||||
Statistical analysis description |
The analysis is based on a Poisson regression model allowing for over-dispersion. For subjects withdrawing prematurely, the log planned treatment duration is used as offset; for completers, the log actual treatment duration is used.
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Comparison groups |
Adolescents (12-17 years) v Adults (18-70 years)
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Number of subjects included in analysis |
36
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Analysis specification |
Pre-specified
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Analysis type |
other | ||||||||||||
Method |
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Parameter type |
Poisson regression | ||||||||||||
Point estimate |
2.55
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Confidence interval |
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level |
95% | ||||||||||||
sides |
2-sided
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lower limit |
1.24 | ||||||||||||
upper limit |
5.23 | ||||||||||||
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End point title |
Haemostatic Effect of N8-GP When used for Treatment of Bleeding Episodes, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None) | ||||||||||||||||||||||||
End point description |
Haemostatic effect of N8-GP for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Evaluation during trial was done by subject 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. Results were based on the FAS which included all participants exposed to N8-GP in this trial.
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End point type |
Secondary
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End point timeframe |
From start of treatment until visit 7
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| No statistical analyses for this end point | |||||||||||||||||||||||||
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End point title |
Consumption of N8-GP for Treatment of Bleeding Episodes | ||||||||||||
End point description |
The mean number of injections of N8-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). Results were based on the FAS which included all subjects exposed to N8-GP in this trial
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End point type |
Secondary
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End point timeframe |
From start of treatment until visit 7
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Consumption of N8-GP for Prophylaxis | ||||||||||||
End point description |
The mean consumption of N8-GP for prophylaxis per year per subject was reported and it was measured in international units per kilogram per year (IU/kg/year). Results were based on the FAS which included all subjects exposed to N8-GP in this trial.
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End point type |
Secondary
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End point timeframe |
From start of treatment until visit 7
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| No statistical analyses for this end point | |||||||||||||
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End point title |
FVIII Trough Activity During Prophylaxis | ||||||||||||
End point description |
Trough levels of FVIII was reported for all subjects who received prophylaxis treatment. Chromogenic assay was performed with N8-GP product specific standard (PSS) as a calibrator. Data of Visit 7 is presented. Results were based on the FAS which included all subjects exposed to N8-GP in this trial.
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End point type |
Secondary
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End point timeframe |
From start of treatment (excluding the first exposure) until visit 7
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Number of Serious Adverse Events (SAEs) | |||||||||
End point description |
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. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration.Results were based on the SAS which included all subjects exposed to N8-GP in this trial.
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End point type |
Secondary
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End point timeframe |
From start of treatment until end of trial
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| No statistical analyses for this end point | ||||||||||
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End point title |
Number of Adverse Events (AEs) | |||||||||
End point description |
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 N8-GP administration. Results were based on the safety analysis set (SAS) which included all subjects exposed to N8-GP in this trial.
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End point type |
Secondary
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End point timeframe |
From start of treatment until end of trial
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| No statistical analyses for this end point | ||||||||||
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End point title |
Incidence Rate of Confirmed FVIII Inhibitors ≥0.6 BU | ||||||||||||
End point description |
A subject 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 subjects with neutralising antibodies while the denominator was included for all subjects with a minimum of 50 exposures plus any patients with less than 50 exposures but with neutralising inhibitor. Results were based on the FAS which included all subjects exposed to N8-GP in this trial.
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End point type |
Secondary
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End point timeframe |
From start of treatment until visit 7
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| No statistical analyses for this end point | |||||||||||||
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End point title |
FVIII activity 30 min Post-injection (C30min) | ||||||||||||||||||
End point description |
FVIII plasma activity was measured after 30 mins of injection. This was measured at two time points Visit 2a and visit 7 during the study. Chromogenic assay was performed. The Pharmacokinetic (PK) analysis set included sub-set of subjects from FAS who were included for PK assessments. Number analysed = Number of subjects with available data. Here, n= number of subjects from the respective arms analysed for specific timepoints.
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End point type |
Secondary
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End point timeframe |
Single-dose: 30 min ± 5 min post-injection at visit 2a, Steady-state: 30 min ± 5 min post-injection at visit 7
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Incremental Recovery (IR) | ||||||||||||||||||
End point description |
Incremental recovery was defined as the dose-normalised activity recorded 30 min after end of injection. This was measured at two time points Visit 2a and visit 7 during the study. Chromogenic assay was performed. PK analysis set included sub-set of subjects from FAS who were included for PK assessments. Number analysed = Number of subjects with available data for respective arm. Here, n = number of subjects from the respective arms analysed for specific timepoints.
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End point type |
Secondary
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End point timeframe |
Single-dose: 30 min ± 5 min post-injection at visit 2a, Steady-state: 30 min ± 5 min post-injection at visit 7
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Area under the Curve (AUC) | ||||||||||||||||||
End point description |
Area under the plasma activity versus time profile from time zero to infinity (AUC0-inf) and area under the plasma activity versus time profile from time zero to 96 hours (AUC0-96h) were measured at the time points Visit 2a and visit 7 respectively during the study. It is the measure of total plasma exposure. Chromogenic assay was performed. PK analysis set included sub-set of subjects from FAS who were included for PK assessments. Number analysed = Number of subjects with available data. Here, n= number of subjects from the respective arms analysed for specific timepoints.
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End point type |
Secondary
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End point timeframe |
Single-dose: 0−inf post-injection at visit 2a, Steady-state: 0−96 h post-injection at visit 7
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Terminal Half-life (t½) | ||||||||||||||||||
End point description |
t½ = 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. This was measured at Visit 2a and visit 7 during the study. Chromogenic assay was performed. PK analysis set included sub-set of subjects from FAS who were included for PK assessments. Number analysed = Number of subjects with available data. Here, n= number of subjects from the respective arms analysed for specific timepoints.
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End point type |
Secondary
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End point timeframe |
Single-dose: 0−96 h post-injection at visit 2a, Steady-state: 0−96 h post-injection at visit 7
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
FVIII Trough Activity 96 h Post-injection (C96h) | ||||||||||||||||||
End point description |
FVIII plasma activity was measured after 96 h of injection. This was measured at two time points Visit 2a and visit 7 during the study. Chromogenic assay was performed. PK analysis set included sub-set of subjects from FAS who were included for PK assessments. Number analysed = Number of subjects with available data. Here, n= number of subjects from the respective arms analysed for specific timepoints.
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End point type |
Secondary
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End point timeframe |
Single-dose: 96 h ± 8 h post-injection at visit 2a, Steady-state: 96 h ± 8 h post-injection at visit 7
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Clearance (CL) | ||||||||||||||||||
End point description |
Total plasma clearance (CL) of drug after intravenous administration was reported. Clearance was calculated using the formula CL= Dose / AUC0-inf for single dose and CL= Dose / AUC0-96 h for steady state. This was measured at two time points Visit 2a and visit 7 during the study. Chromogenic assay was performed. PK analysis set included sub-set of subjects from FAS who were included for PK assessments. Number analysed = Number of subjects with available data. Here, n= number of subjects from the respective arms analysed for specific timepoints.
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End point type |
Secondary
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End point timeframe |
Single-dose: 0−96 h post-injection at visit 2a, Steady-state: 0−96 h post-injection at visit 7
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| No statistical analyses for this end point | |||||||||||||||||||
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Adverse events information
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Timeframe for reporting adverse events |
From start of treatment until end of trial
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Adverse event reporting additional description |
All presented AEs are TEAEs. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. Results were based on the SAS which included all subjects exposed to N8-GP in this trial.
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Assessment type |
Systematic | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
25.1
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Reporting groups
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Reporting group title |
Adults (18-70 years)
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Reporting group description |
Subjects with haemophilia A aged 18-70 years with ≥150 exposure days to other FVIII product received one single bolus dose of 50 IU/kg of N8-GP, administered IV every 4th day (96 hours) or twice weekly (investigator's discretion). All bleeds were to be treated with doses between 20-75 IU/kg according to the severity and location of the bleeding episode. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Adolescents (12-17 years)
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Reporting group description |
Subjects with haemophilia A aged 12-17 years with ≥150 exposure days to other FVIII product received one single bolus dose of 50 IU/kg of N8-GP, administered IV every 4th day (96 hours) or twice weekly (investigator's discretion). All bleeds were to be treated with doses between 20-75 IU/kg according to the severity and location of the bleeding episode. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
