Trial Outcomes & Findings for Research Study to Look at Side Effects During Regular Injection With Factor VIII Medicine Named Turoctocog Alfa for a 8 Weeks Period (NCT NCT03449342)
NCT ID: NCT03449342
Last Updated: 2020-04-17
Results Overview
The number of participants who confirmed the presence of FVIII inhibitor development (≥ 0.6 BU) during 8 weeks of treatment period.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
60 participants
Primary outcome timeframe
Weeks 0-8
Results posted on
2020-04-17
Participant Flow
The trial was conducted at 10 sites in India.
Participant milestones
| Measure |
Adolescents (12 - <18 Years)
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
|
Adults (≥18 Years)
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
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|---|---|---|
|
Overall Study
STARTED
|
10
|
50
|
|
Overall Study
COMPLETED
|
10
|
50
|
|
Overall Study
NOT COMPLETED
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0
|
0
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Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Research Study to Look at Side Effects During Regular Injection With Factor VIII Medicine Named Turoctocog Alfa for a 8 Weeks Period
Baseline characteristics by cohort
| Measure |
Adolescents (12 - <18 Years)
n=10 Participants
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
|
Adults (≥18 Years)
n=50 Participants
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
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Total
n=60 Participants
Total of all reporting groups
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|---|---|---|---|
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Age, Continuous
|
13.90 years
STANDARD_DEVIATION 1.91 • n=5 Participants
|
27.10 years
STANDARD_DEVIATION 7.28 • n=7 Participants
|
24.90 years
STANDARD_DEVIATION 8.32 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
50 Participants
n=7 Participants
|
60 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=5 Participants
|
50 Participants
n=7 Participants
|
60 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
10 Participants
n=5 Participants
|
50 Participants
n=7 Participants
|
60 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Weeks 0-8Population: Results are based on the full analysis set (FAS), that included all dosed participants with data after dosing during 8 weeks of treatment.
The number of participants who confirmed the presence of FVIII inhibitor development (≥ 0.6 BU) during 8 weeks of treatment period.
Outcome measures
| Measure |
Adults (≥18 Years)
n=50 Participants
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
|
Adolescents (12 - <18 Years)
n=10 Participants
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
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|---|---|---|
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Occurrence of Confirmed FVIII Inhibitor Development (≥ 0.6 BU)
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0 Participants
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0 Participants
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SECONDARY outcome
Timeframe: Weeks 0-12Population: Results are based on the safety analysis set (SAS), that included all dosed participants with data after dosing during 8 weeks of treatment.
Incidence of adverse drug reactions (ARs) and serious adverse reactions (SARs) were calculated as the number of adverse reactions per patient years. All presented ARs and SARs are treatment emergent and related to trial product, which were defined as the events reported after trial product administration until the follow-up, 12 weeks after first treatment.
Outcome measures
| Measure |
Adults (≥18 Years)
n=50 Participants
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
|
Adolescents (12 - <18 Years)
n=10 Participants
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
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|---|---|---|
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Incidence of Adverse Drug Reactions (ARs) and Serious Adverse Reactions (SARs)
Serious adverse reactions
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0 Number of ARs per patient years
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0 Number of ARs per patient years
|
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Incidence of Adverse Drug Reactions (ARs) and Serious Adverse Reactions (SARs)
Adverse drug reaction
|
0 Number of ARs per patient years
|
0 Number of ARs per patient years
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SECONDARY outcome
Timeframe: Weeks 0-8Population: Results are based on the FAS that included all dosed participants with data after dosing during 8 weeks of treatment. "Overall Number of Participants Analyzed" = Number of participants with bleeding episodes treated with turoctocog alfa.
The haemostatic effect (HE) of turoctocog alfa when used for treatment of bleeding episodes was evaluated during 8 weeks of treatment. Successful haemostatic effect means the haemostatic response when used for treatment of a bleeding episode was either excellent or good. Excellent haemostatic respose: Abrupt pain relief and/or clear improvement in objective signs of bleeding episode within approximately 8 hours after a single injection. Good haemostatic response: Definite pain relief and/or improvement in signs of bleeding episode within approximately 8 hours after an injection, but possibly requiring more than 1 injection for complete resolution.
Outcome measures
| Measure |
Adults (≥18 Years)
n=46 bleeding episodes
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
|
Adolescents (12 - <18 Years)
n=3 bleeding episodes
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
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|---|---|---|
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Number of Bleeding Episodes With Successful Haemostatic Effect of Turoctocog Alfa
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38 Bleeding episodes with successfull HE
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2 Bleeding episodes with successfull HE
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SECONDARY outcome
Timeframe: Weeks 0-8Population: Results are based on the FAS that included all dosed participants with data after dosing during 8 weeks of treatment.
Total consumption of turoctocog alfa was evaluated during 8 weeks of treatment and it was presented as IU of turoctocog alfa/kg body weight (BW) per year per participant.
Outcome measures
| Measure |
Adults (≥18 Years)
n=50 Participants
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
|
Adolescents (12 - <18 Years)
n=10 Participants
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
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|---|---|---|
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Total Annualised Consumption of Turoctocog Alfa
|
6086 IU/kg BW/year/participant
Standard Deviation 1735
|
7030 IU/kg BW/year/participant
Standard Deviation 1053
|
SECONDARY outcome
Timeframe: Weeks 0-12Population: Results are based on the SAS, that included all dosed participants with data after dosing during 8 weeks of treatment.
Incidence of allergic or infusion reactions related to trial products were calculated as the number of reactions per patient years. Allergic reactions are a class of adverse events related to allergy.
Outcome measures
| Measure |
Adults (≥18 Years)
n=50 Participants
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
|
Adolescents (12 - <18 Years)
n=10 Participants
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
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|---|---|---|
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Incidence of Allergic or Infusion Reactions Related to the Trial Product
|
0 Number of reactions per patient years
|
0 Number of reactions per patient years
|
Adverse Events
Adolescents (12 - <18 Years)
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Adults (>=18 Years)
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Adolescents (12 - <18 Years)
n=10 participants at risk
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
|
Adults (>=18 Years)
n=50 participants at risk
Participants were to receive prophylactic (preventive) treatment of turoctocog alfa as intravenous (i.v.) injections at a frequency of 'every second day' or '3 times a week' at a dose in the range of 20-50 IU/kg at the investigator's discretion. Dosing for prophylaxis was according to the approved prescribing information. The total treatment duration for each participant was 8 weeks. Bleeds were treated with one or more turoctocog alfa intravenous (i.v.) bolus injections. The individual dose levels were decided by the investigator based on recommendations from the World Federation of Hemophilia (WFH). Participants who underwent surgery were treated with turoctocog alfa according to WFH recommendations.
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|---|---|---|
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Nervous system disorders
Headache
|
10.0%
1/10 • Number of events 1 • Week 0 - 12 (8 weeks of treatment period + 4 weeks of follow-up period).
All the adverse events are based on the safety analysis set (SAS) which included all dosed participants with data after dosing.
|
0.00%
0/50 • Week 0 - 12 (8 weeks of treatment period + 4 weeks of follow-up period).
All the adverse events are based on the safety analysis set (SAS) which included all dosed participants with data after dosing.
|
|
Infections and infestations
Upper respiratory tract infection
|
10.0%
1/10 • Number of events 2 • Week 0 - 12 (8 weeks of treatment period + 4 weeks of follow-up period).
All the adverse events are based on the safety analysis set (SAS) which included all dosed participants with data after dosing.
|
4.0%
2/50 • Number of events 2 • Week 0 - 12 (8 weeks of treatment period + 4 weeks of follow-up period).
All the adverse events are based on the safety analysis set (SAS) which included all dosed participants with data after dosing.
|
Additional Information
Clinical Reporting Anchor and Disclosure (1452)
Novo Nordisk A/S
Phone: (+1) 866-867-7178
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee At the end of the trial, one or more scientific publications may be prepared collaboratively by the investigator(s) and Novo Nordisk. Novo Nordisk reserves the right to postpone publication and/or communication for up to 60 days to protect intellectual property.
- Publication restrictions are in place
Restriction type: OTHER