Congenital Bleeding DisorderPhase 3guardian™4NCT01493778
Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A
A Phase 3 study testing turoctocog alfa in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 69 sites in 18 countries.
Trial summary
This trial is conducted in Asia, Europe and North America. The purpose of the trial is to evaluate the safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with haemophilia A. Read more about this study
- Who is it for?
- Ages 0 to 6 years, men only
- Conditions the study is for
- Congenital Bleeding Disorder, Haemophilia A
- What is being tested?
- turoctocog alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 69 sitesin 18 countries
- Study dates
- 17 Sep 2012to 5 Dec 2018
- 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 0 to 6 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Age below 6 years
- Informed consent obtained before any trial-related activities (trial-related activities are any procedure that would not have been performed during normal management of the patient)
- Male patients diagnosed with congenital severe haemophilia A (FVIII level equal to or below 1%)
- No prior use of purified clotting factor products (previous exposure, equal to or less than 5 ED to blood components, e.g. cryoprecipitate, fresh frozen plasma, is accepted) including commercially available NovoEight® /Novoeight®
Who cannot join
- Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products
- Previous participation in this trial defined as withdrawal after administration of trial product
- Congenital or acquired coagulation disorders other than haemophilia A
- Any history of Factor VIII inhibitor
- Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG), routine systemic corticosteroids)
Trial locations and contacts
69 sites in 18 countries.
69 sites in 18 countries
United States25 sites
Novo Nordisk Investigational Site
Augusta, Georgia, 30912
Novo Nordisk Investigational Site
Brooklyn, New York, 11201-5425
Novo Nordisk Investigational Site
Brooklyn, New York, 11220
Novo Nordisk Investigational Site
Charleston, South Carolina, 29425
Novo Nordisk Investigational Site
Charlotte, North Carolina, 28204
Novo Nordisk Investigational Site
Charlottesville, Virginia, 22908
Novo Nordisk Investigational Site
Cincinnati, Ohio, 45229
Novo Nordisk Investigational Site
Dayton, Ohio, 45404
Novo Nordisk Investigational Site
Detroit, Michigan, 48201
Novo Nordisk Investigational Site
Gainesville, Florida, 32610
Novo Nordisk Investigational Site
Las Vegas, Nevada, 89109
Novo Nordisk Investigational Site
Long Beach, California, 90806
Novo Nordisk Investigational Site
Macon, Georgia, 31201
Novo Nordisk Investigational Site
New Brunswick, New Jersey, 08901
Novo Nordisk Investigational Site
Oak Lawn, Illinois, 60453
Novo Nordisk Investigational Site
Oklahoma City, Oklahoma, 73104
Novo Nordisk Investigational Site
Orlando, Florida, 32827
Novo Nordisk Investigational Site
Phoenix, Arizona, 85016-7710
Novo Nordisk Investigational Site
Portland, Oregon, 97239
Novo Nordisk Investigational Site
Sacramento, California, 95817
Novo Nordisk Investigational Site
Salt Lake City, Utah, 84113
Novo Nordisk Investigational Site
Savannah, Georgia, 31404
Novo Nordisk Investigational Site
Tampa, Florida, 33606
Novo Nordisk Investigational Site
Tampa, Florida, 33607
Novo Nordisk Investigational Site
Washington D.C., District of Columbia, 20010-2978
Austria7 sites
Novo Nordisk Investigational Site
Graz, 8036
Novo Nordisk Investigational Site
Innsbruck, 6020
Novo Nordisk Investigational Site
Klagenfurt, A 9026
Novo Nordisk Investigational Site
Linz, 4020
Novo Nordisk Investigational Site
Salzburg, A 5020
Novo Nordisk Investigational Site
Sankt Pölten, A 3100
Novo Nordisk Investigational Site
Vienna, A 1090
China6 sites
Novo Nordisk Investigational Site
Beijing, Beijing Municipality, 100045
Novo Nordisk Investigational Site
Beijing, 100032
Novo Nordisk Investigational Site
Chonqqing, Chongqing Municipality, 400014
Novo Nordisk Investigational Site
Guangzhou, Guangdong, 510515
Novo Nordisk Investigational Site
Hangzhou, Zhejiang, 310003
Novo Nordisk Investigational Site
Tianjin, Tianjin Municipality, 300020
Turkey (Türkiye)5 sites
Novo Nordisk Investigational Site
Adana, 01130
Novo Nordisk Investigational Site
Antalya, 01010
Novo Nordisk Investigational Site
Bornova-IZMIR, 35100
Novo Nordisk Investigational Site
İzmit, 41380
Novo Nordisk Investigational Site
Samsun, 55319
Japan4 sites
Novo Nordisk Investigational Site
Aichi, 466-8560
Novo Nordisk Investigational Site
Hyōgo, 654-0047
Novo Nordisk Investigational Site
Shizuoka, 420-8660
Novo Nordisk Investigational Site
Tokyo, 157-8535
Russia3 sites
Novo Nordisk Investigational Site
Krasnodar, 350007
Novo Nordisk Investigational Site
Moscow, 119049
Novo Nordisk Investigational Site
Saint Petersburg, 191065
Spain3 sites
Novo Nordisk Investigational Site
Barcelona, 08035
Novo Nordisk Investigational Site
El Palmar, 30120
Novo Nordisk Investigational Site
Madrid, 28046
Algeria2 sites
Novo Nordisk Investigational Site
Algiers, 16000
Novo Nordisk Investigational Site
Annaba, 23000
Brazil2 sites
Novo Nordisk Investigational Site
Campinas, São Paulo, 13081-970
Novo Nordisk Investigational Site
Curitiba, Paraná, 80250-060
Greece2 sites
Novo Nordisk Investigational Site
Athens, GR-11527
Novo Nordisk Investigational Site
Thessaloniki, GR 54642
Hungary2 sites
Novo Nordisk Investigational Site
Budapest, 1089
Novo Nordisk Investigational Site
Debrecen, 4032
Poland2 sites
Novo Nordisk Investigational Site
Bydgoszcz, 85-094
Novo Nordisk Investigational Site
Lublin, 20-093
About this study
Official title: Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients With Haemophilia A
This trial is conducted in Asia, Europe and North America. The purpose of the trial is to evaluate the safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with haemophilia A.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Being tested
- turoctocog alfa
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
turoctocog alfaDrug
Patients will be scheduled to receive treatment with turoctocog alfa for at least 100 exposure days. In most cases, treatment will be given at home with intravenous (i.v., into the vein) self-injection by the parent/caregiver/support person.
| Study group | Role | What they receive |
|---|---|---|
| turoctocog alfa | Experimental | Not described Receives: Drug: turoctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence Rate of Factor VIII Inhibitors (Above or Equal to 0.6 BU (Bethesda Units)/mL) for the Main Phase of the Trial
The incidence rate (percentage of participants with inhibitors) of inhibitors defined as inhibitor titres ≥0.6 BU for main phase of the trial.
Measured: From Visit 2 (21 days after screening) to Visit 5 (50-55 exposure day)
Secondary outcomes (13)
Haemostatic Effect of Turoctocog Alfa on Treatment of Bleeds Assessed on a Predefined Four Point Scale: Excellent, Good, Moderate and None
The haemostatic effect of turoctocog alfa was summarised by frequency tables containing count of all bleeds and assessed on a predefined four point scale: Excellent, Good, Moderate and None. The analysis was based on the total number of bleeds and their response to treatment. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Annualised Bleeding Rate (ABR)
Annualised bleeding rate defined as number of bleeds in total per patient per year following treatment were estimated by a Poisson model allowing for over-dispersion. The Poisson estimate is presented with a 95% confidence interval (CI). The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Number of Turoctocog Alfa (N8) Injections Required Per Bleed
The number of injections of turoctocog alfa (N8) required per bleed was calculated as the number of injections of turoctocog alfa used in the time period from start of the bleed to stop of the bleed. The mean number of turoctocog alfa injections required to stop the bleed is presented. The analysis was based on the total number of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Month
Mean total consumption of turoctocog alfa (N8) used for treatment (includes all injections given: prevention, treatment of bleeds and during surgery) per patient per month. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Year
Mean total consumption of turoctocog alfa (N8) used for treatment (includes all injections given: prevention, treatment of bleeds and during surgery) per patient per year. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Consumption of Turoctocog Alfa (N8) (IU/kg/Bleed) Per Bleed
Mean consumption of turoctocog alfa (N8) used for treatment of bleed from start to stop of bleed. The analysis was based on number of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Consumption of Turoctocog Alfa (N8) (IU/kg/Months) for Bleed Prevention
Mean consumption of turoctocog alfa (N8) used for preventive treatment per month per patient. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs) Reported During the Trial Period
Number of adverse events and serious adverse events per patient years of exposure. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial (exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Incidence Rate of Clinically Relevant Inhibitors Defined as an Inhibitor Titre (≥ 0.6 BU/mL) Combined With a Decreased Recovery (<66% of Expected Level)
The incidence rates (number of patients with new inhibitor in the period/number of participants at risk, expressed in percentage) of clinically relevant inhibitors were defined as an inhibitor titre (≥ 0.6 BU) combined with a decreased recovery (<66% of expected level). Incidence rate of clinically relevant inhibitors according to the type of assay used is presented. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation) and the combined main and extension phase (from Visit 2 to end of trial).
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Incidence Rate of High-titre Inhibitors Defined as Inhibitor Titre ≥ 5 BU (Bethesda Units)/mL)
Incidence rate (number of patients with new inhibitor in the period/number of participants at risk, expressed in percentage) of high-titre inhibitors defined as inhibitor titre ≥ 5 BU (Bethesda Units)/mL). The inhibitors were evaluated for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation) and the combined main and extension phase (from Visit 2 to end of trial).
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Change in Total Scores for Parent Reported Treatment Satisfaction
The caregivers/parent reported treatment satisfaction is assessed by the parents using the haemophilia satisfaction questionnaire (HEMO-SAT). The questionnaire contained questions related to treatment that covered 6 domains (ease and convenience, efficacy, burden, specialist, centre and general satisfaction). Adults completing the questionnaire could achieve a score from 0 to 100, with lower scores reflecting greater treatment satisfaction. The scale range for each of the 6 domains was 0-100 with lower scores reflecting greater treatment satisfaction.
The scores of the domains at visit 3 (10th-15th ED), visit 5 (50th-55th ED) and end of trial (EoT) are presented.
Measured: Visit 3 (10th-15th ED); Visit 5 (50th-55th ED); End of trial (within 8 weeks of their last scheduled visit in the extension phase)
Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Month Per Patient)
Resource utilisation and caregiver burden are analysed in terms of average number of days absent from work and use of mobility aids (e.g. wheelchair or crutches) as a consequence of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Year Per Patient)
Resource utilisation and caregiver burden are analysed in terms of average number of days of absence from work and use of mobility aids (e.g. wheelchair or crutches) as a consequence of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 [50-55 exposure day], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Measured: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Dec 2011 (estimated)
- Study start17 Sep 2012
- Primary completion16 Aug 2017
- Study completion5 Dec 2018
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 Congenital Bleeding DisorderStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Registry (GCR, 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01493778
- Sponsor's protocol ID
- NN7008-3809
- Other IDs
- U1111-1119-6116, 2011-001033-16, P/50/2010, JapicCTI-142544, CTR20150455
- Acronym
- guardian™4
- Status last verified
- Nov 2020
- First submitted
- 15 Dec 2011
- Last updated
- 30 Nov 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Paediatric Previously Untreated Patients With Haemophilia A
- ClinicalTrials.gov ID
- NCT01493778
- Acronym / sponsor ID
- guardian™4 / NN7008-3809
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 17 Sep 2012 (actual)
- Primary completion
- 16 Aug 2017 (actual)
- Study completion
- 5 Dec 2018 (actual)
- First posted
- 16 Dec 2011 (estimated)
- Results
- Posted 30 Oct 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This trial is conducted in Asia, Europe and North America. The purpose of the trial is to evaluate the safety and efficacy of turoctocog alfa in prevention and treatment of bleeds in previously untreated children with haemophilia A.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| turoctocog alfa | Experimental | Drug: turoctocog alfa |
Who may qualify
- Ages
- 0 to 6 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Age below 6 years
- Informed consent obtained before any trial-related activities (trial-related activities are any procedure that would not have been performed during normal management of the patient)
- Male patients diagnosed with congenital severe haemophilia A (FVIII level equal to or below 1%)
- No prior use of purified clotting factor products (previous exposure, equal to or less than 5 ED to blood components, e.g. cryoprecipitate, fresh frozen plasma, is accepted) including commercially available NovoEight® /Novoeight®
Key exclusion criteria (5 listed)
- Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products
- Previous participation in this trial defined as withdrawal after administration of trial product
- Congenital or acquired coagulation disorders other than haemophilia A
- Any history of Factor VIII inhibitor
- Ongoing treatment or planned treatment during the trial with immunomodulatory agents (e.g. intravenous immunoglobulin (IVIG), routine systemic corticosteroids)
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 |
|---|---|---|
| turoctocog alfaPatients will be scheduled to receive treatment with turoctocog alfa for at least 100 exposure days. In most cases, treatment will be given at home with intravenous (i.v., into the vein) self-injection by the parent/caregiver/support person. | Drug | turoctocog alfa |
Primary outcome
Incidence Rate of Factor VIII Inhibitors (Above or Equal to 0.6 BU (Bethesda Units)/mL) for the Main Phase of the Trial
Time frame: From Visit 2 (21 days after screening) to Visit 5 (50-55 exposure day)
The incidence rate (percentage of participants with inhibitors) of inhibitors defined as inhibitor titres ≥0.6 BU for main phase of the trial.
13 secondary outcomes
Haemostatic Effect of Turoctocog Alfa on Treatment of Bleeds Assessed on a Predefined Four Point Scale: Excellent, Good, Moderate and None
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Annualised Bleeding Rate (ABR)
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Number of Turoctocog Alfa (N8) Injections Required Per Bleed
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Month
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Year
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Consumption of Turoctocog Alfa (N8) (IU/kg/Bleed) Per Bleed
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Consumption of Turoctocog Alfa (N8) (IU/kg/Months) for Bleed Prevention
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs) Reported During the Trial Period
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Incidence Rate of Clinically Relevant Inhibitors Defined as an Inhibitor Titre (≥ 0.6 BU/mL) Combined With a Decreased Recovery (<66% of Expected Level)
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Incidence Rate of High-titre Inhibitors Defined as Inhibitor Titre ≥ 5 BU (Bethesda Units)/mL)
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Change in Total Scores for Parent Reported Treatment Satisfaction
Time frame: Visit 3 (10th-15th ED); Visit 5 (50th-55th ED); End of trial (within 8 weeks of their last scheduled visit in the extension phase)
Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Month Per Patient)
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Year Per Patient)
Time frame: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trial
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Clinical Trials at Novo NordiskLink provided by the sponsor.
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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.
Global Clinical Registry (GCR, 1452) study director
Novo Nordisk A/S
69 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing turoctocog alfa in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 69 sites in 18 countries.
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 4 inclusion and 5 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 30 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 69 sites in United States, Austria, China, Turkey (Türkiye), Japan and 13 more countries. 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 30 Oct 2018). 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?
Incidence Rate of Factor VIII Inhibitors (Above or Equal to 0.6 BU (Bethesda Units)/mL) for the Main Phase of the Trial (time frame: From Visit 2 (21 days after screening) to Visit 5 (50-55 exposure day)). 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 30 Nov 2020.