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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01480180
Other study ID # NN7088-3859
Secondary ID U1111-1119-74162
Status Completed
Phase Phase 3
First received
Last updated
Start date January 30, 2012
Est. completion date December 10, 2018

Study information

Verified date November 2020
Source Novo Nordisk A/S
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This trial is conducted globally. The aim of the trial is to evaluate the safety and efficacy, including pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in subjects with Haemophilia A.


Recruitment information / eligibility

Status Completed
Enrollment 186
Est. completion date December 10, 2018
Est. primary completion date December 10, 2018
Accepts healthy volunteers No
Gender Male
Age group 12 Years and older
Eligibility Inclusion Criteria: - Male patients with severe congenital haemophilia A (FVIII activity below 1%, according to medical records) - Documented history of at least 150 EDs (exposure days) to other FVIII products - At least 12 years and body weight at least 35 kg (except for Croatia, France, Russia, Israel and the Netherlands where the lower age limit will be 18 years) Exclusion Criteria: - Previous participation in this trial defined as withdrawal after administration N8-GP - Any history of FVIII inhibitors - FVIII inhibitors above or equal to 0.6 BU/mL at screening - HIV (human immunodeficiency virus) positive, defined by medical records with CD4+ (T-lymphocyte subtype) count below or equal to 200/mcL or a viral load of more than 400000 copies/mL. If the data is not available in medical records within last 6 months, CD4+ will be measured at the screening visit - Congenital or acquired coagulation disorders other than haemophilia A - Previous significant thromboembolic events (e.g. myocardial infarction, cerebrovascular disease or deep venous thrombosis) as defined by available medical records - Platelet count below 50,000 platelets/mcL (laboratory value at the screening visit) - ALAT (alanine aminotransferase) above 3 times the upper limit of normal reference ranges at central laboratory - Creatinine level equal to or greater than 1.5 times above upper normal limit (according to central laboratory reference ranges) - Ongoing immune modulating or chemotherapeutic medication

Study Design


Intervention

Drug:
turoctocog alfa pegol
Administered i.v.

Locations

Country Name City State
Australia Novo Nordisk Investigational Site Camperdown New South Wales
Australia Novo Nordisk Investigational Site Parkville Victoria
Australia Novo Nordisk Investigational Site South Brisbane Queensland
Brazil Novo Nordisk Investigational Site Campinas Sao Paulo
Bulgaria Novo Nordisk Investigational Site Sofia
Croatia Novo Nordisk Investigational Site Split
Croatia Novo Nordisk Investigational Site Zagreb
Denmark Novo Nordisk Investigational Site Århus N
Denmark Novo Nordisk Investigational Site København Ø
France Novo Nordisk Investigational Site Bron Cedex
France Novo Nordisk Investigational Site Kremlin-Bicêtre
France Novo Nordisk Investigational Site Nantes Cedex 1
France Novo Nordisk Investigational Site Paris
Germany Novo Nordisk Investigational Site Berlin
Germany Novo Nordisk Investigational Site Bonn
Germany Novo Nordisk Investigational Site Frankfurt/M.
Germany Novo Nordisk Investigational Site Hannover
Germany Novo Nordisk Investigational Site Homburg
Germany Novo Nordisk Investigational Site München
Hungary Novo Nordisk Investigational Site Budapest
Hungary Novo Nordisk Investigational Site Debrecen
Israel Novo Nordisk Investigational Site Tel-Hashomer
Italy Novo Nordisk Investigational Site Firenze
Italy Novo Nordisk Investigational Site Milano
Italy Novo Nordisk Investigational Site Udine
Italy Novo Nordisk Investigational Site Vicenza
Japan Novo Nordisk Investigational Site Aichi
Japan Novo Nordisk Investigational Site Hiroshima
Japan Novo Nordisk Investigational Site Kitakyusyu, Fukuoka
Japan Novo Nordisk Investigational Site Nara
Japan Novo Nordisk Investigational Site Saitama
Japan Novo Nordisk Investigational Site Shimotsuke-shi, Tochigi
Japan Novo Nordisk Investigational Site Shizuoka
Japan Novo Nordisk Investigational Site Tokyo
Japan Novo Nordisk Investigational Site Tokyo
Japan Novo Nordisk Investigational Site Tokyo
Japan Novo Nordisk Investigational Site Yokohama-shi, Kanagawa
Korea, Republic of Novo Nordisk Investigational Site Daejeon
Malaysia Novo Nordisk Investigational Site Kuala Lumpur
Malaysia Novo Nordisk Investigational Site Selangor Darul Ehsan
Netherlands Novo Nordisk Investigational Site Groningen
Netherlands Novo Nordisk Investigational Site Rotterdam
Norway Novo Nordisk Investigational Site Oslo
Puerto Rico Novo Nordisk Investigational Site San Juan
Russian Federation Novo Nordisk Investigational Site Saint-Petersburg
Spain Novo Nordisk Investigational Site Madrid
Spain Novo Nordisk Investigational Site Málaga
Sweden Novo Nordisk Investigational Site Malmö
Switzerland Novo Nordisk Investigational Site Genève 14
Switzerland Novo Nordisk Investigational Site Lausanne
Switzerland Novo Nordisk Investigational Site Zürich
Taiwan Novo Nordisk Investigational Site Changhua
Taiwan Novo Nordisk Investigational Site Taipei
Turkey Novo Nordisk Investigational Site Adana
Turkey Novo Nordisk Investigational Site Bornova-IZMIR
Turkey Novo Nordisk Investigational Site Samsun
United Kingdom Novo Nordisk Investigational Site Basingstoke
United Kingdom Novo Nordisk Investigational Site Cardiff
United Kingdom Novo Nordisk Investigational Site London
United Kingdom Novo Nordisk Investigational Site London
United Kingdom Novo Nordisk Investigational Site Oxford
United Kingdom Novo Nordisk Investigational Site Sheffield
United States Novo Nordisk Investigational Site Augusta Georgia
United States Novo Nordisk Investigational Site Baltimore Maryland
United States Novo Nordisk Investigational Site Boise Idaho
United States Novo Nordisk Investigational Site Boston Massachusetts
United States Novo Nordisk Investigational Site Charleston South Carolina
United States Novo Nordisk Investigational Site Charlottesville Virginia
United States Novo Nordisk Investigational Site Cincinnati Ohio
United States Novo Nordisk Investigational Site Dayton Ohio
United States Novo Nordisk Investigational Site Detroit Michigan
United States Novo Nordisk Investigational Site East Lansing Michigan
United States Novo Nordisk Investigational Site Houston Texas
United States Novo Nordisk Investigational Site Iowa City Iowa
United States Novo Nordisk Investigational Site Long Beach California
United States Novo Nordisk Investigational Site Minneapolis Minnesota
United States Novo Nordisk Investigational Site Nashville Tennessee
United States Novo Nordisk Investigational Site New Brunswick New Jersey
United States Novo Nordisk Investigational Site New Orleans Louisiana
United States Novo Nordisk Investigational Site Newark New Jersey
United States Novo Nordisk Investigational Site Norfolk Virginia
United States Novo Nordisk Investigational Site Omaha Nebraska
United States Novo Nordisk Investigational Site Orlando Florida
United States Novo Nordisk Investigational Site Philadelphia Pennsylvania
United States Novo Nordisk Investigational Site Philadelphia Pennsylvania
United States Novo Nordisk Investigational Site Phoenix Arizona
United States Novo Nordisk Investigational Site Portland Oregon
United States Novo Nordisk Investigational Site Sacramento California
United States Novo Nordisk Investigational Site Spokane Washington
United States Novo Nordisk Investigational Site Tampa Florida
United States Novo Nordisk Investigational Site Torrance California
United States Novo Nordisk Investigational Site Washington District of Columbia
United States Novo Nordisk Investigational Site Washington District of Columbia

Sponsors (1)

Lead Sponsor Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  Australia,  Brazil,  Bulgaria,  Croatia,  Denmark,  France,  Germany,  Hungary,  Israel,  Italy,  Japan,  Korea, Republic of,  Malaysia,  Netherlands,  Norway,  Puerto Rico,  Russian Federation,  Spain,  Sweden,  Switzerland,  Taiwan,  Turkey,  United Kingdom, 

References & Publications (3)

Giangrande P, Abdul Karim F, Nemes L, You CW, Landorph A, Geybels MS, Curry N. Long-term safety and efficacy of N8-GP in previously treated adults and adolescents with hemophilia A: Final results from pathfinder2. J Thromb Haemost. 2020 Sep;18 Suppl 1:5-1 — View Citation

Giangrande P, Andreeva T, Chowdary P, Ehrenforth S, Hanabusa H, Leebeek FW, Lentz SR, Nemes L, Poulsen LH, Santagostino E, You CW, Clausen WH, Jönsson PG, Oldenburg J; Pathfinder™2 Investigators. Clinical evaluation of glycoPEGylated recombinant FVIII: Ef — View Citation

Giangrande P, Chowdary P, Enhrenforth S, Hanabusa H, Leebeek FW, Lentz SR, Nemes L, Poulsen LH, Santagostino E, You CW, Clausen WHO, Oldenburg J and on behalf of for the pathfinder™2 Investigators. Clinical evaluation of novel recombinant glycopegylated F

Outcome

Type Measure Description Time frame Safety issue
Primary The Incidence Rate of FVIII-inhibitors =0.6 BU: After Approximately 19 Months All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as =0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors. After approximately 19 months
Primary Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 19 Months Annualised bleeding rate (ABR) is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP. After approximately 19 months
Primary The Incidence Rate of FVIII-inhibitors =0.6 BU: After Approximately 25 Months All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as =0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors. After approximately 25 months
Primary Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 25 Months ABR is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP. After approximately 25 months
Primary Incidence Rate of FVIII-inhibitors =0.6 BU: At Approximately 80 Months All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as =0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors. At approximately 80 months
Primary Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 80 Months Annualised bleeding rate (ABR) is the number of bleeding episodes per year reported during the prophylactic treatment with N8-GP. After approximately 80 months
Secondary Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 19 Months 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 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. After approximately 19 months
Secondary Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 25 Months 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 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. After approximately 25 months
Secondary Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 80 Months 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 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. After approximately 80 months
Secondary Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 19 Months The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported. After approximately 19 months
Secondary Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 25 Months The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported. After approximately 25 months
Secondary Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 80 Months The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported. After approximately 80 months
Secondary Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 19 Months The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported. After approximately 19 months
Secondary Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 25 Months The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported. After approximately 25 months
Secondary Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 80 Months The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported. After approximately 80 months
Secondary Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 19 Months Number of infusions are presented as average dose used during propphylaxis and on-demand treatment. After approximately 19 months
Secondary Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 25 Months Number of infusions are presented as average dose used during prophylaxis and on-demand treatment. After approximately 25 months
Secondary Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 80 Months Number of infusions are presented as average dose used during prophylaxis and on-demand treatment. After approximately 80 months
Secondary Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 19 Months The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported. After approximately 19 months
Secondary Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 25 Months The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported. After approximately 25 months
Secondary Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 80 Months The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported. After approximately 80 months
Secondary Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 19 Months The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported. After approximately 19 months
Secondary Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 25 Months The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported. After approximately 25 months
Secondary Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 80 Months The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported. After approximately 80 months
Secondary Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 19 Months Recovery and trough levels of FVIII:C was reported for all participants at Visit 3 (Week 4) and end of main phase (approx. 19 months). The data was reported for all participants who received prophylaxis treatment. Chromogenic assay was performed with N8-GP product specific standard (PSS) as a calibrator. After approximately 19 months
Secondary Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 25 Months Recovery and trough levels of FVIII:C was reported for all participants at the end of extension phase 1 study (approx. 25 months). The data was reported for all participants who received prophylaxis treatment. Chromogenic assay was performed with N8-GP product specific standard (PSS) as a calibrator. After approximately 25 months
Secondary Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 80 Months Since patients were allowed to change prophylaxis regimen at any time during the extension phase part 2, and since the visit intervals were different for the 2 prophylaxis treatment regimens (Q4D and Q7D), FVIII activity data are reported only as incremental recovery at this timepoint. After approximately 80 months
Secondary Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Patients 13-16 Years Old) After Approx 19 and 25 Months Reported results are change from baseline (Month 0) measured at end of main phase (approx Month 19) and change from Month 19 at end of Extension 1 (approx Month 25) of the study. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. After approx 19 and 25 months
Secondary Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Patients 13-16 Years Old): After Approx 80 Months Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs
Secondary Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Parents of Patients 13-16 Years Old): After Approx 19 and 25 Months Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The questionnaire was completed by parents of the patients in the 13-16 years old age bracket. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. After approx 19 and 25 months
Secondary Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Parents of Patients 13-16 Years Old): After Approx 80 Months Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. The questionnaire was completed by parents of the patients in the 13-16 years old age bracket. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs
Secondary Patient Reported Outcomes - Change in HAEM-A-QOL (>=17 Years) Total Scores: After Approx 19 and 25 Months Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The HAEM-A-QOL (for adults (>=17 years)) assessment included questions on questions on physical health, feeling, view of yourself, sports and leisure, work and school, dealing with haemophilia, treatment, future, family planning, and partnership and sexuality. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. After approx 19 and 25 months
Secondary Patient Reported Outcomes - Change in HAEM-A-QOL (>=17 Years) Total Scores: After Approximately 80 Months Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The HAEM-A-QOL (for adults (>=17 years)) assessment included questions on physical health, feeling, view of yourself, sports and leisure, work and school, dealing with haemophilia, treatment, future, family planning, and partnership and sexuality. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs
Secondary Patient Reported Outcomes - Change in HEMO-SAT (Patients) Scores: After Approx 19 and 25 Months Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The HEMO-SAT (Hematology-satisfaction) assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction (reported by patients). 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. A decrease in the score would mean improvement. After approx 19 and 25 months
Secondary Patient Reported Outcomes - Change in HEMO-SAT (Patients) Scores: After Approximately 80 Months Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The HEMO-SAT assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction (reported by patients). 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. A decrease in the score would mean improvement. 1-<2 yrs, 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs
Secondary Patient Reported Outcomes - Change in HEMO-SAT Scores (Parents): After Approx 19 and 25 Months The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). The HEMO-SAT assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction (reported by parents). 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. A decrease in the score would mean improvement. After approx 19 and 25 months
Secondary Patient Reported Outcomes - Change in HEMO-SAT (Parents) Scores: After Approximately 80 Months Reported results are from Visit 1 (Month 0), and change from visit 1 upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Max. no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = max. number of participants contributed to the analysis for each time point. The HEMO-SAT assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, 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. A decrease in the score would mean improvement. 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs
Secondary Patient Reported Outcomes - Change in EQ-5D-VAS Scores: After Approx 19 and 25 Months Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The European quality of life visual analogue scale (EQ5D-VAS) records the patient's self-rated health on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'. The VAS can be used as a quantitative measure of health outcome that reflect the patient's own judgement. EQ-5D-VAS: range 0 to 100. A higher score indicates better self reported health status. A positive change indicates an improvement. After approx 19 and 25 months
Secondary Patient Reported Outcomes - Change in EQ-5D-VAS Scores: After Approximately 80 Months Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The EQ5D-VAS records the patient's self-rated health on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'. The VAS can be used as a quantitative measure of health outcome that reflect the patient's own judgement. EQ-5D-VAS: range 0 to 100. A higher score indicates better self reported health status. A positive change indicates an improvement. 1-<2 yrs, 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs
Secondary Patient Reported Outcomes - Change in European Quality of Life Utility Index: After Approx 19 and 25 Months Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The European quality of life utility index comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels where 1 indicates better health state (no problems) and 3 indicates worst health state (confined to bed). Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score is transformed and results in a total score range -0.594 to 1.000; a positive change indicates an improvement. After approx 19 and 25 months
Secondary Patient Reported Outcomes - Change in European Quality of Life Utility Index Scores: After Approximately 80 Months Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It is possible that a patient answers more than one questionnaire in a single time interval. Overall units analysed = Max no. of questionnaires answered by participants for this endpoint. Overall no. of participants analysed = max no. of participants analysed at each time point. This utility index has 5 dimensions: mobility, self-care, usual activities, pain/discomfort & anxiety/depression. Each dimension has 3 levels where 1 indicates better health state (no problems) and 3 indicates worst health state (confined to bed). Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score is transformed and results in a total score range -0.594 to 1.000; a positive change indicates an improvement. 1-<2 yrs, 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs
Secondary Number of Hospital Admissions During the Trial The number of hospital admissions that took place in the study were reported. After approx 19, 25 and 80 months
Secondary Number of Days at the Hospital During the Trial The mean number of days that participants spent at the hospital during the study were reported. After approx 19, 25 and 80 months
Secondary Number of Admissions to the Emergency Room (ER) During the Trial The number of admissions to the ER that took place in the study were reported for each group. After approx 19, 25 and 80 months
Secondary Number of Days Missing School or Work The mean number of days that participants missed to go to school or work were reported. Approx 19, 25 and 80 months
Secondary Number of Days Using Mobility Aid The mean number of days that participants used any aids for mobility during the study were reported. Approx 19, 25 and 80 months
Secondary Number of Participants Using Pain Medication The number of participants using pain medication during the main plus extension phase 1 of the study (approximately 25 months) and during extension phase 2(approximately 80 months) were reported. After approx 25 and 80 months
Secondary Number of Bleeds Using Pain Medication The mean number of bleeds using pain medication in the main phase of the study (approximately 19 months) were reported. After approx 19 months
Secondary Number of Adverse Events Reported During the Trial Period: After Approximately 19 Months All presented adverse events (AEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. After approx 19 months
Secondary Number of Adverse Events Reported During the Trial Period: After Approximately 25 Months All presented adverse events (AEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. After approx. 25 months
Secondary Number of Adverse Events Reported During the Trial Period: After Approximately 80 Months The number of adverse events observed during the study after approximately 80 months was reported. After approximately 80 months
Secondary Number of Serious Adverse Events Reported During the Trial Period: After Approximately 19 Months All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. After approximately 19 months
Secondary Number of Serious Adverse Events Reported During the Trial Period: After Approximately 25 Months All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. After approximately 25 months
Secondary Number of Serious Adverse Events Reported During the Trial Period: After Approximately 80 Months All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. After approximately 80 months
Secondary Change in Blood Pressure: After Approximately 19 Months The mean change in the systolic and diastolic blood pressure values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). After approximately 19 months
Secondary Change in Blood Pressure: After Approximately 25 Months The mean change in the systolic and diastolic blood pressure values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19). After approximately 19 and 25 months
Secondary Change in Blood Pressure: After Approximately 80 Months Change in the blood pressure was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa. After approximately 80 months
Secondary Change in Pulse: After Approximately 19 Months The mean change in the pulse values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). After approximately 19 months
Secondary Change in Pulse: After Approximately 25 Months The mean change in the pulse values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19). After approximately 25 months
Secondary Change in Pulse: After Approximately 80 Months Change in pulse was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa. After approximately 80 months
Secondary Change in Body Temperature: After Approximately 19 Months The mean change in the body temperature values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). After approximately 19 months
Secondary Change in Body Temperature: After Approximately 25 Months The mean change in the body temperature (C) values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19). After approximately 25 months
Secondary Change in Body Temperature: After Approximately 80 Months Change in body temperature was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa. After approximately 80 months
Secondary Change in Respiratory Rate: After Approximately 19 Months The mean change in the respiratory rate values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). After approximately 19 months
Secondary Change in Respiratory Rate: After Approximately 25 Months The mean change in the respiratory rate (breaths/min) values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19). After approximately 25 months
Secondary Change in Respiratory Rate: After Approximately 80 Months Change in respiratory rate was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa. After approximately 80 months
Secondary FVIII Activity 30 Min Post -Injection (C30min) FVIII plasma activity was measured after 30 mins of injection. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator Week 0, week 28
Secondary Incremental Recovery (Single Dose and Steady State) 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 (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. Week 0, week 28
Secondary Trough Level (Single Dose and Steady State) Trough level was defined as the plasma FVIII activity recorded immediately before next dose is given. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. Week 0, week 28
Secondary Area Under the Curve (AUC0-inf) Area under the plasma activity versus time profile from time zero to infinity (AUC0-inf) was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. It is the measure of total plasma exposure. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28
Secondary Area Under the Curve (AUC0-t) Area under the plasma activity versus time profile from time zero to the last measurable activity (AUC0-t) was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28
Secondary Terminal Half Life (t1/2) t½ = ln(2) / ?z, where ?z 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 (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28
Secondary Clearance (CL) Total plasma clearance (CL) of drug after intravenous administration was reported. Clearance was calculated using the formula CL= Dose / AUC(0-inf). This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28
Secondary Mean Residence Time (MRT) MRT = AUMC/AUC(0-inf), where AUMC is the area under the first moment curve, i.e. the area under the curve t·C(t), calculated with the same method as AUC(0-inf) (linear trapezoidal method + extrapolated area). This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28
Secondary Volume of Distribution at Steady State (Vss) Apparent volume of distribution at steady state is a product of the mean residence time and clearance and was calculated using the formula - Vss = CL x MRT. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28
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