A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A (pathfinder8)
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|ClinicalTrials.gov Identifier: NCT03528551|
Recruitment Status : Completed
First Posted : May 18, 2018
Results First Posted : November 26, 2021
Last Update Posted : December 22, 2022
|Condition or disease||Intervention/treatment||Phase|
|Congenital Bleeding Disorder Haemophilia A||Drug: Turoctocog alfa pegol||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||160 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Prophylaxis and Treatment of Bleeds in Previously N8-GP Treated Patients With Severe Haemophilia A|
|Actual Study Start Date :||April 30, 2018|
|Actual Primary Completion Date :||December 3, 2020|
|Actual Study Completion Date :||December 3, 2020|
Experimental: N8-GP, once weekly
All participants will receive turoctocog alfa pegol (N8-GP) once weekly.
Drug: Turoctocog alfa pegol
Turoctocog alfa pegol 75 IU/kg body weight will be administered once weekly as intravenous injections for a duration of 2 years.
Experimental: N8-GP, twice weekly
All participants will receive N8-GP twice weekly.
Drug: Turoctocog alfa pegol
Turoctocog alfa pegol 60 IU/kg body weight (for patients younger than 12 years) and 50 IU/kg body weight (for patients, 12 years or older) will be administered twice weekly as intravenous injections for a duration of 2 years.
Experimental: N8-GP, three times weekly
All participants will receive N8-GP three times weekly.
Drug: Turoctocog alfa pegol
Turoctocog alfa pegol 50 IU/kg body weight will be administered three times weekly as intravenous injections for a duration of 2 years.
- Number of Adverse Events Reported [ Time Frame: Week 0 to week 108 ]An adverse event (AE) was defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. All AEs mentioned here are treatment emergent adverse events (TEAEs). The TEAE is defined as an event reported from date of first trial product administration until end of the treatment visit (week 104) or follow-up visit if relevant (1 month after end of the treatment).
- Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units (BU) [ Time Frame: Week 0 to week 104 ]The Incidence of inhibitors against coagulation factor eight (FVIII) is defined as titre greater than or equal to (≥) 0.6 Bethesda unit. The inhibitor antibodies were measured using a heat modified Nijmegen FVIII Bethesda assay. The number of participants who developed inhibitors against FVIII are reported.
- Number of Bleeding Episodes on Prophylaxis [ Time Frame: Week 0 to week 104 ]Number of bleeding episodes per participant in the prophylaxis regimen was evaluated during 104 weeks.
- Number of Spontaneous Bleeding Episodes on Prophylaxis [ Time Frame: Week 0 to week 104 ]Spontaneous bleeding referred as bleeding episodes that occurred without apparent cause. The number of spontaneous bleeding episodes were evaluated during 104 weeks.
- Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes Assessed as: Excellent, Good, Moderate, or None [ Time Frame: Week 0 to week 104 ]The haemostatic effect after treatment of a bleed with N8-GP was assessed using a 4-point scale: 'excellent', 'good', 'moderate' or 'none'. The evaluation was done as follows: 1. Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. 2. Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after an injection, but possibly requiring more than one injection for complete resolution. 3. Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection; usually requiring more than one injection 4. None: No improvement or worsening of symptoms.
- Mean Number of N8-GP Injections Required Per Bleeding Episode [ Time Frame: Week 0 to week 104 ]The mean number of N8-GP injections required per bleeding episode from start to stop of a bleed for participants was presented from week 0 to week 104.
- Pre-dose FVIII Activity Levels on N8-GP Prophylaxis [ Time Frame: Week 0 to week 104 ]The pre-dose FVIII activity levels were assessed in International units per millilitre (IU/mL) units from week 0 to week 104 to get an estimate of the pre-dose level for N8-GP at steady-state using mixed model.
- Change in Joint Health Status From Start to End of Trial (Based on Haemophilia Joint Health Score) [ Time Frame: Week 0, Week 104 ]Haemophilia Joint Health Score is a validated outcome tool developed for the assessment of joint health in patients with hemophilia. It comprises an evaluation of the elbow, knee and ankle joints with regards to swelling, muscular atrophy, crepitation and range of motion, joint pain, strength, motion and axial alignment. The score range is from 0 to 24 points (a score of 0 indicates no joint damage. Higher the score higher the joint damage). Change from week 0 to end of trial (week 104) in the domain scores was presented.
- Haemostatic Response During Major Surgical Interventions Assessed as: Excellent, Good, Moderate, or None [ Time Frame: Week 0 to week 104 ]The Haemostatic response to N8-GP during major surgical interventions was assessed using a 4-point scale: 'excellent', 'good', 'moderate' or 'none'. The evaluation was done as follows: 1. Excellent: Better than expected/predicted in this type of procedure. 2. Good: As expected in this type of procedure 3. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen 4. None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required. This endpoint was measured from week 0 to week 104.
- Consumption of N8-GP Per Bleed [ Time Frame: Week 0 to week 104 ]The average dose of N8-GP consumed for treatment of bleed was assessed in International units per kilogram per bleed(IU/kg/bleed). This endpoint was evaluated from week 0 to week 104.
- Consumption of N8-GP During Prophylaxis Treatment [ Time Frame: Week 0 to week 104 ]The average dose of N8-GP consumed for prevention of bleed was assessed. This endpoint was evaluated from week 0 to week 104.
- Change From Start Till End of Trial in Treatment Satisfaction (Based on Hemo-SAT Score) [ Time Frame: Week 0, Week 104 ]The treatment satisfaction of a bleed with N8-GP was assessed using HEMO-SAT assessment tool which contains a questionnaire with 6 domains (Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction), with a scale of 0-100. The lower scores reflecting greater treatment satisfaction. In other words, decrease in the score would mean improvement. The summary of change presented was based on individual changes since week 0. Data is presented for total score.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03528551
|Study Director:||Clinical Reporting Anchor and Disclosure 2834||Novo Nordisk A/S|