Clinical Trial Results:
A Phase 3, Prospective, Multicenter, Open-label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (ADYNOVATE) Administered for Prophylaxis and Treatment of Bleeding in Chinese Previously Treated Patients with Severe Hemophilia A (FVIII <1%).
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Summary
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EudraCT number |
2023-000502-26 |
Trial protocol |
Outside EU/EEA |
Global end of trial date |
05 Sep 2024
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Results information
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Results version number |
v1(current) |
This version publication date |
21 Mar 2025
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First version publication date |
21 Mar 2025
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Other versions |
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Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
TAK-660-3001
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT05707351 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Takeda
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Sponsor organisation address |
95 Hayden Ave, Lexington, MA, United States, 02421
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Public contact |
Study Director, Takeda, TrialDisclosures@takeda.com
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Scientific contact |
Study Director, Takeda, TrialDisclosures@takeda.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
Yes
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
05 Sep 2024
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Is this the analysis of the primary completion data? |
No
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Global end of trial reached? |
Yes
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Global end of trial date |
05 Sep 2024
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
The main purpose of this study was to evaluate the safety, efficacy, and pharmacokinetics of adynovate in chinese participants with severe Hemophilia A.
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Protection of trial subjects |
Each participant signed an informed consent form (ICF) before participating in the study. For participants <18 years old, participants gave assent and their parents/legally authorized representative signed the ICF accordingly.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
27 Mar 2023
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
China: 37
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Worldwide total number of subjects |
37
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EEA total number of subjects |
0
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
10
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Adults (18-64 years) |
27
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From 65 to 84 years |
0
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85 years and over |
0
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Recruitment
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Recruitment details |
Participants took part in the study at various investigative sites in China from 27 March 2023 to 05 September 2024. | ||||||||||||
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Pre-assignment
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Screening details |
Participants with a diagnosis of severe hemophilia A were enrolled in this study to receive Adynovate (45 [±5] international units per kilogram [IU/kg]), infusion, intravenously (IV). | ||||||||||||
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Period 1
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Period 1 title |
Overall Study (overall period)
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Is this the baseline period? |
Yes | ||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||
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Arms
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Arm title
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Adynovate | ||||||||||||
Arm description |
Participants received prophylactic treatment with Adynovate (45 [±5] IU/kg), infusion, IV, twice weekly, for at least 50 EDs or approximately 28 weeks. | ||||||||||||
Arm type |
Experimental | ||||||||||||
Investigational medicinal product name |
Adynovate
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Investigational medicinal product code |
TAK-660
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Other name |
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Pharmaceutical forms |
Concentrate for solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
Adynovate 45±5 IU/kg, twice-weekly, for at least 50 exposure days (EDs), or approximately 28 weeks.
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Baseline characteristics reporting groups
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Reporting group title |
Adynovate
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Reporting group description |
Participants received prophylactic treatment with Adynovate (45 [±5] IU/kg), infusion, IV, twice weekly, for at least 50 EDs or approximately 28 weeks. | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Adynovate
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Reporting group description |
Participants received prophylactic treatment with Adynovate (45 [±5] IU/kg), infusion, IV, twice weekly, for at least 50 EDs or approximately 28 weeks. | ||
Subject analysis set title |
Adynovate
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Subject analysis set type |
Full analysis | ||
Subject analysis set description |
Participants received prophylactic treatment with Adynovate (45 [±5] IU/kg), infusion, IV, twice weekly, for at least 50 EDs, or approximately 28 weeks.
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End point title |
Total Annualized Bleeding Rates (ABR) [1] | ||||||||
End point description |
Total ABR was defined as the number of treated and non-treated bleeding episodes (BEs) that occurred during the treatment period, calculated as, ABR= number of unique bleeds during treatment period/(length of treatment period [days]/365.25). Total ABR for all BEs, spontaneous or traumatic, recorded in the participant's electronic diary and/or recorded in the physician/nurse/study site notes were reported. The FAS included all participants who were assigned to receive a treatment regimen of Adynovate.
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End point type |
Primary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Only descriptive analysis was planned for this endpoint. |
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| No statistical analyses for this end point | |||||||||
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End point title |
ABR Based on Bleeding Site | ||||||||||||
End point description |
ABR= number of unique bleeds during treatment period/(length of treatment period [days]/365.25). ABR for BEs based on bleeding site: joint or non-joint, recorded in the participant's electronic diary and/or recorded in the physician/nurse/study site notes were reported. The FAS included all participants who were assigned to receive a treatment regimen of Adynovate.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Number of Adynovate Infusions per Week During the Prophylactic Treatment Period | ||||||||
End point description |
The FAS included all participants who were assigned to receive a treatment regimen of Adynovate.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||
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End point title |
ABR Based on Bleeding Cause | ||||||||||||
End point description |
ABR= number of unique bleeds during treatment period/(length of treatment period [days]/365.25). ABR for BEs based on bleeding cause: spontaneous/unknown or injury, recorded in the participant's electronic diary and/or recorded in the physician/nurse/study site notes were reported. The FAS included all participants who were assigned to receive a treatment regimen of Adynovate.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Number of Adynovate Infusions per Month During the Prophylactic Treatment Period | ||||||||
End point description |
The FAS included all participants who were assigned to receive a treatment regimen of Adynovate.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||
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End point title |
Weight-adjusted Consumption of Adynovate per Week During the Prophylactic Treatment Period | ||||||||
End point description |
Weight-adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. The FAS included all participants who were assigned to receive a treatment regimen of Adynovate.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||
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End point title |
Weight-adjusted Consumption of Adynovate per Month During the Prophylactic Treatment Period | ||||||||
End point description |
Weight-adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. The SAS included all participants treated with at least 1 Adynovate dose. Subjects analysed is the number of participants with treated bleeding episodes.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of Bleeding Events in Each Category of Hemostatic Efficacy Rating at Resolution of Breakthrough Bleeding Episode | ||||||||||||||||||
End point description |
Hemostatic efficacy for treatment of BEs was rated on 4-point Likert scale as: excellent=full relief of pain & cessation of objective signs of bleeding after single infusion, no additional infusion is required for control of bleeding & administration of further infusion to maintain hemostasis would not affect scoring; good=definite pain relief and/or improvement in signs of bleeding after single infusion, possibly requires more than 2 infusions for complete resolution & administration of further infusion to maintain hemostasis would not affect scoring; fair=probable and/or slight relief of pain & slight improvement in signs of bleeding after single infusion, required multiple infusions for complete resolution; none=no improvement of signs/symptoms/conditions worsen. Missing indicates number of unique bleeding episodes without any overall hemostatic efficacy rating at resolution of breakthrough bleeding episode. FAS included all participants treated with at least 1 Adynovate dose.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Average Time Interval Between Bleeding Episodes (BEs) | ||||||||
End point description |
Average time interval between bleeding episodes (days)= Length of treatment period (days)/ Number of unique bleeds during treatment period. Average time interval was computed for participants with more than 1 unique BEs. The FAS included all participants who were assigned to receive a treatment regimen of Adynovate. Subjects analysed is the number of participants with more than 1 unique BEs.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||
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End point title |
Percentage of Participants With Zero Bleeding Episodes During the Study | ||||||||
End point description |
Percentages were rounded off to the nearest single decimal place. The FAS included all participants who were assigned to receive a treatment regimen of Adynovate.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||
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End point title |
Weight-adjusted Consumption of Adynovate per Bleeding Episode | ||||||||
End point description |
Weight-adjusted consumption (IU/kg) was derived as the total units infused (IU) divided by the last available body weight (kg) prior to the infusion. The SAS included all participants treated with at least 1 Adynovate dose. Subjects analysed is the number of participants with treated bleeding episodes.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of Minor Surgeries With Hemostatic Efficacy Based on Global Hemostatic Efficacy Assessment (GHEA) Score as Assessed by the Operating Surgeon/Investigator | ||||||||
End point description |
GHEA score consisted of 3 individual rating scales: (1) Intra-operative Efficacy Assessment Scale, (2) Post-operative Efficacy Assessment Scale, and (3) Peri-operative Efficacy Assessment Scale. Each rating scale is based on 4 points scale ranging from: 3 (Excellent), 2 (Good), 1 (Fair), and 0 (None). The scores of 3 individual ratings scales were added together to form a GHEA score. Total score ranged from 0 to 9, where scores evaluate as: excellent (7 to 9), good (5 to 7), fair (3 to 4), and none (0 to 2). For a GHEA score of 7 to be rated “excellent” no individual assessment scores could be less than (<) 2 and at least 1 assessment score had to be equal to (=) 3; otherwise a score of 7 was rated “good”. The FAS included all participants treated with at least 1 Adynovate dose. Only participants with hemostatic efficacy were to be assessed for this endpoint.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| Notes [2] - Subjects analysed is zero as no participant reported blood loss for minor surgeries. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Volume of Actual and Predicted Intra-operative and Post-operative Blood Loss After the Surgery as Assessed by the Operating Surgeon/Investigator | ||||||||
End point description |
The FAS included all participants treated with at least 1 Adynovate dose. Only subjects with blood loss for minor surgeries were to be assessed for this endpoint.
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End point type |
Secondary
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End point timeframe |
Post-operative: Day 1 and at discharge Week 26
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| Notes [3] - Subjects analysed is zero as no participant reported blood loss for minor surgeries. |
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of Adynovate Infusions per Bleeding Episode | ||||||||
End point description |
The Safety Analysis Set (SAS) included all subjects treated with at least 1 Adynovate dose. Subjects analysed is the number of participants with treated bleeding episodes.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||||
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End point title |
Number of Participants who Required Perioperative Transfusion of Blood, Red blood Cells, Platelets, and Other Blood Products | ||||||
End point description |
The FAS included all participants treated with at least 1 Adynovate dose.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| No statistical analyses for this end point | |||||||
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End point title |
FVIII Activity Level in Plasma Assessed by a 1-stage Clotting Assay | ||||||||||||||||||||||||||||||||||||||||||||||||||||
End point description |
As per planned analysis, data for this outcome measure was collected and reported for initial pharmacokinetic (PK) assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit. FVIII activity level reported was corrected for pre-infusion measurement. The Pharmacokinetic Full Analysis Set (PK FAS) included all participants who consented to PK evaluation, were treated with at least 1 Adynovate dose, and had at least 1 evaluable PK concentration post dose. 'n' denotes the number of participants with data available for analysis at the specified time-point.
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End point type |
Secondary
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End point timeframe |
Day -1 and Week 20: pre-infusion, post-infusion at multiple time-points up to 96 hours
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| No statistical analyses for this end point | |||||||||||||||||||||||||||||||||||||||||||||||||||||
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End point title |
Number of Participants With Confirmed Inhibitory Antibodies to Factor VIII (FVIII), Binding Immunoglobulin G (IgG) and Immunoglobulin M (IgM) Antibodies to Adynovate and Chinese Hamster Ovary (CHO) Protein | ||||||||||||||
End point description |
The SAS included all participants treated with at least 1 Adynovate dose.
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End point type |
Secondary
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End point timeframe |
Up to approximately 28 weeks
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| No statistical analyses for this end point | |||||||||||||||
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End point title |
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Treatment-emergent Adverse Events (Serious TEAEs) | ||||||||||
End point description |
Adverse event(AE): any untoward medical occurrence in participant administered pharmaceutical product; untoward medical occurrence does not necessarily have causal relationship with this treatment. AE can therefore be any unfavorable & unintended sign (including an abnormal laboratory finding), symptom/disease temporally associated with use of medicinal (investigational) product whether or not it is related to medicinal product. TEAE: any AE either reported for first time or worsening of pre-existing event after first dose of study drug & within 30 days of last administration of study drug. Serious TEAEs: any untoward medical occurrence that: results in death, is life-threatening, requires inpatient hospitalization/prolongation of existing hospitalization, results in persistent or significant disability/incapacity, leads to congenital anomaly/birth defect in offspring of participant or is medically important. The SAS included all participants treated with at least 1 Adynovate dose.
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End point type |
Secondary
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End point timeframe |
Up to approximately 28 weeks
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| No statistical analyses for this end point | |||||||||||
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End point title |
Daily Intra-Operative and Post-Operative Weight-Adjusted Consumption Dose of Adynovate | ||||||
End point description |
The FAS included all participants treated with at least 1 Adynovate dose. Only participants who were administered Adynovate for minor surgeries were to be assessed for this endpoint.
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End point type |
Secondary
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End point timeframe |
Baseline through study completion or ≥50 EDs whichever occurred last (approximately 28 weeks)
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| Notes [4] - Subjects analysed is zero as no participants were administered Adynovate for minor surgeries. |
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| No statistical analyses for this end point | |||||||
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End point title |
Incremental Recovery Over Time During Adynovate Prophylactic Treatment | ||||||||||||||
End point description |
Incremental recovery (IR) was calculated as IR (international units per deciliter)/(international units per kilogram [(IU/dL)/(IU/kg)] = [PostFVIII (IU/dL)-PreFVIII (IU/dL)]/Weight Adjusted Dose (IU/kg). The SAS included all participants treated with at least 1 Adynovate dose. Subjects analysed is the number of participants with data available for analyses. 'n' denotes the number of participants with data available for analysis at the specified time-point.
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End point type |
Secondary
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End point timeframe |
Baseline, Week 6, and Study Completion (approximately Week 28)
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| No statistical analyses for this end point | |||||||||||||||
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End point title |
Pre-dose Level of FVIII Activity in Plasma | ||||||||||||||||||
End point description |
The SAS included all participants treated with at least 1 Adynovate dose. Subjects analysed is the number of participants with data available for analyses. 'n' denotes the number of participants with data available for analysis at the specified time-point.
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End point type |
Secondary
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End point timeframe |
Baseline, Weeks 2, 6, 12, and Study Completion (approximately Week 28): Within 30 minutes pre-infusion
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| No statistical analyses for this end point | |||||||||||||||||||
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End point title |
Pre-dose Level of FVIII Antigen in Plasma | ||||||||||||||||||||
End point description |
IU/mL stands for international units per milliliter. The SAS included all participants treated with at least 1 Adynovate dose. 'n' denotes the number of participants with data available for analysis at the specified time-point.
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End point type |
Secondary
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End point timeframe |
Baseline, Weeks 2, 6, 12, and Study Completion (approximately Week 28): Within 30 minutes pre-infusion
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| No statistical analyses for this end point | |||||||||||||||||||||
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End point title |
Pre-dose Level of Von Willebrand Factor (VWF) Antigen in Plasma | ||||||||||||||||||||
End point description |
The SAS included all participants treated with at least 1 Adynovate dose. 'n' denotes the number of participants with data available for analysis at the specified time-point.
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End point type |
Secondary
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End point timeframe |
Baseline, Weeks 2, 6, 12, and Study Completion (approximately Week 28): Within 30 minutes pre-infusion
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| No statistical analyses for this end point | |||||||||||||||||||||
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End point title |
Clearance (CL) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate | ||||||||||||
End point description |
Clearance reported was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit. [(dL/h)/kg] stands for deciliters per hour per kilogram. The PK FAS included all participants who consented to PK evaluation, were treated with at least 1 Adynovate dose, and had at least 1 evaluable PK concentration post dose. 'n' denotes the number of participants with data available for analysis at the specified time-point.
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End point type |
Secondary
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End point timeframe |
Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
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| No statistical analyses for this end point | |||||||||||||
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End point title |
Volume of Distribution for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate | ||||||||||||
End point description |
Volume of distribution was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit.'n' denotes the number of participants with data available for analysis at the specified time-point. The PK FAS included all participants who consented to PK evaluation, were treated with at least 1 Adynovate dose, and had at least 1 evaluable PK concentration post dose. 'n' denotes the number of participants with data available for analysis at the specified time-point.
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End point type |
Secondary
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End point timeframe |
Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
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| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||
End point title |
Area Under the Concentration Versus Time Curve From 0 to 96 Hours (AUC0-96) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate | ||||||||||||
End point description |
AUC0-96 was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit. h*IU/dL stands for hour*international units per deciliter. The PK FAS included all participants who consented to PK evaluation, were treated with at least 1 Adynovate dose, and had at least 1 evaluable PK concentration post dose. 'n' denotes the number of participants with data available for analysis at the specified time-point.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
|
||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||
End point title |
Maximum Concentration (Cmax) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate | ||||||||||||
End point description |
Cmax was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit. The PK FAS included all participants who consented to PK evaluation, were treated with at least 1 Adynovate dose, and had at least 1 evaluable PK concentration post dose. 'n' denotes the number of participants with data available for analysis at the specified time-point.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
|
||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||
End point title |
Pre-dose Concentration (Cpredose) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate | ||||||||||||
End point description |
Cpredose was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit. The PK FAS included all participants who consented to PK evaluation, were treated with at least 1 Adynovate dose, and had at least 1 evaluable PK concentration post dose. 'n' denotes the number of participants with data available for analysis at the specified time-point.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
|
||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||
End point title |
Terminal Phase Elimination Half-life (T1/2) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate | ||||||||||||
End point description |
T1/2 was calculated based on pre-infusion corrected concentration data. As per planned analysis, data for this outcome measure was collected and reported for initial PK assessment and second PK assessment. The initial PK assessment was performed prior to the baseline visit at Day -1. The second PK assessment was performed during the Week 20 visit. The PK FAS included all participants who consented to PK evaluation, were treated with at least 1 Adynovate dose, and had at least 1 evaluable PK concentration post dose. 'n' denotes the number of participants with data available for analysis at the specified time-point.
|
||||||||||||
End point type |
Secondary
|
||||||||||||
End point timeframe |
Day -1 and Week 20: pre-infusion, post-infusion at multiple timepoints up to 96 hours
|
||||||||||||
|
|||||||||||||
| No statistical analyses for this end point | |||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Adverse events information
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Timeframe for reporting adverse events |
Up to approximately 28 weeks
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Adverse event reporting additional description |
The SAS included all participants treated with at least 1 Adynovate dose.
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Dictionary used for adverse event reporting
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
27.0
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Adynovate
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
Participants received prophylactic treatment with Adynovate (45 [±5] IU/kg), infusion, IV, twice weekly, for at least 50 EDs or approximately 28 weeks. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
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|
|||
Substantial protocol amendments (globally) |
|||
| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
|||
| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
|||
| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
