Trial Outcomes & Findings for ADYNOVATE Drug Use-Results Survey (NCT NCT03169972)
NCT ID: NCT03169972
Last Updated: 2025-02-04
Results Overview
Number of previously treated patients (PTPs) and previously untreated patients (PUPs) who discontinued the use of ADYNOVATE were reported.
Recruitment status
COMPLETED
Target enrollment
135 participants
Primary outcome timeframe
Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs
Results posted on
2025-02-04
Participant Flow
Participants took part in the survey at 66 investigative sites in Japan, from 1 February 2017 to 15 September 2023.
Participants with a historical diagnosis of hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) were enrolled. This included previously treated patients with Factor VIII deficiency (PTPs) and previously untreated patients with Factor VIII deficiency (PUPs) who were treated with ADYNOVATE. Participants received ADYNOVATE intravenous Infusion as part of a routine medical care.
Participant milestones
| Measure |
Previously Treated Patients (PTPs)
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Overall Study
STARTED
|
123
|
12
|
|
Overall Study
COMPLETED
|
123
|
12
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
ADYNOVATE Drug Use-Results Survey
Baseline characteristics by cohort
| Measure |
Previously Treated Patients (PTPs)
n=123 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=12 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
Total
n=135 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Customized
< 12 years
|
29 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
35 Participants
n=206 Participants
|
|
Age, Customized
>= 12 years, < 18 years
|
4 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Age, Customized
>= 18 years, < 65 years
|
77 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
80 Participants
n=206 Participants
|
|
Age, Customized
65 years or more
|
7 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Age, Customized
Unknown
|
6 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
123 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
134 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
123 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
135 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
123 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
135 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
Japan
|
123 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
135 Participants
n=206 Participants
|
|
Healthcare Category
Inpatient
|
4 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Healthcare Category
Outpatient
|
119 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
126 Participants
n=206 Participants
|
|
Experience of Serious Bleeding
Had No Experience of Serious Bleeding
|
98 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
110 Participants
n=206 Participants
|
|
Experience of Serious Bleeding
Had Experience of Serious Bleeding
|
19 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
19 Participants
n=206 Participants
|
|
Experience of Serious Bleeding
Unknown
|
6 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Experience of Surgery
Had No Experience of Surgery
|
91 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
100 Participants
n=206 Participants
|
|
Experience of Surgery
Had Experience of Surgery
|
27 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
29 Participants
n=206 Participants
|
|
Experience of Surgery
Unknown
|
5 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Medical History
Had No Medical History
|
86 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
96 Participants
n=206 Participants
|
|
Medical History
Had Medical History
|
33 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
35 Participants
n=206 Participants
|
|
Medical History
Unknown
|
4 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Medical Complications
Had No Medical Complications
|
90 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
98 Participants
n=206 Participants
|
|
Medical Complications
Had Medical Complications
|
32 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
35 Participants
n=206 Participants
|
|
Medical Complications
Unknown
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Hemophilic Arthropathy
Had No Hemophilic Arthropathy
|
61 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
69 Participants
n=206 Participants
|
|
Hemophilic Arthropathy
Had Hemophilic Arthropathy
|
60 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
63 Participants
n=206 Participants
|
|
Hemophilic Arthropathy
Unknown
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Target Joint of Hemophilic Arthropathy
Had No Target Joint of Hemophilic Arthropathy
|
100 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
110 Participants
n=206 Participants
|
|
Target Joint of Hemophilic Arthropathy
Had Target Joint of Hemophilic Arthropathy
|
17 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
17 Participants
n=206 Participants
|
|
Target Joint of Hemophilic Arthropathy
Unknown
|
6 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Hepatic Impairment
Had No Hepatic Impairment
|
114 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
126 Participants
n=206 Participants
|
|
Hepatic Impairment
Had Hepatic Impairment
|
8 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Hepatic Impairment
Unknown
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Renal Impairment
Had No Renal Impairment
|
120 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
132 Participants
n=206 Participants
|
|
Renal Impairment
Had Renal Impairment
|
2 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Renal Impairment
Unknown
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Allergy
Had No Allergy
|
111 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
122 Participants
n=206 Participants
|
|
Allergy
Had Allergy
|
7 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Allergy
Unknown
|
5 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Congenital Hemophilia A
|
123 Participants
n=99 Participants
|
12 Participants
n=107 Participants
|
135 Participants
n=206 Participants
|
|
Age of Diagnosis of Hemophilia A
< 1 year old
|
38 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
41 Participants
n=206 Participants
|
|
Age of Diagnosis of Hemophilia A
>= 1, < 12 years old
|
28 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
31 Participants
n=206 Participants
|
|
Age of Diagnosis of Hemophilia A
<=18 years
|
3 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Age of Diagnosis of Hemophilia A
>= 18, < 65 years old
|
4 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
|
Age of Diagnosis of Hemophilia A
>= 65 years old
|
1 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
|
Age of Diagnosis of Hemophilia A
Unknown
|
49 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
50 Participants
n=206 Participants
|
|
Times of Bleeding in One Year
< 1 time per a year
|
29 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
33 Participants
n=206 Participants
|
|
Times of Bleeding in One Year
>= 1, < 10 times per a year
|
44 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
44 Participants
n=206 Participants
|
|
Times of Bleeding in One Year
>= 10 times per a year
|
14 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
14 Participants
n=206 Participants
|
|
Times of Bleeding in One Year
Unknown
|
36 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
44 Participants
n=206 Participants
|
|
Severity of Hemophilia A
Severe
|
93 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
101 Participants
n=206 Participants
|
|
Severity of Hemophilia A
Moderate
|
16 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
18 Participants
n=206 Participants
|
|
Severity of Hemophilia A
Mild
|
7 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Severity of Hemophilia A
Unknown
|
7 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
|
Dosing Regimen before the Start of Administration of ADYNOVATE
Prophylaxis Therapy
|
78 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
78 Participants
n=206 Participants
|
|
Dosing Regimen before the Start of Administration of ADYNOVATE
On-demand Replacement Therapy
|
21 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
25 Participants
n=206 Participants
|
|
Dosing Regimen before the Start of Administration of ADYNOVATE
Prophylaxis and On-demand Replacement Therapy
|
22 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
22 Participants
n=206 Participants
|
|
Dosing Regimen before the Start of Administration of ADYNOVATE
The Other Therapy
|
2 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
3 Participants
n=206 Participants
|
|
Dosing Regimen before the Start of Administration of ADYNOVATE
Had Not Treated
|
0 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
|
Factor VIII Inhibitor Development
Had No Factor VIII Inhibitor Development
|
97 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
104 Participants
n=206 Participants
|
|
Factor VIII Inhibitor Development
Had Factor VIII Inhibitor Development
|
7 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
|
Factor VIII Inhibitor Development
Unknown
|
19 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
24 Participants
n=206 Participants
|
|
Family History of Hemophilia
Had No Family History of Hemophilia
|
52 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
56 Participants
n=206 Participants
|
|
Family History of Hemophilia
Had Family History of Hemophilia
|
46 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
51 Participants
n=206 Participants
|
|
Family History of Hemophilia
Unknown
|
25 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
28 Participants
n=206 Participants
|
|
Family History of Inhibitor Development
Had No Family History of Inhibitor Development
|
85 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
90 Participants
n=206 Participants
|
|
Family History of Inhibitor Development
Had Family History of Inhibitor Development
|
6 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Family History of Inhibitor Development
Unknown
|
32 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
39 Participants
n=206 Participants
|
|
Status of Concomitant Drugs
|
34 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
41 Participants
n=206 Participants
|
|
Status of Concomitant Therapies
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
Number of previously treated patients (PTPs) and previously untreated patients (PUPs) who discontinued the use of ADYNOVATE were reported.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=123 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=12 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Number of Participants Who Discontinued the Use of Study Drug
|
10 Participants
|
5 Participants
|
PRIMARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen and experienced spontaneous bleeding episodes during the administration period.
Annual bleed rate (ABR) of spontaneous bleeding episodes in PTPs and PUPs on a prophylaxis regimen were reported. Annual bleed rate is calculated by the number of bleeding episodes observed during administration period divided by the duration of administration period, after that multiplied with 365.2425.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=24 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Annual Bleed Rate (ABR) of Spontaneous Bleeding Episodes on a Prophylaxis Regimen
|
1.99 Bleeds per year
Interval 1.0 to 3.59
|
—
|
PRIMARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen and experienced breakthrough bleeding episodes during the administration period.
Annual bleed rate (ABR) of breakthrough bleeding episodes in PTPs and PUPs on a prophylaxis regimen were reported. Annual bleed rate is calculated by the number of bleeding episodes observed during administration period divided by the duration of administration period, after that multiplied with 365.2425.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=31 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=1 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Annual Bleed Rate (ABR) of Breakthrough Bleeding Episodes on a Prophylaxis Regimen
|
3.16 Bleeds per year
Interval 1.11 to 5.1
|
2.91 Bleeds per year
Interval 2.91 to 2.91
|
PRIMARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPs.Population: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen during the administration period.
Duration of treatment of study drug on a prophylaxis regimen was reported.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=110 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=7 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Duration of Treatment of Study Drug on a Prophylaxis Regimen
|
343.0 days
Standard Deviation 69.33
|
510.7 days
Standard Deviation 256.04
|
PRIMARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with an on-demand regimen during the administration period.
Duration of treatment of study drug on an on-demand regimen was reported.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=14 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=3 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Duration of Treatment of Study Drug an On-Demand Regimen
|
13.3 days
Standard Deviation 22.42
|
5.0 days
Standard Deviation 2.65
|
PRIMARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen during the administration period.
Dose per administration of study drug on a prophylaxis regimen was reported.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=110 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=7 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Dose Per Administration of Study Drug on a Prophylaxis Regimen
|
39.4 IU per kilograms (kg)
Standard Deviation 12.69
|
45.8 IU per kilograms (kg)
Standard Deviation 7.70
|
PRIMARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with an on-demand regimen during the administration period.
Dose per administration of study drug on an on-demand regimen was reported.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=14 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=3 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Dose Per Administration of Study Drug an On-Demand Regimen
|
36.8 IU per kilograms (kg)
Standard Deviation 13.88
|
29.7 IU per kilograms (kg)
Standard Deviation 12.42
|
PRIMARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation. The number analyzed was the number of participants with data available for analysis who treated with an on-demand regimen during the administration period.
Number of doses per a bleeding episode of study drug on an on-demand regimen was reported.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=58 Count of Bleeding Episodes
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=3 Count of Bleeding Episodes
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Number of Doses Per a Bleeding Episode of Study Drug an On-Demand Regimen
|
6.0 Number of doses per a bleeding episode
Standard Deviation 4.45
|
5.0 Number of doses per a bleeding episode
Standard Deviation 4.36
|
PRIMARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Efficacy analysis set: all participants who received at least one dose of protocol treatment without major protocol deviation. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen and experienced breakthrough bleeding episodes during the administration period.
Percentage of each category of hemostatic effectiveness for treatment of breakthrough bleeding episodes in a prophylaxis regimen assessed by the investigator was reported. Hemostatic effectiveness was assessed by the investigator with following 4-point ordinal scale: Excellent, Good, Fair, Poor.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=86 Count of Bleeding Episodes
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=5 Count of Bleeding Episodes
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen
Poor
|
0.00 Percentage of bleeding episodes
|
20.00 Percentage of bleeding episodes
|
|
Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen
Fair
|
0.00 Percentage of bleeding episodes
|
0.00 Percentage of bleeding episodes
|
|
Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen
Excellent
|
37.21 Percentage of bleeding episodes
|
20.00 Percentage of bleeding episodes
|
|
Hemostatic Effectiveness of Study Drug on Treatment of Breakthrough Bleeding Episodes With a Prophylaxis Regimen
Good
|
62.79 Percentage of bleeding episodes
|
40.00 Percentage of bleeding episodes
|
PRIMARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with on-demand regimen during the administration period.
Percentage of each category of hemostatic effectiveness for an on-demand regimen assessed by the investigator was reported. Hemostatic effectiveness was assessed by the investigator with following 4-point ordinal scale: Excellent, Good, Fair, Poor.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=58 Count of Bleeding Episodes
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=3 Count of Bleeding Episodes
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Hemostatic Effectiveness of Study Drug on an On-Demand Regimen
Excellent
|
32.76 Percentage of bleeding episodes
|
33.33 Percentage of bleeding episodes
|
|
Hemostatic Effectiveness of Study Drug on an On-Demand Regimen
Good
|
60.34 Percentage of bleeding episodes
|
33.33 Percentage of bleeding episodes
|
|
Hemostatic Effectiveness of Study Drug on an On-Demand Regimen
Fair
|
6.90 Percentage of bleeding episodes
|
0.00 Percentage of bleeding episodes
|
|
Hemostatic Effectiveness of Study Drug on an On-Demand Regimen
Poor
|
0.00 Percentage of bleeding episodes
|
33.33 Percentage of bleeding episodes
|
SECONDARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey. The number analyzed was the number of participants with data available for analysis who treated with a prophylaxis regimen during the administration period.
Number of doses per a week of study drug on a prophylaxis regimen was reported.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=110 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=7 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Number of Doses Per a Week of Study Drug on a Prophylaxis Regimen
|
2.0 Number of doses per a week
Standard Deviation 0.38
|
1.8 Number of doses per a week
Standard Deviation 0.41
|
SECONDARY outcome
Timeframe: Throughout the study participation period: 1 year for previously treated patients for PTPs and 2 years for previously untreated patients for PUPsPopulation: Safety Analysis Set, The safety analysis set was defined as all participants who completed the survey.
Number of PTPs and PUPs who experienced factor VIII inhibition, dermatitis atopic or eczema as an AE related to development of inhibitors, shock or anaphylaxis was reported.
Outcome measures
| Measure |
Previously Treated Patients (PTPs)
n=123 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=12 Participants
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE)
Factor VIII Inhibition
|
1 Participants
|
3 Participants
|
|
Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE)
Dermatitis Atopic
|
0 Participants
|
1 Participants
|
|
Number of Participants Who Experience Factor VIII Inhibition, Dermatitis Atopic or Eczema as an Adverse Event (AE)
Eczema
|
0 Participants
|
1 Participants
|
Adverse Events
Previously Treated Patients (PTPs)
Serious events: 2 serious events
Other events: 1 other events
Deaths: 0 deaths
Previously Untreated Patients (PUPs)
Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Previously Treated Patients (PTPs)
n=123 participants at risk
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=12 participants at risk
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Hepatic cancer
|
0.81%
1/123 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
0.00%
0/12 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
|
Blood and lymphatic system disorders
Factor VIII inhibition
|
0.00%
0/123 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
8.3%
1/12 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
|
Nervous system disorders
Thrombotic cerebral infarction
|
0.81%
1/123 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
0.00%
0/12 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
Other adverse events
| Measure |
Previously Treated Patients (PTPs)
n=123 participants at risk
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously treated patients with Factor VIII deficiency (PTPs) who had 4 or more exposure days to other Factor VIII (FVIII) products.
|
Previously Untreated Patients (PUPs)
n=12 participants at risk
Participants with hemophilia A who received Recombinant Factor VIII (FVIII) PEGylated (ADYNOVATE) intravenous Infusion as part of a routine medical care. Participants in this group were previously untreated patients with Factor VIII deficiency (PUPs) who had 3 or less previous exposure days to other Factor VIII (FVIII) products.
|
|---|---|---|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/123 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
8.3%
1/12 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
|
Blood and lymphatic system disorders
Factor VIII inhibition
|
0.81%
1/123 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
16.7%
2/12 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
|
Skin and subcutaneous tissue disorders
Dermatitis atopic
|
0.00%
0/123 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
8.3%
1/12 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
0.00%
0/123 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
8.3%
1/12 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
|
Musculoskeletal and connective tissue disorders
Haemarthrosis
|
0.00%
0/123 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
8.3%
1/12 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
|
Injury, poisoning and procedural complications
Subcutaneous haematoma
|
0.00%
0/123 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
8.3%
1/12 • Up to 1 year for previously treated patients (PTPs) or up to 2 years for previously untreated patients (PUPs)
At each visit the investigator had to document any occurrence of AEs and abnormal laboratory findings. Any event spontaneously reported by the participant or observed by the investigator was recorded, irrespective of the relation to the survey treatment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place