Trial Outcomes & Findings for Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients (NCT NCT00157157)
NCT ID: NCT00157157
Last Updated: 2021-05-24
Results Overview
Percentage of treated participants who developed factor VIII inhibitors
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
66 participants
Primary outcome timeframe
Assessed during study period which was to be at least 75 exposure days or 3 years (whichever came first)
Results posted on
2021-05-24
Participant Flow
Recruitment was conducted in the U.S., Canada, and Europe at 24 study sites.
Participants screened for maximum 21 days. Study was not randomized; it was an open-label evaluation. Prior to initial infusion, a minimum washout period of 3 days was required. 11 participants who enrolled did not receive any rAHF-PFM infusions (3 withdrew consent, 6 screen failures, 1 non-compliance with screening, 1 pre-existing low hemoglobin)
Participant milestones
| Measure |
Previously Untreated Patients (PUPs)
rAHF-PFM was dosed according to a therapeutic regimen which was determined by the investigator (ie: standard regimen \[25 to 50 IU/kg body weight, 3 to 4 times per week\]; a modified prophylactic regimen \[dose and frequency selected by investigator\] or on-demand treatment \[dose selected by investigator\]). The dosing regimen used to treat bleeding episodes (BEs) was at the discretion of the investigator and in accordance with the institution's standard of care for the type of bleeding episodes diagnosed.
|
|---|---|
|
Overall Study
STARTED
|
55
|
|
Overall Study
COMPLETED
|
44
|
|
Overall Study
NOT COMPLETED
|
11
|
Reasons for withdrawal
| Measure |
Previously Untreated Patients (PUPs)
rAHF-PFM was dosed according to a therapeutic regimen which was determined by the investigator (ie: standard regimen \[25 to 50 IU/kg body weight, 3 to 4 times per week\]; a modified prophylactic regimen \[dose and frequency selected by investigator\] or on-demand treatment \[dose selected by investigator\]). The dosing regimen used to treat bleeding episodes (BEs) was at the discretion of the investigator and in accordance with the institution's standard of care for the type of bleeding episodes diagnosed.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
6
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Enrolled in another study
|
1
|
|
Overall Study
Met exclusion criteria
|
1
|
|
Overall Study
Inclusion not met- baseline FVIII value
|
1
|
Baseline Characteristics
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
Baseline characteristics by cohort
| Measure |
PUPs
n=55 Participants
|
|---|---|
|
Age, Customized
<6 months
|
21 Participants
n=39 Participants
|
|
Age, Customized
6-12 months
|
26 Participants
n=39 Participants
|
|
Age, Customized
≥13 months
|
8 Participants
n=39 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=39 Participants
|
|
Sex: Female, Male
Male
|
55 Participants
n=39 Participants
|
PRIMARY outcome
Timeframe: Assessed during study period which was to be at least 75 exposure days or 3 years (whichever came first)Population: Participants who received at least 1 infusion of rAHF-PFM
Percentage of treated participants who developed factor VIII inhibitors
Outcome measures
| Measure |
PUPs
n=55 Participants
|
PUPs -During On-Demand Treatment
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Factor VIII Inhibitor Development
|
29.1 percentage
Interval 17.1 to 41.1
|
—
|
—
|
SECONDARY outcome
Timeframe: Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first)Population: Participants who received at least 1 infusion of rAHF-PFM for the treatment of bleeding episodes
The number of bleeding episodes treated with 1, 2, 3, or ≥4 infusions of rAHF-PFM to achieve adequate hemostasis
Outcome measures
| Measure |
PUPs
n=44 Participants
|
PUPs -During On-Demand Treatment
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Bleeding Episodes Treated With 1 to ≥4 Infusions
1 infusion
|
356 Bleeding episodes
|
—
|
—
|
|
Bleeding Episodes Treated With 1 to ≥4 Infusions
2 infusions
|
107 Bleeding episodes
|
—
|
—
|
|
Bleeding Episodes Treated With 1 to ≥4 Infusions
3 infusions
|
35 Bleeding episodes
|
—
|
—
|
|
Bleeding Episodes Treated With 1 to ≥4 Infusions
4 or more infusions
|
19 Bleeding episodes
|
—
|
—
|
SECONDARY outcome
Timeframe: Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first)Population: Participants who received at least 1 infusion of rAHF-PFM and had at least 1 treated bleeding episode. The 1 rating of "none" was for the first 2 infusions, the last infusion was rated as "good".
Number of rAHF-PFM-treated bleeding episodes with treater assessment of hemostasis (4-point ordinal scale): Excellent: Full pain relief \& bleeding cessation within \~8 hrs of 1 infusion. Additional infusions may have been given to maintain hemostasis; Good: Definite pain relief and/or improvement in bleeding within \~8 hrs after infusion. Possibly requires \>1 infusion for complete resolution; Fair: Probable or slight relief of pain \& slight improvement in bleeding within \~8 hrs after infusion. Requires \>1 infusion for complete resolution; or None: No improvement or condition worsens.
Outcome measures
| Measure |
PUPs
n=44 Participants
|
PUPs -During On-Demand Treatment
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes
Excellent
|
258 bleeding episodes
|
—
|
—
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes
Good
|
177 bleeding episodes
|
—
|
—
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes
Fair
|
30 bleeding episodes
|
—
|
—
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes
None
|
1 bleeding episodes
|
—
|
—
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes
Unknown/not assessed
|
51 bleeding episodes
|
—
|
—
|
SECONDARY outcome
Timeframe: Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first)Population: Participants treated for at least 3 months with investigator-defined on-demand treatment (for dose) or prophylaxis (for dose and infusion frequency) for \>80% of the treatment period
Number of bleeding episodes per subject annualized over 1 year for all etiologies
Outcome measures
| Measure |
PUPs
n=43 Participants
|
PUPs -During On-Demand Treatment
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Annualized Rate of Bleeding Episodes
|
4.95 bleeding episodes per subject per year
Interval 1.01 to 32.7
|
—
|
—
|
SECONDARY outcome
Timeframe: Reported during study period which was to be at least 75 exposure days or 3 years (whichever came first)Population: Participants treated for at least 3 months with investigator-defined on-demand treatment (for dose) or prophylaxis (for dose and infusion frequency) for \>80% of the treatment period
Weight-Adjusted Weekly Dose for Prophylaxis, On-Demand Treatment, and Perioperative Management. rAHF-PFM dose determined by the investigator (ie: standard regimen \[25-50 IU/kg body weight, 3-4 times per week\]; modified prophylactic regimen \[dose and frequency selected by investigator\] or on-demand treatment \[dose selected by investigator\]). Dosing to treat BEs was at investigator's discretion and in accordance with institution's standard of care. rAHF-PFM was administered I.V. via bolus infusion, except for perioperative management when it was given either by continuous or bolus infusion.
Outcome measures
| Measure |
PUPs
n=36 Participants
|
PUPs -During On-Demand Treatment
n=47 Participants
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
n=25 Participants
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Weekly rAHF-PFM Utilization
|
87.1 IU/kg
Interval 6.5 to 352.3
|
12.5 IU/kg
Interval 2.4 to 176.8
|
606.4 IU/kg
Interval 256.5 to 1951.0
|
SECONDARY outcome
Timeframe: 30 minutes pre-infusion to 30 minutes post-infusionPopulation: Participants who received pharmacokinetic rAHF-PFM infusions, did not develop inhibitors, and had assessments
Change in factor VIII concentration from pre- to post-infusion at initial and termination study visits.
Outcome measures
| Measure |
PUPs
n=4 Participants
|
PUPs -During On-Demand Treatment
n=12 Participants
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
In Vivo Incremental Recovery
|
1.81 IU/dL per IU/kg
Interval 0.74 to 1.92
|
1.71 IU/dL per IU/kg
Interval 1.32 to 2.11
|
—
|
SECONDARY outcome
Timeframe: Assessed at the time of discharge from recovery roomPopulation: Number of participants who underwent a surgical procedure with an intraoperative assessment of hemostatic efficacy
Number of surgical procedures managed with rAHF-PFM and with surgeon's assessment of hemostasis based on a 4-point ordinal scale: Excellent: ≤ average predicted blood loss for matched procedures in healthy individuals Good: \> average predicted blood loss, but ≤ maximal predicted blood loss for matched procedures in healthy individuals Fair: \> maximal predicted blood loss for matched procedures in healthy individuals, and hemostasis was achieved None: uncontrolled hemostasis with proper dosing, necessitating a change in treatment regimen
Outcome measures
| Measure |
PUPs
n=22 Participants
|
PUPs -During On-Demand Treatment
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Assessment of Intra-operative Hemostasis
Excellent
|
18 Procedures
|
—
|
—
|
|
Assessment of Intra-operative Hemostasis
Good
|
4 Procedures
|
—
|
—
|
|
Assessment of Intra-operative Hemostasis
Not Applicable
|
0 Procedures
|
—
|
—
|
|
Assessment of Intra-operative Hemostasis
Not Done
|
0 Procedures
|
—
|
—
|
SECONDARY outcome
Timeframe: Assessed at the time of discharge from hospital or clinicPopulation: Number of participants who underwent a surgical procedure with a postoperative assessment of hemostatic efficacy
Number of surgical procedures managed with rAHF-PFM and with investigator's assessment of hemostasis based on a 4-point ordinal scale: Excellent: hemostasis was as good as or better than other licensed factor VIII products for matched procedure Good: hemostasis was probably as good as other licensed factor VIII products for matched procedure Fair: hemostasis was clearly \< optimal for matched procedure, without need to change regimen None: bleeding from inadequate response with proper dosing, necessitating a change in regimen
Outcome measures
| Measure |
PUPs
n=27 Participants
|
PUPs -During On-Demand Treatment
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Assessment of Postoperative Hemostasis
Excellent
|
23 Procedures
|
—
|
—
|
|
Assessment of Postoperative Hemostasis
Good
|
2 Procedures
|
—
|
—
|
|
Assessment of Postoperative Hemostasis
Not Applicable
|
1 Procedures
|
—
|
—
|
|
Assessment of Postoperative Hemostasis
Not Done
|
1 Procedures
|
—
|
—
|
SECONDARY outcome
Timeframe: Predicted volumes preoperatively estimated and actual volumes intraoperatively recordedPopulation: Number of participants who underwent a surgical procedure with blood loss assessments
Percentage of actual intraoperative blood loss compared to preoperatively predicted average and maximal blood loss in hemostatically normal matched individuals (from institutional blood bank records)
Outcome measures
| Measure |
PUPs
n=27 Participants
|
PUPs -During On-Demand Treatment
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Assessment of Blood Loss During Surgical Procedures
Blood loss as percentage of predicted average
|
50.0 Percentage Blood Loss
Interval 0.0 to 250.0
|
—
|
—
|
|
Assessment of Blood Loss During Surgical Procedures
Blood loss as percentage of predicted maximal
|
20.0 Percentage Blood Loss
Interval 0.0 to 100.0
|
—
|
—
|
SECONDARY outcome
Timeframe: Reported during the study period which was to be at least 75 exposure days or 3 years (whichever came first)Population: Participants who received at least 1 infusion of rAHF-PFM
Percentage of participants who reported AEs deemed related to treatment with rAHF-PFM
Outcome measures
| Measure |
PUPs
n=55 Participants
|
PUPs -During On-Demand Treatment
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Adverse Events Deemed Related to Treatment
|
36.4 Percentage of Participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Assessed at baseline, throughout the duration of the study, which was to be at least 75 exposure days or 3 years (whichever came first), and at the termination visit.Population: Participants who received at least 1 infusion of rAHF-PFM and had assessments of heterologous antibodies
Percentage of treated participants who developed antibodies to heterologous proteins (ie, Chinese Hamster Ovary Cell Protein, Murine IgG, or Recombinant Human VWF)
Outcome measures
| Measure |
PUPs
n=55 Participants
|
PUPs -During On-Demand Treatment
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Development of Antibodies to Heterologous Proteins
Antibodies to Chinese Hamster Ovary Cell Protein
|
0.00 Percentage of Participants
|
—
|
—
|
|
Development of Antibodies to Heterologous Proteins
Antibodies to Murine IgG
|
0.00 Percentage of Participants
|
—
|
—
|
|
Development of Antibodies to Heterologous Proteins
Antibodies to Recombinant Human VWF (n=54)
|
0.00 Percentage of Participants
|
—
|
—
|
POST_HOC outcome
Timeframe: Duration of study which was to be at least 75 exposure days or 3 years (whichever came first)Population: Immunogenicity Analysis Set- Participants who developed an inhibitor or who were inhibitor-free with ≥10 exposure days to rAHF-PFM
Number of treated participants who developed an inhibitor
Outcome measures
| Measure |
PUPs
n=34 Participants
|
PUPs -During On-Demand Treatment
n=16 Participants
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Factor VIII Inhibitor Risk Factor: Genetic Risk Factor- Family History of Inhibitors
Has Family History of Inhibitors
|
6 Participants
|
8 Participants
|
—
|
|
Factor VIII Inhibitor Risk Factor: Genetic Risk Factor- Family History of Inhibitors
Unknown Family History of Inhibitors
|
2 Participants
|
1 Participants
|
—
|
|
Factor VIII Inhibitor Risk Factor: Genetic Risk Factor- Family History of Inhibitors
No Family History of Inhibitors
|
26 Participants
|
7 Participants
|
—
|
POST_HOC outcome
Timeframe: Duration of study which was to be at least 75 exposure days or 3 years (whichever came first)Population: Immunogenicity Analysis Set- Participants who developed an inhibitor or who were inhibitor-free with ≥10 exposure days to rAHF-PFM
Number of treated participants who developed an inhibitor
Outcome measures
| Measure |
PUPs
n=34 Participants
|
PUPs -During On-Demand Treatment
n=16 Participants
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Factor VIII Inhibitor Risk Factor: Race
Non-Caucasian
|
8 Participants
|
9 Participants
|
—
|
|
Factor VIII Inhibitor Risk Factor: Race
Caucasian
|
26 Participants
|
7 Participants
|
—
|
POST_HOC outcome
Timeframe: Duration of study which was to be at least 75 exposure days or 3 years (whichever came first)Population: Immunogenicity Analysis Set- Participants who developed an inhibitor or who were inhibitor-free with ≥10 exposure days to rAHF-PFM
Immunogenicity Analysis Set- Participants with 5 consecutive study days of a mean infusion dose of FVIII \>50 IU/kg within ≤20 EDs who developed an inhibitor
Outcome measures
| Measure |
PUPs
n=34 Participants
|
PUPs -During On-Demand Treatment
n=16 Participants
The dosing regimen used to treat BEs was at the discretion of the investigator and in accordance with the institution's standard of care for the type of BE diagnosed.
|
PUPs -During Perioperative Management
rAHF-PFM was administered intravenously via bolus infusion, or continuous infusion. The dosing regimen used was at the discretion of the investigator and in accordance with the institution's standard of care.
|
|---|---|---|---|
|
Factor VIII Inhibitor Risk Factor: Number of Participants With Intensive Treatment and High Dose (≤20 Exposure Days (EDs))
Received intensive treatment & high dose
|
4 Participants
|
6 Participants
|
—
|
|
Factor VIII Inhibitor Risk Factor: Number of Participants With Intensive Treatment and High Dose (≤20 Exposure Days (EDs))
No intensive treatment & high dose
|
30 Participants
|
10 Participants
|
—
|
Adverse Events
PUPs
Serious events: 28 serious events
Other events: 52 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
PUPs
n=55 participants at risk
|
|---|---|
|
Blood and lymphatic system disorders
Factor VIII inhibition
|
29.1%
16/55 • Number of events 16 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Blood and lymphatic system disorders
Coagulopathy
|
3.6%
2/55 • Number of events 3 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Injury, poisoning and procedural complications
Mouth injury
|
3.6%
2/55 • Number of events 2 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
General disorders
Pyrexia
|
3.6%
2/55 • Number of events 2 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Bacteraemia
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial hyperreactivity
|
1.8%
1/55 • Number of events 3 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Bronchitis
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Catheter bacteraemia
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Catheter related infection
|
1.8%
1/55 • Number of events 3 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
General disorders
Catheter site haematoma
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Injury, poisoning and procedural complications
Contusion
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
1.8%
1/55 • Number of events 2 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Metabolism and nutrition disorders
Dehydration
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Musculoskeletal and connective tissue disorders
Haemarthrosis
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Vascular disorders
Haematoma
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Injury, poisoning and procedural complications
Head injury
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Pneumonia
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Respiratory syncytial virus infection
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Injury, poisoning and procedural complications
Skin laceration
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Injury, poisoning and procedural complications
Tongue injury
|
1.8%
1/55 • Number of events 1 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
1.8%
1/55 • Number of events 2 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
Other adverse events
| Measure |
PUPs
n=55 participants at risk
|
|---|---|
|
General disorders
Pyrexia
|
70.9%
39/55 • Number of events 137 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Nasopharyngitis
|
50.9%
28/55 • Number of events 80 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
41.8%
23/55 • Number of events 65 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
40.0%
22/55 • Number of events 54 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Gastrointestinal disorders
Diarrhea
|
36.4%
20/55 • Number of events 31 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Ear infection
|
36.4%
20/55 • Number of events 44 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Gastrointestinal disorders
Vomiting
|
30.9%
17/55 • Number of events 29 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Upper respiratory tract infection
|
29.1%
16/55 • Number of events 35 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
27.3%
15/55 • Number of events 48 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Skin and subcutaneous tissue disorders
Rash
|
23.6%
13/55 • Number of events 18 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Blood and lymphatic system disorders
Anaemia
|
18.2%
10/55 • Number of events 13 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Eye disorders
Conjuctivitis
|
16.4%
9/55 • Number of events 12 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
16.4%
9/55 • Number of events 11 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
12.7%
7/55 • Number of events 8 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Otitis media
|
12.7%
7/55 • Number of events 13 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
10.9%
6/55 • Number of events 10 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Hand-foot-and-mouth disease
|
9.1%
5/55 • Number of events 5 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
9.1%
5/55 • Number of events 8 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
9.1%
5/55 • Number of events 5 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
7.3%
4/55 • Number of events 5 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Bronchitis
|
7.3%
4/55 • Number of events 10 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Influenza
|
7.3%
4/55 • Number of events 8 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
General disorders
Pain
|
7.3%
4/55 • Number of events 6 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Pharyngitis
|
7.3%
4/55 • Number of events 4 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Rhinitis
|
7.3%
4/55 • Number of events 14 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Varicella
|
7.3%
4/55 • Number of events 4 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Gastrointestinal disorders
Abdominal pain
|
5.5%
3/55 • Number of events 5 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.5%
3/55 • Number of events 4 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Croup infectious
|
5.5%
3/55 • Number of events 3 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Ear and labyrinth disorders
Ear pain
|
5.5%
3/55 • Number of events 3 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Gasteroenteritis
|
5.5%
3/55 • Number of events 3 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Injury, poisoning and procedural complications
Mouth injury
|
5.5%
3/55 • Number of events 4 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.5%
3/55 • Number of events 4 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Injury, poisoning and procedural complications
Skin laceration
|
5.5%
3/55 • Number of events 3 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
|
Infections and infestations
Viral infection
|
5.5%
3/55 • Number of events 5 • Study period was to be at least 75 exposure days or 3 years (whichever came first)
Median days on study was 498 (range: 82 to 1360) days. Median days of rAHF-PFM exposure was 76 (range: 1 to 414) exposure days.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Baxter's agreements with PIs vary per individual PI, but contain common elements. For this study, PIs are restricted from independently publishing results until the earlier of the primary multicenter publication or 1 year after study completion. Baxter requires a review of results communications (e.g., for confidential information) 30 days prior to submission or communication. Baxter may request an additional delay of up to 90 days (e.g., to allow for intellectual property protection).
- Publication restrictions are in place
Restriction type: OTHER