Trial Outcomes & Findings for Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients (NCT NCT04396639)
NCT ID: NCT04396639
Last Updated: 2021-05-03
Results Overview
FVIII inhibitor development was defined as an inhibitor titer of \>=0.6 Bethesda units per milliliter (BU)/mL as confirmed by the central laboratory during the course of the study.
COMPLETED
PHASE4
50 participants
24 exposure days or 8 weeks of treatment during the study (whichever occurred first)
2021-05-03
Participant Flow
In this study, participants aged greater than or equal to (\>=) 12 years to less than or equal to (\<=) 65 years, with moderate or severe hemophilia A (circulating factor VIII \[FVIII: C\] \<=5 percent \[%\]), who previously had at least 50 exposure days to FVIII-containing products were enrolled.
Participant milestones
| Measure |
Moroctocog Alfa (AF-CC)
Moroctocog alfa was administered prophylactically at a dose of 30 international unit per kilogram (IU/kg), 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
|
|---|---|
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Overall Study
STARTED
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50
|
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Overall Study
Completed Follow up
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48
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|
Overall Study
COMPLETED
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48
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Overall Study
NOT COMPLETED
|
2
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Reasons for withdrawal
| Measure |
Moroctocog Alfa (AF-CC)
Moroctocog alfa was administered prophylactically at a dose of 30 international unit per kilogram (IU/kg), 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
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|---|---|
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Overall Study
Withdrawal by Subject
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1
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Overall Study
Other
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1
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Baseline Characteristics
Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients
Baseline characteristics by cohort
| Measure |
Moroctocog Alfa (AF-CC)
n=50 Participants
Moroctocog alfa was administered prophylactically at a dose of 30 IU/kg, 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
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|---|---|
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Age, Continuous
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29.58 Years
STANDARD_DEVIATION 9.80 • n=5 Participants
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Sex: Female, Male
Female
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0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
50 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
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0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
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50 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
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|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
50 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: 24 exposure days or 8 weeks of treatment during the study (whichever occurred first)Population: Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
FVIII inhibitor development was defined as an inhibitor titer of \>=0.6 Bethesda units per milliliter (BU)/mL as confirmed by the central laboratory during the course of the study.
Outcome measures
| Measure |
Moroctocog Alfa (AF-CC)
n=50 Participants
Moroctocog alfa was administered prophylactically at a dose of 30 IU/kg, 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
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|---|---|
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Percentage of Participants Who Developed Factor VIII (FVIII) Inhibitors
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0 percentage of participants
Interval 0.0 to 0.06
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SECONDARY outcome
Timeframe: Day 1 up to 28 days after last dose of study drug (approximately maximum up to 12 Weeks)Population: Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Treatment emergent are events between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pretreatment state. In this outcome measure all AEs are reported.
Outcome measures
| Measure |
Moroctocog Alfa (AF-CC)
n=50 Participants
Moroctocog alfa was administered prophylactically at a dose of 30 IU/kg, 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
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|---|---|
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Number of Participants With Treatment Emergent Adverse Events (TEAEs)
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3 Participants
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SECONDARY outcome
Timeframe: Day 1 up to 28 days after last dose of study drug (approximately maximum up to 12 Weeks)Population: Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
SAEs: an adverse event resulting in any of the following outcomes or deemed significant for any other reason: death; inpatient hospitalization or prolongation of existing hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly/birth defect. Treatment emergent are events between first dose of study drug and up to 28 days after last dose that were absent before treatment or that worsened relative to pretreatment state.
Outcome measures
| Measure |
Moroctocog Alfa (AF-CC)
n=50 Participants
Moroctocog alfa was administered prophylactically at a dose of 30 IU/kg, 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
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|---|---|
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Number of Participants With Treatment Emergent Serious Adverse Events (SAEs)
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0 Participants
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SECONDARY outcome
Timeframe: 24 exposure days or 8 weeks of treatment during the study (whichever occurred first)Population: Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
Participants who had bleeding episode, ABR was derived by the following formula: ABR = number of bleeds per treatment interval duration per 365.25. Participants who did not have bleeding episodes, ABR was 0. In this outcome measure, mean ABR was reported considering all the participants (with bleeding episodes and without bleeding episodes).
Outcome measures
| Measure |
Moroctocog Alfa (AF-CC)
n=50 Participants
Moroctocog alfa was administered prophylactically at a dose of 30 IU/kg, 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
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|---|---|
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Mean Annualized Bleeding Rate (ABR) During Prophylaxis
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0.79 bleed rate per year
Standard Deviation 2.042
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SECONDARY outcome
Timeframe: 24 exposure days or 8 weeks of treatment during the study (whichever occurred first)Population: Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
The total amount in international units (IU) infused for each infusion recorded were summed to calculate the TFC for each participant during the treatment interval duration (up to 8 weeks of treatment, or sooner, once 24 exposure days are achieved). The annualized TFC of moroctocog alfa was derived for each participant by using the following formula: Annualized TFC = (TFC / treatment interval duration)\*365.25.
Outcome measures
| Measure |
Moroctocog Alfa (AF-CC)
n=50 Participants
Moroctocog alfa was administered prophylactically at a dose of 30 IU/kg, 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
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|---|---|
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Mean Annualized Total Factor Consumption (TFC)
|
287432.26 international units
Standard Deviation 93866.233
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SECONDARY outcome
Timeframe: 24 exposure days or 8 weeks of treatment during the study (whichever occurred first)Population: Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
The total amount in IU infused for each infusion recorded was summed to calculate the TFC for each participants during the treatment interval duration (up to 8 weeks of treatment, or sooner, once 24 exposure days are achieved). The annualized TFC of moroctocog alfa was derived for each participant by using the following formula: Annualized TFC = (TFC / treatment interval duration)\*365.25. To calculate the annualized TFC per weight, the most recently recorded weight measurement was used.
Outcome measures
| Measure |
Moroctocog Alfa (AF-CC)
n=50 Participants
Moroctocog alfa was administered prophylactically at a dose of 30 IU/kg, 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
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|---|---|
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Mean Annualized Total Factor Consumption (TFC) by Weight
|
4175.67 international units per kilogram
Standard Deviation 858.383
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SECONDARY outcome
Timeframe: 24 exposure days or 8 weeks of treatment during the study (whichever occurred first)Population: Safety analysis set included all participants who received at least 1 dose of moroctocog alfa. Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure.
Number of moroctocog alfa infusions used to treat each bleed was calculated by adding the initial (on-demand) infusion to any subsequent (on-demand) infusions for the same bleed (same bleed start date/time). If there was more than one bleed location (e.g., ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence.
Outcome measures
| Measure |
Moroctocog Alfa (AF-CC)
n=7 bleeds
Moroctocog alfa was administered prophylactically at a dose of 30 IU/kg, 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
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|---|---|
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Mean Number of Moroctocog Alfa (AF-CC) Infusions Used to Treat Each New Bleed
|
1.0 infusions per bleed
Standard Deviation 0.00
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Adverse Events
Moroctocog Alfa (AF-CC)
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Moroctocog Alfa (AF-CC)
n=50 participants at risk
Moroctocog alfa was administered prophylactically at a dose of 30 IU/kg, 3 times weekly in accordance with local product document and with procedures provided by physicians. For on-demand treatment, the amount administered and the frequency of administration of moroctocog alfa was tailored to the clinical effectiveness in individual participants by their physicians. Participants continued participating in the study until 24 exposure days or until 8 weeks of treatment (whichever occurred first). Post treatment participants were followed up to 28 days.
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|---|---|
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Ear and labyrinth disorders
Vertigo
|
2.0%
1/50 • Day 1 up to 28 days after last dose of study drug (approximately maximum up to 12 Weeks)
Same event may appear as AE and SAEs, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
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|
Gastrointestinal disorders
Diarrhoea
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2.0%
1/50 • Day 1 up to 28 days after last dose of study drug (approximately maximum up to 12 Weeks)
Same event may appear as AE and SAEs, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
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Gastrointestinal disorders
Vomiting
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2.0%
1/50 • Day 1 up to 28 days after last dose of study drug (approximately maximum up to 12 Weeks)
Same event may appear as AE and SAEs, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
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|
General disorders
Pyrexia
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2.0%
1/50 • Day 1 up to 28 days after last dose of study drug (approximately maximum up to 12 Weeks)
Same event may appear as AE and SAEs, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
2.0%
1/50 • Day 1 up to 28 days after last dose of study drug (approximately maximum up to 12 Weeks)
Same event may appear as AE and SAEs, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
|
|
Vascular disorders
Hypertension
|
2.0%
1/50 • Day 1 up to 28 days after last dose of study drug (approximately maximum up to 12 Weeks)
Same event may appear as AE and SAEs, what is presented are distinct events. Event may be categorized as serious in 1 participant and as non-serious in another participant or 1 participant may have experienced both serious and non-serious event during study. Safety analysis set included all participants who received at least 1 dose of moroctocog alfa.
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
- Publication restrictions are in place
Restriction type: OTHER