Trial Outcomes & Findings for The Hemophilia Inhibitor Prevention Trial (NCT NCT04303559)
NCT ID: NCT04303559
Last Updated: 2023-04-11
Results Overview
The proportion developing anti-FVIII inhibitors.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
1 participants
Primary outcome timeframe
48 weeks
Results posted on
2023-04-11
Participant Flow
Participant milestones
| Measure |
Eloctate
Arm A: Eloctate 65 IU/kg will be administered weekly by intravenous infusion in previously untreated children with severe hemophilia A beginning before the first bleed and continued up to 48 weeks.
Eloctate Injectable Product: This is a factor VIII-Fc fusion protein.
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Eloctate
Arm A: Eloctate 65 IU/kg will be administered weekly by intravenous infusion in previously untreated children with severe hemophilia A beginning before the first bleed and continued up to 48 weeks.
Eloctate Injectable Product: This is a factor VIII-Fc fusion protein.
|
|---|---|
|
Overall Study
Study closed due to poor enrollment.
|
1
|
Baseline Characteristics
The Hemophilia Inhibitor Prevention Trial
Baseline characteristics by cohort
| Measure |
Eloctate
n=1 Participants
Arm A: Eloctate 65 IU/kg will be administered weekly by intravenous infusion in previously untreated children with severe hemophilia A beginning before the first bleed and continued up to 48 weeks.
Eloctate Injectable Product: This is a factor VIII-Fc fusion protein.
|
|---|---|
|
Age, Categorical
<=18 years
|
1 Participants
n=99 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
|
Age, Continuous
|
1 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=99 Participants
|
|
Number and Type of Bleeds in the last year
|
0 Bleeds
n=99 Participants
|
|
Prior Circumcision,
|
1 Participants
n=99 Participants
|
|
Anti-FVIII level > 0.6 BU
|
0 Participants
n=99 Participants
|
|
Number of FVIII exposures
|
0 FVIII exposures
n=99 Participants
|
PRIMARY outcome
Timeframe: 48 weeksPopulation: Only patient was enrolled on study.
The proportion developing anti-FVIII inhibitors.
Outcome measures
| Measure |
Eloctate
n=1 Participants
Arm A: Eloctate 65 IU/kg will be administered weekly by intravenous infusion in previously untreated children with severe hemophilia A beginning before the first bleed and continued up to 48 weeks.
Eloctate Injectable Product: This is a factor VIII-Fc fusion protein.
|
|---|---|
|
Inhibitor Formation
|
1 Participants
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: Only patient was enrolled on study.
The number of bleeding events:hematoma, joint, central nervous system, other bleeds.
Outcome measures
| Measure |
Eloctate
n=1 Participants
Arm A: Eloctate 65 IU/kg will be administered weekly by intravenous infusion in previously untreated children with severe hemophilia A beginning before the first bleed and continued up to 48 weeks.
Eloctate Injectable Product: This is a factor VIII-Fc fusion protein.
|
|---|---|
|
Bleeding Events
|
1 bleeding events
|
SECONDARY outcome
Timeframe: 48 weeksPopulation: specimen collected, discarded with out analysis due to study being close dearly due to low enrollment
The FVIII trough activity by chromogenic assay
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 48 weeksPopulation: specimen collected, discarded with out analysis due to study being close dearly due to low enrollment
The number of HLA haplotype variants.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 48 weeksPopulation: specimen collected, discarded with out analysis due to study being close dearly due to low enrollment
The number of FVIII mutation variants.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 48 weeksPopulation: Only patient was enrolled on study.
Number of FVIII exposures,
Outcome measures
| Measure |
Eloctate
n=1 Participants
Arm A: Eloctate 65 IU/kg will be administered weekly by intravenous infusion in previously untreated children with severe hemophilia A beginning before the first bleed and continued up to 48 weeks.
Eloctate Injectable Product: This is a factor VIII-Fc fusion protein.
|
|---|---|
|
Number of FVIII Exposures
|
2 FVIII exposures
|
Adverse Events
Eloctate
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Eloctate
n=1 participants at risk
Arm A: Eloctate 65 IU/kg will be administered weekly by intravenous infusion in previously untreated children with severe hemophilia A beginning before the first bleed and continued up to 48 weeks.
Eloctate Injectable Product: This is a factor VIII-Fc fusion protein.
|
|---|---|
|
Blood and lymphatic system disorders
Joint bleed requiring factor
|
100.0%
1/1 • Number of events 1 • 48 weeks
Adverse events were self-reported.
|
Additional Information
Dr. Margaret Ragni, Principal Investigator
University of Pittsburgh
Phone: 412-209-7288
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place