Trial Outcomes & Findings for The Hemophilia Inhibitor Eradication Trial (NCT NCT04303572)

NCT ID: NCT04303572

Last Updated: 2023-02-21

Results Overview

The proportion eradicating anti-FVIII inhibitors

• Recruitment status: TERMINATED
• Study phase: PHASE3
• Target enrollment: 1 participants
• Primary outcome timeframe: 48 weeks
• Results posted on: 2023-02-21

Participant Flow

Participant milestones

Measure	Eloctate ITI Eloctate 100 IU/kg every other day by intravenous infusion in children and adults with severe hemophilia A and anti-FVIII inhibitor, continued up to 48 weeks. Eloctate ITI: This is a factor VIII-Fc infusion protein.
Overall Study STARTED	1
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Eloctate ITI Eloctate 100 IU/kg every other day by intravenous infusion in children and adults with severe hemophilia A and anti-FVIII inhibitor, continued up to 48 weeks. Eloctate ITI: This is a factor VIII-Fc infusion protein.
Overall Study Study closed due to poor enrollment	1

Baseline Characteristics

The Hemophilia Inhibitor Eradication Trial

Baseline characteristics by cohort

Measure	Eloctate ITI n=1 Participants Eloctate 100 IU/kg every other day by intravenous infusion in children and adults with severe hemophilia A and anti-FVIII inhibitor, continued up to 48 weeks. Eloctate ITI: This is a factor VIII-Fc infusion protein.
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	1 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 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
Region of Enrollment United States	1 participants n=5 Participants
Number and Type of Bleeds in the last year	0 Bleeds n=5 Participants
Prior Circumcision	1 Participants n=5 Participants
Anti-FVIII level > 0.6 BU	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 48 weeks

Population: Only patient was enrolled on study.

The proportion eradicating anti-FVIII inhibitors

Outcome measures

Measure	Eloctate ITI n=1 Participants Eloctate 100 IU/kg every other day by intravenous infusion in children and adults with severe hemophilia A and anti-FVIII inhibitor, continued up to 48 weeks. Eloctate ITI: This is a factor VIII-Fc infusion protein.
Inhibitor Eradication	1 Participants

SECONDARY outcome

Timeframe: 48 weeks

Population: Only patient was enrolled on study.

The number of bleeding events: hematoma, joint, central nervous system, other bleeds.

Outcome measures

Measure	Eloctate ITI n=1 Participants Eloctate 100 IU/kg every other day by intravenous infusion in children and adults with severe hemophilia A and anti-FVIII inhibitor, continued up to 48 weeks. Eloctate ITI: This is a factor VIII-Fc infusion protein.
Number of Bleeding Events	0 number of bleeding events

SECONDARY outcome

Timeframe: 48 weeks

Population: 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 weeks

Population: 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 weeks

Population: 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

Adverse Events

Eloctate ITI

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Margaret Ragni, Principal Investigator

University of Pittsburgh

Phone: 412-209-7288

Email: ragni@pitt.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place