Trial Outcomes & Findings for Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert (NCT NCT01467947)

Last Updated: 2017-02-06

Results Overview

Subjects with no positive baseline result and at least one positive post-baseline result for inhibitory anti-C1-INH antibodies.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

46 participants

Primary outcome timeframe

Baseline to approximately 9 months

Results posted on

2017-02-06

Participant Flow

60 subjects were screened and enrolled in the study. 14 subjects did not experience an HAE attack before the study ended and so did not receive study treatment. 46 subjects started and completed the active treatment period of the study.

Participant milestones

Participant milestones
Measure	C1 Esterase Inhibitor Subjects received 20 IU C1 Esterase Inhibitor (C1-INH)/kg body weight by slow intravenous infusion for each acute HAE attack requiring treatment during a 9-month period beginning after their first HAE attack while on study.
Overall Study STARTED	46
Overall Study COMPLETED	46
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	C1 Esterase Inhibitor n=46 Participants Subjects received 20 IU/kg C1 Esterase Inhibitor (C1-INH)/kg body weight by slow intravenous infusion for each acute HAE attack requiring treatment during a 9-month period beginning after their first HAE attack while on study.
Age, Categorical <=18 years	2 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	42 Participants n=99 Participants
Age, Categorical >=65 years	2 Participants n=99 Participants
Age, Continuous	38.9 years STANDARD_DEVIATION 14.42 • n=99 Participants
Gender Female	32 Participants n=99 Participants
Gender Male	14 Participants n=99 Participants
Region of Enrollment Romania	15 participants n=99 Participants
Region of Enrollment Hungary	8 participants n=99 Participants
Region of Enrollment Poland	13 participants n=99 Participants
Region of Enrollment Bulgaria	10 participants n=99 Participants

PRIMARY outcome

Timeframe: Baseline to approximately 9 months

Subjects with no positive baseline result and at least one positive post-baseline result for inhibitory anti-C1-INH antibodies.

Outcome measures

Outcome measures
Measure	C1 Esterase Inhibitor n=46 Participants Subjects received 20 IU C1 Esterase Inhibitor (C1-INH)/kg body weight by slow intravenous infusion for each acute HAE attack requiring treatment during a 9-month period beginning after their first HAE attack while on study.
Number of Subjects With Inhibitory Anti-C1-esterase-inhibitor Antibodies	0 subjects

SECONDARY outcome

Timeframe: Baseline to approximately 9 months

Subjects with at least one positive result for inhibitory or non-inhibitory anti-C1-INH antibodies.

Outcome measures

Outcome measures
Measure	C1 Esterase Inhibitor n=46 Participants Subjects received 20 IU C1 Esterase Inhibitor (C1-INH)/kg body weight by slow intravenous infusion for each acute HAE attack requiring treatment during a 9-month period beginning after their first HAE attack while on study.
Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies Antibodies detected on Day 1 (Baseline)	9 Subjects
Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies Antibodies detected post-baseline	10 Subjects
Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies Antibodies first detected at Month 3	2 Subjects
Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies Antibodies first detected at Month 6	1 Subjects
Number of Subjects With Any (Inhibitory or Non-inhibitory) Anti-C1-esterase-inhibitor Antibodies Antibodies first detected at Month 9	1 Subjects

Adverse Events

C1 Esterase Inhibitor

Serious events: 2 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	C1 Esterase Inhibitor n=46 participants at risk Subjects received 20 IU C1 Esterase Inhibitor (C1-INH)/kg body weight by slow intravenous infusion for each acute HAE attack requiring treatment during a 9-month period beginning after their first HAE attack while on study.
Congenital, familial and genetic disorders Hereditary angioedema	2.2% 1/46 • Number of events 1 • Baseline to approximately 9 months.
Pregnancy, puerperium and perinatal conditions Abortion spontaneous	2.2% 1/46 • Number of events 1 • Baseline to approximately 9 months.

Other adverse events

Other adverse events
Measure	C1 Esterase Inhibitor n=46 participants at risk Subjects received 20 IU C1 Esterase Inhibitor (C1-INH)/kg body weight by slow intravenous infusion for each acute HAE attack requiring treatment during a 9-month period beginning after their first HAE attack while on study.
Vascular disorders Hypotension	6.5% 3/46 • Number of events 3 • Baseline to approximately 9 months.
Nervous system disorders Headache	13.0% 6/46 • Number of events 26 • Baseline to approximately 9 months.

Additional Information

Program Director, Clinical R&D

CSL Behring

Phone: Use email contact

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee CSL agreements and restrictions on publishing may vary with individual investigators; however, CSL will not prohibit any investigator from publishing. CSL supports the publication of results from all centers of a multi-center trial and generally requires that reports based on single-site data not precede the primary publication of the entire clinical trial.
Publication restrictions are in place

Restriction type: OTHER