Trial Outcomes & Findings for C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study) (NCT NCT00292981)

NCT ID: NCT00292981

Last Updated: 2015-05-07

Results Overview

The start of symptom relief was determined by subject self-assessment.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 57 participants
• Primary outcome timeframe: Up to 24 h after start of study treatment
• Results posted on: 2015-05-07

Participant Flow

This was a multicenter, open-label extension study enrolling subjects at 15 sites in North America that had participated in study CE1145_3001 (NCT00168103). Enrollment occurred between August 2005 and January 2008.

Participant milestones

Measure	C1 Esterase Inhibitor
Overall Study STARTED	57
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	39

Reasons for withdrawal

Measure	C1 Esterase Inhibitor
Overall Study Adverse Event	1
Overall Study Lost to Follow-up	13
Overall Study Withdrawal by Subject	22
Overall Study Other	3

Baseline Characteristics

C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)

Baseline characteristics by cohort

Measure	C1 Esterase Inhibitor n=57 Participants
Age, Continuous	31.9 years STANDARD_DEVIATION 11.98 • n=5 Participants
Sex: Female, Male Female	38 Participants n=5 Participants
Sex: Female, Male Male	19 Participants n=5 Participants
Type of HAE Type I HAE	49 participants n=5 Participants
Type of HAE Type II HAE	7 participants n=5 Participants
Type of HAE Unknown	1 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 24 h after start of study treatment

Population: Intent to treat (ITT) subject population: All subjects admitted to the study who received any portion of the study medication.

The start of symptom relief was determined by subject self-assessment.

Outcome measures

Measure	C1 Esterase Inhibitor n=57 Participants
Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population)	0.46 hours Interval 0.39 to 0.53

PRIMARY outcome

Timeframe: Up to 24 h after start of study treatment

Population: ITT attack population: All attacks in subjects admitted to the study for which any portion of study medication was administered.

The start of symptom relief was determined by subject self-assessment.

Outcome measures

Measure	C1 Esterase Inhibitor n=1085 HAE Attacks
Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population)	0.37 hours Interval 0.33 to 0.42

SECONDARY outcome

Timeframe: Up to Day 9 following an attack

Population: Intent to treat (ITT) subject population: All subjects admitted to the study who received any portion of the study medication.

Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card.

Outcome measures

Measure	C1 Esterase Inhibitor n=57 Participants
Time to Complete Resolution of All HAE Symptoms (ITT Subject Population)	15.48 hours Interval 11.64 to 21.59

SECONDARY outcome

Timeframe: Up to Day 9 following an attack

Population: ITT attack population: All attacks in subjects admitted to the study for which any portion of study medication was administered.

Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card.

Outcome measures

Measure	C1 Esterase Inhibitor n=1085 HAE Attacks
Time to Complete Resolution of All HAE Symptoms (ITT Attack Population)	14.28 hours Interval 12.07 to 15.8

Adverse Events

C1 Esterase Inhibitor

Serious events: 1 serious events

Other events: 25 other events

Deaths: 0 deaths

Serious adverse events

Measure	C1 Esterase Inhibitor n=57 participants at risk
Congenital, familial and genetic disorders Hereditary angioedema	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Staphylococcal infection	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.

Other adverse events

Measure	C1 Esterase Inhibitor n=57 participants at risk
Congenital, familial and genetic disorders Hereditary angioedema	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Eye disorders Conjunctivitis	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Gastrointestinal disorders Abdominal discomfort	3.5% 2/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Gastrointestinal disorders Abdominal pain	3.5% 2/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Gastrointestinal disorders Abdominal distension	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Gastrointestinal disorders Abdominal tenderness	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Gastrointestinal disorders Constipation	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Gastrointestinal disorders Dry mouth	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Gastrointestinal disorders Nausea	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
General disorders Influenza like illness	3.5% 2/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
General disorders Local swelling	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
General disorders Pyrexia	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
General disorders Infusion related reaction	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Nasopharyngitis	5.3% 3/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Upper respiratory tract infection	3.5% 2/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Vulvovaginal mycotic infection	3.5% 2/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Erythema infectiosum	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Herpes zoster	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Periodontal infection	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Pharyngitis streptococcal	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Sinusitis	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Urinary tract infection	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Viral infection	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Gastroenteritis bacterial	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Infections and infestations Vaginitis bacterial	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Injury, poisoning and procedural complications Clavicle fracture	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Injury, poisoning and procedural complications Hand fracture	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Injury, poisoning and procedural complications Muscle strain	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Investigations Blood potassium decreased	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Investigations Red blood cells urine positive	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Investigations Urine leukocyte esterase positive	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Musculoskeletal and connective tissue disorders Back pain	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Musculoskeletal and connective tissue disorders Joint swelling	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Musculoskeletal and connective tissue disorders Muscle spasms	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Musculoskeletal and connective tissue disorders Myalgia	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Musculoskeletal and connective tissue disorders Neck pain	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Nervous system disorders Headache	8.8% 5/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Nervous system disorders Dizziness	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Psychiatric disorders Insomnia	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Reproductive system and breast disorders Dysmenorrhoea	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Skin and subcutaneous tissue disorders Rash	3.5% 2/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Skin and subcutaneous tissue disorders Dermatitis contact	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
Skin and subcutaneous tissue disorders Pruritus	1.8% 1/57 • For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack. Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.

Additional Information

Clinical Program Director

CSL Behring

Phone: Use email contact

Email: clinicaltrials@cslbehring.com

Results disclosure agreements

• Principal investigator is a sponsor employee The investigator must provide a copy of any results communication to the sponsor for review at least 30 days prior to public release. The sponsor may request any changes necessary to prevent forfeiture of patent rights to data not in the public domain. For a multi-center study, the investigator must wait (i) at least 1 year after the study is completed at all sites or (ii) until notified by the sponsor that no multi-center publication is planned before seeking publication review.
• Publication restrictions are in place

Restriction type: OTHER