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Trial Outcomes & Findings for A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor (NCT NCT02247739)

NCT ID: NCT02247739

Last Updated: 2017-12-08

Results Overview

Average number of HAE attacks normalized to a 28 day period

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

32 participants

Primary outcome timeframe

28 days

Results posted on

2017-12-08

Participant Flow

Participant milestones

Participant milestones
Measure
Treatment Sequence A
rhC1INH twice weekly / rhC1INH once weekly / saline
Treatments Sequence B
rhC1INH once weekly / rhC1INH twice weekly / Saline
Treatment Sequesce C
Saline / rhC1INH once weekly / rhC1INH twice weekly
Treatment Sequence D
Saline / rhC1INH twice weekly / rhC1INH once weekly
Treatment Sequence E
rhC1INH twice weekly / saline / rhC1INH once weekly
Treatment Sequence F
rhC1INH once weekly / saline / rhC1INH twice weekly
Overall Study
STARTED
5
6
5
5
6
5
Overall Study
COMPLETED
5
3
4
5
5
4
Overall Study
NOT COMPLETED
0
3
1
0
1
1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
All Study Participants
n=32 Participants
All randomized patients
Age, Categorical
<=18 years
1 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
28 Participants
n=5 Participants
Age, Categorical
>=65 years
3 Participants
n=5 Participants
Age, Continuous
46 years
n=5 Participants
Sex: Female, Male
Female
26 Participants
n=5 Participants
Sex: Female, Male
Male
6 Participants
n=5 Participants
Region of Enrollment
Canada
2 participants
n=5 Participants
Region of Enrollment
Czech Republic
2 participants
n=5 Participants
Region of Enrollment
Romania
6 participants
n=5 Participants
Region of Enrollment
United States
7 participants
n=5 Participants
Region of Enrollment
Macedonia, The Former Yugoslav Republic of
8 participants
n=5 Participants
Region of Enrollment
Italy
1 participants
n=5 Participants
Region of Enrollment
Serbia
2 participants
n=5 Participants
Region of Enrollment
Israel
4 participants
n=5 Participants

PRIMARY outcome

Timeframe: 28 days

Population: Intent-to-Treat (ITT) Population: All patients who were randomized into one of the treatment sequences. The statistical analyses are based on the treatments to which the patient was randomized to receive during that treatment period.

Average number of HAE attacks normalized to a 28 day period

Outcome measures

Outcome measures
Measure
rhC1INH Twice Weekly
n=32 Participants
rhC1INH administered twice weekly Recombinant human C1 inhibitor
rhC1INH Once Weekly
n=32 Participants
rhC1INH administered once weekly Recombinant human C1 inhibitor
Placebo (Saline) Twice Weekly
n=32 Participants
Placebo (Saline) administered twice weekly Placebo
Number of HAE Attacks
2.74 attacks
Interval 1.8 to 3.7
4.36 attacks
Interval 3.1 to 5.6
7.18 attacks
Interval 5.8 to 8.6

SECONDARY outcome

Timeframe: 20 weeks

Population: Safety Population: All patients who received at least a partial injection of study drug. The statistical analyses are based on the actual treatment the patient received.

Number of participants that experienced Treatment Emergent Adverse Events observed in safety population

Outcome measures

Outcome measures
Measure
rhC1INH Twice Weekly
n=29 Participants
rhC1INH administered twice weekly Recombinant human C1 inhibitor
rhC1INH Once Weekly
n=29 Participants
rhC1INH administered once weekly Recombinant human C1 inhibitor
Placebo (Saline) Twice Weekly
n=28 Participants
Placebo (Saline) administered twice weekly Placebo
Number of Participants With Adverse Events
10 Participants
13 Participants
8 Participants

SECONDARY outcome

Timeframe: 28 days

Population: Both the rhC1INH twice weekly treatments and the rhC1INH once weekly treatment periods are compared to the placebo treatment for the safety population. One subject withdrew before receiving any treatment and is excluded from the analysis.

Percentage of participants achieving at least 50% reduction in the number of attacks normalized to a 28-day period as compared to the placebo treatment period

Outcome measures

Outcome measures
Measure
rhC1INH Twice Weekly
n=31 Participants
rhC1INH administered twice weekly Recombinant human C1 inhibitor
rhC1INH Once Weekly
n=31 Participants
rhC1INH administered once weekly Recombinant human C1 inhibitor
Placebo (Saline) Twice Weekly
Placebo (Saline) administered twice weekly Placebo
Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks
74 percentage of participants
Interval 57.0 to 86.0
42 percentage of participants
Interval 26.0 to 59.0
—

OTHER_PRE_SPECIFIED outcome

Timeframe: 20 weeks

Population: Participants analyzed for Neutralizing antibodies after confirmed testing of anti-C1INH and anti rhC1INH IgM or IgG antibodies. Count of participants displays the number of positives.

Number of participants analyzed for neutralizing C1INH-specific antibodies and neutralizing rhC1INH-specific antibodies after confirmed anti-C1INH and anti rhC1INH IgM or IgG antibodies

Outcome measures

Outcome measures
Measure
rhC1INH Twice Weekly
n=3 Participants
rhC1INH administered twice weekly Recombinant human C1 inhibitor
rhC1INH Once Weekly
n=5 Participants
rhC1INH administered once weekly Recombinant human C1 inhibitor
Placebo (Saline) Twice Weekly
n=2 Participants
Placebo (Saline) administered twice weekly Placebo
Immunogenicity
0 Participants
0 Participants
0 Participants

Adverse Events

rhC1INH Twice Weekly

Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths

rhC1INH Once Weekly

Serious events: 0 serious events
Other events: 7 other events
Deaths: 0 deaths

Placebo (Saline) Twice Weekly

Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
rhC1INH Twice Weekly
n=29 participants at risk
rhC1INH administered twice weekly Recombinant human C1 inhibitor
rhC1INH Once Weekly
n=29 participants at risk
rhC1INH administered once weekly Recombinant human C1 inhibitor
Placebo (Saline) Twice Weekly
n=28 participants at risk
Placebo (Saline) administered twice weekly Placebo
Congenital, familial and genetic disorders
Phimosis
3.4%
1/29 • Number of events 1 • 20 weeks
0.00%
0/29 • 20 weeks
0.00%
0/28 • 20 weeks

Other adverse events

Other adverse events
Measure
rhC1INH Twice Weekly
n=29 participants at risk
rhC1INH administered twice weekly Recombinant human C1 inhibitor
rhC1INH Once Weekly
n=29 participants at risk
rhC1INH administered once weekly Recombinant human C1 inhibitor
Placebo (Saline) Twice Weekly
n=28 participants at risk
Placebo (Saline) administered twice weekly Placebo
Nervous system disorders
Headache
17.2%
5/29 • Number of events 5 • 20 weeks
6.9%
2/29 • Number of events 2 • 20 weeks
0.00%
0/28 • 20 weeks
Infections and infestations
Nasopharyngitis
0.00%
0/29 • 20 weeks
10.3%
3/29 • Number of events 3 • 20 weeks
7.1%
2/28 • Number of events 2 • 20 weeks
Psychiatric disorders
Anxiety
0.00%
0/29 • 20 weeks
6.9%
2/29 • Number of events 2 • 20 weeks
0.00%
0/28 • 20 weeks

Additional Information

VP Clinical Research & Medical Affairs

Pharming Group NV

Results disclosure agreements

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

Restriction type: LTE60