Trial Outcomes & Findings for Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema Attacks (NCT NCT01756157)

NCT ID: NCT01756157

Last Updated: 2021-06-03

Results Overview

Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Participants who were dosed but did not have any attacks in the period were assigned a value of zero. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

47 participants

Primary outcome timeframe

From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period

Results posted on

2021-06-03

Participant Flow

This study was conducted at 24 sites (United States=20, Europe=4) between 04 February 2013 (first participant dosed) and 13 September 2013 (last participant contact). Of 52 screened participants, 47 were randomized and treated. Screen failure reasons were consent withdrawn by 1 participant and violation of eligibility criteria by 4 participants.

Due to emergence of, and unexpected incidence and titer of, non-neutralizing anti-rHuPH20 antibodies in some subjects after administration of CINRYZE+rHuPH20, sponsor decided to stop dosing subjects with rHuPH20 and thus close the study. However, the study was completed with collection of safety data as outlined in the protocol.

Participant milestones

Participant milestones
Measure
Treatment Sequence A/B
Participants received Treatment A in Period 1 and Treatment B in Period 2; for 8 weeks each as a single 20 milliliter (mL) subcutaneous (SC) injection per dose. A washout period of at least 7 days and no more than 30 days was maintained between the last dose in Period 1 and the first dose in Period 2. Treatment A: 1000 U CINRYZE with 24,000 U rHuPH20 twice weekly (every 3 or 4 days) for 8 weeks. Treatment B: 2000 U CINRYZE with 48,000 U rHuPH20 twice weekly (every 3 or 4 days) for 8 weeks.
Treatment Sequence B/A
Participants received Treatment B in Period 1 and Treatment A in Period 2; for 8 weeks each as a single 20 mL SC injection per dose. A washout period of at least 7 days and no more than 30 days was maintained between the last dose in Period 1 and the first dose in Period 2. Treatment B: 2000 U CINRYZE with 48,000 U rHuPH20 twice weekly (every 3 or 4 days) for 8 weeks. Treatment A: 1000 U CINRYZE with 24,000 U rHuPH20 twice weekly (every 3 or 4 days) for 8 weeks.
First Intervention Period
STARTED
23
24
First Intervention Period
COMPLETED
22
22
First Intervention Period
NOT COMPLETED
1
2
Washout Period (at Least 7 Days)
STARTED
22
22
Washout Period (at Least 7 Days)
COMPLETED
22
22
Washout Period (at Least 7 Days)
NOT COMPLETED
0
0
Second Intervention Period
STARTED
22
22
Second Intervention Period
COMPLETED
22
22
Second Intervention Period
NOT COMPLETED
0
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Treatment Sequence A/B
Participants received Treatment A in Period 1 and Treatment B in Period 2; for 8 weeks each as a single 20 milliliter (mL) subcutaneous (SC) injection per dose. A washout period of at least 7 days and no more than 30 days was maintained between the last dose in Period 1 and the first dose in Period 2. Treatment A: 1000 U CINRYZE with 24,000 U rHuPH20 twice weekly (every 3 or 4 days) for 8 weeks. Treatment B: 2000 U CINRYZE with 48,000 U rHuPH20 twice weekly (every 3 or 4 days) for 8 weeks.
Treatment Sequence B/A
Participants received Treatment B in Period 1 and Treatment A in Period 2; for 8 weeks each as a single 20 mL SC injection per dose. A washout period of at least 7 days and no more than 30 days was maintained between the last dose in Period 1 and the first dose in Period 2. Treatment B: 2000 U CINRYZE with 48,000 U rHuPH20 twice weekly (every 3 or 4 days) for 8 weeks. Treatment A: 1000 U CINRYZE with 24,000 U rHuPH20 twice weekly (every 3 or 4 days) for 8 weeks.
First Intervention Period
Withdrawal by Subject
1
0
First Intervention Period
Lost to Follow-up
0
2

Baseline Characteristics

Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema Attacks

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Entire Study Population
n=47 Participants
Included participants who received 1000 U CINRYZE with 24,000 U rHuPH20 twice weekly (every 3 or 4 days) for 8 weeks (Treatment A) first and 2000 U CINRYZE with 48,000 U rHuPH20 twice weekly (every 3 or 4 days) for 8 weeks (Treatment B) first.
Age, Continuous
39.0 years
STANDARD_DEVIATION 14.6 • n=93 Participants
Sex: Female, Male
Female
33 Participants
n=93 Participants
Sex: Female, Male
Male
14 Participants
n=93 Participants

PRIMARY outcome

Timeframe: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period

Population: Intent-to-treat efficacy (ITT-E) population included all participants who completed both randomized treatment periods and fulfilled a priori defined evaluability criteria.

Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Participants who were dosed but did not have any attacks in the period were assigned a value of zero. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.

Outcome measures

Outcome measures
Measure
Treatment A (1000 U CINRYZE + 24000 U rHuPH20)
n=22 Participants
Participants received Treatment A (1000 U CINRYZE with 24,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Treatment B (2000 U CINRYZE + 48000 U rHuPH20)
n=22 Participants
Participants received Treatment B (2000 U CINRYZE with 48,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Normalized Number of Angioedema Attacks During the Treatment Period
1.58 angioedema attacks
Interval 0.88 to 2.29
0.97 angioedema attacks
Interval 0.41 to 1.53

SECONDARY outcome

Timeframe: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period

Population: ITT-E population

Cumulative Attack-severity score was the sum of maximum symptom severity recorded for each angioedema attack, determined on the last day of symptoms and recorded as None=0, Mild=1, Moderate=2, and Severe=3 and summing over the unique attacks, yields a Cumulative Attack-severity score. None: no angioedema attack symptom; Mild: the angioedema attack symptom was noticeable to the participant but was easily tolerated and did not interfere with routine activities; Moderate: the angioedema attack symptom interfered with work/school or the ability to participate in family life and social activities; Severe: the angioedema attack symptom significantly limited the participant's ability to attend work/school or participate in family life and social activities. Cumulative attack-severity was normalized for the number of days participants participated in a given period and expressed as the monthly frequency. The scores ranged from 0 to 168 and higher scores represent worse symptoms.

Outcome measures

Outcome measures
Measure
Treatment A (1000 U CINRYZE + 24000 U rHuPH20)
n=22 Participants
Participants received Treatment A (1000 U CINRYZE with 24,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Treatment B (2000 U CINRYZE + 48000 U rHuPH20)
n=22 Participants
Participants received Treatment B (2000 U CINRYZE with 48,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Cumulative Attack-severity During the Treatment Period
3.14 Score on a scale
Standard Deviation 3.79
1.81 Score on a scale
Standard Deviation 2.55

SECONDARY outcome

Timeframe: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period

Population: ITT-E population

Cumulative Daily-severity score was the sum of the severity scores recorded for every day of reported symptoms during the treatment period. Severity scores were recorded as None=0, Mild=1, Moderate=2, and Severe=3. None: no angioedema attack symptom; Mild: the angioedema attack symptom was noticeable to the participant but was easily tolerated and did not interfere with routine activities; Moderate: the angioedema attack symptom interfered with work/school or the ability to participate in family life and social activities; Severe: the angioedema attack symptom significantly limited the participant's ability to attend work/school or participate in family life and social activities. Cumulative daily severity was normalized for the number of days participants participated in a given period and expressed as the monthly frequency. The scores ranged from 0 to 168 and higher scores represent worse symptoms.

Outcome measures

Outcome measures
Measure
Treatment A (1000 U CINRYZE + 24000 U rHuPH20)
n=22 Participants
Participants received Treatment A (1000 U CINRYZE with 24,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Treatment B (2000 U CINRYZE + 48000 U rHuPH20)
n=22 Participants
Participants received Treatment B (2000 U CINRYZE with 48,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Cumulative Daily-severity During the Treatment Period
4.63 Score on a scale
Standard Deviation 5.79
2.81 Score on a scale
Standard Deviation 4.42

SECONDARY outcome

Timeframe: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period

Population: ITT-E population

Cumulative symptomatic days was defined as the sum of the symptomatic days of each angioedema attack reported during the treatment period. Participants who were dosed but did not have any attacks in the period were assigned a value of zero. Cumulative symptomatic days was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.

Outcome measures

Outcome measures
Measure
Treatment A (1000 U CINRYZE + 24000 U rHuPH20)
n=22 Participants
Participants received Treatment A (1000 U CINRYZE with 24,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Treatment B (2000 U CINRYZE + 48000 U rHuPH20)
n=22 Participants
Participants received Treatment B (2000 U CINRYZE with 48,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Cumulative Symptomatic Days During the Treatment Period
3.06 days
Standard Deviation 3.51
2.14 days
Standard Deviation 3.30

SECONDARY outcome

Timeframe: From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period

Population: ITT-E population

Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Participants who were dosed but did not have any attacks in the period were assigned a value of zero. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.

Outcome measures

Outcome measures
Measure
Treatment A (1000 U CINRYZE + 24000 U rHuPH20)
n=22 Participants
Participants received Treatment A (1000 U CINRYZE with 24,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Treatment B (2000 U CINRYZE + 48000 U rHuPH20)
n=22 Participants
Participants received Treatment B (2000 U CINRYZE with 48,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Number of Angioedema Attacks Requiring Acute Treatment During the Treatment Period
0.99 angioedema attacks
Standard Deviation 1.51
0.43 angioedema attacks
Standard Deviation 0.89

Adverse Events

Treatment A (1000 U CINRYZE + 24000 U rHuPH20)

Serious events: 0 serious events
Other events: 42 other events
Deaths: 0 deaths

Treatment B (2000 U CINRYZE + 48000 U rHuPH20)

Serious events: 0 serious events
Other events: 46 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Treatment A (1000 U CINRYZE + 24000 U rHuPH20)
n=44 participants at risk
Participants received Treatment A (1000 U CINRYZE with 24,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Treatment B (2000 U CINRYZE + 48000 U rHuPH20)
n=46 participants at risk
Participants received Treatment B (2000 U CINRYZE with 48,000 U rHuPH20 twice weekly \[every 3 or 4 days\] for 8 weeks) as a single 20 mL SC injection per dose in each treatment period.
Injury, poisoning and procedural complications
Contusion
4.5%
2/44 • Number of events 2 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
0.00%
0/46 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
Congenital, familial and genetic disorders
Hereditary angioedema
72.7%
32/44 • Number of events 108 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
60.9%
28/46 • Number of events 69 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
Nervous system disorders
Headache
4.5%
2/44 • Number of events 2 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
6.5%
3/46 • Number of events 3 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
General disorders
Injection site reactions
84.1%
37/44 • Number of events 1113 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
87.0%
40/46 • Number of events 1212 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
General disorders
Injection site extravasation
9.1%
4/44 • Number of events 15 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
19.6%
9/46 • Number of events 32 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
General disorders
Fatigue
9.1%
4/44 • Number of events 5 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
2.2%
1/46 • Number of events 1 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
General disorders
Chest discomfort
4.5%
2/44 • Number of events 2 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
0.00%
0/46 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
General disorders
Injury associated with device
4.5%
2/44 • Number of events 2 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
0.00%
0/46 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
Gastrointestinal disorders
Nausea
4.5%
2/44 • Number of events 2 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
2.2%
1/46 • Number of events 1 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
Musculoskeletal and connective tissue disorders
Muscle spasms
6.8%
3/44 • Number of events 3 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
4.3%
2/46 • Number of events 2 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
Musculoskeletal and connective tissue disorders
Back pain
4.5%
2/44 • Number of events 2 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
0.00%
0/46 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
Musculoskeletal and connective tissue disorders
Pain in extremity
4.5%
2/44 • Number of events 2 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
0.00%
0/46 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
Infections and infestations
Nasopharyngitis
9.1%
4/44 • Number of events 4 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.
2.2%
1/46 • Number of events 1 • From the time of first dose of study drug up to 7 days after the last dose of study drug within each treatment period (8 weeks)
Treatment-emergent adverse events included adverse events (AEs) that were not present at baseline (that is, prior to the first dose of study drug) but started during or after the first administration of study drug in each treatment period, and AEs that were present at baseline but worsened in frequency and/or severity. ITT-S population.

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Results disclosure agreements

  • Principal investigator is a sponsor employee If a multicentre publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicentre Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
  • Publication restrictions are in place

Restriction type: OTHER