NCT01756157|CompletedPhase 2Results

Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema Attacks

A Phase 2, Randomized, Double-Blind, Multicenter, Dose-Ranging, Crossover Study to Evaluate the Safety and Efficacy of Subcutaneous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) With Recombinant Human Hyaluronidase (rHuPH20) for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

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2 other identifiers

0624-2062012-000083-24

Study Type

interventional

Target

Locations

4 countries

Sites

Timeline

RegisteredDec 2012

StartedFeb 2013

CompletionSep 2013

Brief Summary

The primary objectives of the study are to evaluate the safety, tolerability, and efficacy of two doses of CINRYZE with recombinant human hyaluronidase (rHuPH20) administered by subcutaneous (SC) injection to prevent angioedema attacks.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at P25-P50 for phase_2

Timeline

Completed

Started Feb 2013

Shorter than P25 for phase_2

Geographic Reach

4 countries

23 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

7 months study duration

First Submitted

Initial submission to the registry

June 29, 2012

Completed

6 months until next milestone

First Posted

Study publicly available on registry

December 25, 2012

Completed

1 month until next milestone

Study Start

First participant enrolled

February 4, 2013

Completed

7 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 13, 2013

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 13, 2013

Completed

1.7 years until next milestone

Results Posted

Study results publicly available

June 12, 2015

Completed

Last Updated

June 3, 2021

Status Verified

May 1, 2021

Enrollment Period

7 months

First QC Date

June 29, 2012

Results QC Date

May 29, 2015

Last Update Submit

May 11, 2021

Conditions

Hereditary Angioedema (HAE)

Keywords

CrossoverC1 esterase inhibitorPreventionC1 inhibitorHereditary AngioedemaSubcutaneousRecombinant human hyaluronidase

Outcome Measures

Primary Outcomes (1)

Normalized Number of Angioedema Attacks During the Treatment Period
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.
From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period

Secondary Outcomes (4)

Cumulative Attack-severity During the Treatment Period
From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
Cumulative Daily-severity During the Treatment Period
From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
Cumulative Symptomatic Days During the Treatment Period
From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
Number of Angioedema Attacks Requiring Acute Treatment During the Treatment Period
From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period

Study Arms (2)

SC CINRYZE with rHuPH20 Dose Level 1 followed by Dose Level 2

EXPERIMENTAL

SC CINRYZE with rHuPH20 Dose Level 1 twice weekly (every 3 or 4 days) for 8 weeks followed by SC CINRYZE with rHuPH20 Dose Level 2 twice weekly (every 3 or 4 days) for 8 weeks.

Biological: CINRYZE with rHuPH20

SC CINRYZE with rHuPH20 Dose Level 2 followed by Dose Level 1

EXPERIMENTAL

SC CINRYZE with rHuPH20 Dose Level 2 twice weekly (every 3 or 4 days) for 8 weeks followed by SC CINRYZE with rHuPH20 Dose Level 1 twice weekly (every 3 or 4 days) for 8 weeks.

Biological: CINRYZE with rHuPH20

Interventions

CINRYZE with rHuPH20BIOLOGICAL

Also known as: C1 esterase inhibitor (human), Recombinant human hyaluronidase

SC CINRYZE with rHuPH20 Dose Level 1 followed by Dose Level 2SC CINRYZE with rHuPH20 Dose Level 2 followed by Dose Level 1

Eligibility Criteria

Age12 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Be ≥12 years of age.
Have a confirmed diagnosis of Hereditary Angioedema.

You may not qualify if:

Receipt of any C1 inhibitor (C1 INH) therapy or any blood products for treatment or prevention of an angioedema attack within 7 days before the first dose of study drug.
Be receiving prophylactic intravenous CINRYZE that exceeds 1000 units every 3 or 4 days (maximum weekly dose 2000 units).
Have received any androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 7 days prior to the first dose of study drug.
If female, have started taking or changed the dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progestin containing products) within 3 months prior to the first dose of study drug.
History of allergic reaction to C1 INH products, including CINRYZE or other blood products.
History of abnormal blood clotting.
Have a known allergy to hyaluronidase or any other ingredient in the study formulation.

Contact the study team to confirm eligibility.