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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02316353
Other study ID # CSL830_3002
Secondary ID 2014-001054-42
Status Completed
Phase Phase 3
First received
Last updated
Start date December 31, 2014
Est. completion date September 21, 2017

Study information

Verified date October 2017
Source CSL Behring
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The aim of this study is to assess the long-term safety of C1-esterase inhibitor (C1-INH) in preventing hereditary angioedema (HAE) attacks when it is administered under the skin of subjects with HAE. The safety of participating subjects will be assessed for up to 54 weeks. The long-term efficacy of C1-INH will also be assessed. Each eligible subject will enter the treatment phase, wherein subjects will be randomized to treatment with either low- or medium-volume C1-INH. Subjects who have an insufficient treatment response during the study will be given an opportunity to undergo a dose increase. The study aims to enroll eligible subjects who completed study CSL830_3001 (NCT01912456). Subjects who did not participate in study CSL830_3001 may also participate, if eligible and if space permits. Subjects from the United States (US) who complete Treatment Period 2 will be allowed to participate in an Extension Period. During the Extension Period participating US subjects will continue to receive treatment with open-label CSL830 for up to an additional 88 weeks.


Recruitment information / eligibility

Status Completed
Enrollment 126
Est. completion date September 21, 2017
Est. primary completion date September 21, 2017
Accepts healthy volunteers No
Gender All
Age group 6 Years and older
Eligibility Inclusion Criteria:

- Males or females aged 6 years or older.

- A confirmed diagnosis of HAE type I or II.

- HAE attacks over a consecutive 2-month period that required acute treatment, medical attention, or caused significant functional impairment.

- For subjects who have used oral therapy for prophylaxis against HAE attacks within 3 months of first study visit: use of a stable regimen within 3 months of the first study visit.

Exclusion Criteria:

- Incurable malignancies.

- Any clinical condition that will interfere with the evaluation of C1-INH therapy.

- Clinically significant history of poor response to C1-esterase therapy for the management of HAE.

- Suspected or confirmed diagnosis of acquired HAE or HAE with normal C1-INH.

- Inability to have HAE managed pharmacologically with on-demand treatment.

Study Design


Related Conditions & MeSH terms


Intervention

Biological:
C1-esterase inhibitor


Locations

Country Name City State
Australia Study Site Campbelltown New South Wales
Canada Study Site Hamilton Ontario
Canada Study Site Ottawa Ontario
Canada Study Site Quebec
Canada Study Site Toronto Ontario
Czechia Study Site Plzen
Germany Study Site Berlin
Germany Study Site Frankfurt
Germany Study Site Mainz
Germany Study Site Mörfelden-Walldorf Hesse
Hungary Study Site Budapest
Israel Study Site Tel Aviv
Israel Study Site Tel Hashomer
Italy Study Site Catania
Italy Study Site Milano
Romania Study Site Cluj Napoca
Spain Study Site Madrid
Spain Study Site Madrid
Spain Study Site Valencia
United Kingdom Study Site London
United States Study Site Birmingham Alabama
United States Study Site Chevy Chase Maryland
United States Study Site Cincinnati Ohio
United States Study Site Dallas Texas
United States Study Site Hershey Pennsylvania
United States Study Site La Jolla California
United States Study Site Orange California
United States Study Site Portland Oregon
United States Study Site Richmond Virginia
United States Study Site Scottsdale Arizona
United States Study Site Tulsa Oklahoma
United States Study Site Walnut Creek California

Sponsors (1)

Lead Sponsor Collaborator
CSL Behring

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Czechia,  Germany,  Hungary,  Israel,  Italy,  Romania,  Spain,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Person-time Incidence Rates (Subject Based) Subject-based Analysis for Person-Time Incidence Rate = (the total number of subjects who experienced the event during the respective treatment) / (the sum of the date each subject experienced the event - the subject's start date + 1 day) / (365.25 days). The analysis population for this endpoint was the Safety Population: the Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830. Up to 146 weeks.
Primary The Person-time Incidence Rates (Event Based) Event-based Analysis for Person-Time Incidence Rate = (the total number of events documented during the respective treatment) / (the sum of each subject's end date - the subject's start date + 1 day) / (365.25 days). The analysis population for this endpoint was the Safety Population: The Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830. Up to 146 weeks.
Secondary Percentage of Subjects Who Have Solicited Adverse Events (AEs) The number of subjects having at least 1 solicited local AE during a treatment were divided by the number of subjects in the corresponding treatment. The analysis population for this endpoint was the Safety Population: The Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830. Up to 146 weeks
Secondary Percentage of Injections Followed by At Least One Solicited Adverse Event The percent of injections followed by at least one solicited adverse event. The analysis population for this endpoint was the Safety Population: The Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830. This was assessed across all participants, calculated as total number of events following injections / total number of injections across all participants, in each Arm. Up to 146 weeks
Secondary Percentage of Subjects Who Become Seropositive for Human Immunodeficiency Virus (HIV-1/-2), Hepatitis B Virus, or Hepatitis C Virus. Blood samples to be tested for HIV-1/-2, HBV, and HCV. The analysis population for this endpoint was the Safety Population: The Safety Population comprised all subjects who provided informed consent / assent, who were randomized, and who received at least 1 dose or a partial dose of CSL830. Up to 146 weeks
Secondary Percentage of Subjects Who Experience a Time-normalized HAE Attack Frequency of <1 HAE Attack Per 4-Week Period The percentage of subjects with a time-normalized merged HAE attack frequency of <1 HAE attack per 4-week period. The analysis population for this endpoint was the Intent-to-Treat (ITT) Population: The ITT Population comprised all subjects who provided informed consent / assent and were randomized, regardless of whether or not they received CSL 830. Up to 146 weeks
Secondary Percentage of Subjects Who Are Responders A responder was defined as a subject with a = 50% reduction in the time-normalized number of HAE attacks on CSL830 relative to the time-normalized number of HAE attacks used to qualify for participation in the current study. The analysis population for this endpoint was the Intent-to-Treat (ITT) Population: The ITT Population comprised all subjects who provided informed consent / assent and were randomized, regardless of whether or not they received CSL830. Not all subjects in the ITT were available for this outcome measure. Up to 146 weeks
See also
  Status Clinical Trial Phase
Completed NCT01467947 - Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert Phase 4
Completed NCT01912456 - A Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema Phase 3
Completed NCT01760343 - A Study to Evaluate the Safety and Pharmacokinetics of Two Formulations of C1-esterase Inhibitor Phase 1
Completed NCT04618211 - Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Phase 2
Recruiting NCT06343779 - Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema Phase 3
Recruiting NCT05396105 - Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Phase 2/Phase 3
Withdrawn NCT01832896 - Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema Phase 2
Completed NCT01576523 - A Study to Evaluate the Clinical Pharmacology and Safety of C1-esterase Inhibitor Administered by the Subcutaneous Route Phase 1/Phase 2
Active, not recruiting NCT05047185 - Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II Phase 2