ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneously Administered C1-esterase Inhibitor in the Prevention of Hereditary Angioedema

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02316353
Recruitment Status : Completed
First Posted : December 12, 2014
Last Update Posted : March 20, 2018
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Brief 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.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Types I and II Biological: C1-esterase inhibitor Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 126 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: An Open-label, Randomized Study to Evaluate the Long-term Clinical Safety and Efficacy of Subcutaneous Administration of Human Plasma-derived C1-esterase Inhibitor in the Prophylactic Treatment of Hereditary Angioedema
Actual Study Start Date : December 31, 2014
Actual Primary Completion Date : September 21, 2017
Actual Study Completion Date : September 21, 2017


Arm Intervention/treatment
Experimental: C1-INH - low-volume dose
A low-volume dose of C1-INH will be administered subcutaneously twice a week for up to 52 weeks (up to 146 weeks extension period).
Biological: C1-esterase inhibitor
Experimental: C1-INH - medium-volume dose
A medium-volume dose of C1-INH will be administered subcutaneously twice a week for up to 52 weeks (up to 146 weeks extension period).
Biological: C1-esterase inhibitor



Primary Outcome Measures :
  1. The person-time incidence rates of specified safety events [ Time Frame: During the treatment and extension phases, up to 146 weeks. ]
    The incidence rates of related serious adverse events (SAEs), AEs leading to premature study discontinuation, thromboembolic events, or other specified safety events during treatment with C1-INH


Secondary Outcome Measures :
  1. Percentage of subjects with SAEs or other specified safety events. [ Time Frame: During the treatment and extension phases, up to 146 weeks. ]
  2. Percentage of C1-INH injections resulting in solicited AEs (injection site reactions). [ Time Frame: During the treatment and extension phases, up to 146 weeks. ]
  3. Percentage of subjects with at least 1 solicited AE (injection site reaction). [ Time Frame: During the treatment and extension phases, up to 146 weeks. ]
  4. Percentage of subjects who become seropositive for human immunodeficiency virus, hepatitis B virus, or hepatitis C virus. [ Time Frame: From baseline through the treatment and extension phases, up to 146 weeks. ]
  5. Percentage of subjects who experience < 1 HAE attack per 4-week period. [ Time Frame: During the treatment and extension phases, up to 146 weeks. ]
  6. Percentage of subjects with a ≥ 50% reduction in the time-normalized number of HAE attacks. [ Time Frame: From baseline through the treatment and extension phases, up to 146 weeks. ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02316353


  Show 32 Study Locations
Sponsors and Collaborators
CSL Behring
Investigators
Study Director: Global Clinical Program Director CSL Behring

Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT02316353     History of Changes
Other Study ID Numbers: CSL830_3002
2014-001054-42 ( EudraCT Number )
First Posted: December 12, 2014    Key Record Dates
Last Update Posted: March 20, 2018
Last Verified: October 2017

Additional relevant MeSH terms:
Angioedemas, Hereditary
Hereditary Angioedema Types I and II
Angioedema
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Complement C1s
Complement C1 Inhibitor Protein
Complement C1 Inactivator Proteins
Immunologic Factors
Physiological Effects of Drugs
Complement Inactivating Agents
Immunosuppressive Agents