C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks
LEVP2005-1/Part B: A Double-blind, Placebo-Controlled, Clinical Study to Investigate the Efficacy and Safety of Purified C1 Esterase Inhibitor (Human) as Prophylactic Treatment to Prevent HAE Attacks
1 other identifier
interventional
26
1 country
16
Brief Summary
The study objective was to determine the safety and efficacy of C1INH-nf for the prevention of acute HAE attacks.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Mar 2005
16 active sites
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
Study Start
First participant enrolled
March 14, 2005
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 22, 2007
CompletedStudy Completion
Last participant's last visit for all outcomes
August 22, 2007
CompletedFirst Submitted
Initial submission to the registry
October 29, 2009
CompletedFirst Posted
Study publicly available on registry
November 2, 2009
CompletedResults Posted
Study results publicly available
June 3, 2010
CompletedJune 11, 2021
May 1, 2021
2.4 years
October 29, 2009
March 17, 2010
June 1, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Hereditary Angioedema (HAE) Attacks During Each Prophylactic Therapy Period
An HAE attack was defined as the subject-reported indication of swelling at any location following a report of no swelling on the previous day. Analyses include observed attack counts and normalized attack counts (i.e., the number of attacks observed during each therapy period, normalized for the number of days the subject participated in that period).
12 weeks
Secondary Outcomes (6)
Number of Subject Withdrawals During Each Prophylactic Therapy Period
12 weeks
Average Severity of HAE Attacks During Each Prophylactic Therapy Period
12 weeks
Average Duration of HAE Attacks During Each Prophylactic Therapy Period
12 weeks
Number of Open-label C1INH-nf Infusions Required During Each Prophylactic Therapy Period
12 weeks
Antigenic C1 Inhibitor (C1INH) Serum Levels
Pre-infusion to 1 hour post-infusion at Visit 1 and Weeks 4, 8, and 12
- +1 more secondary outcomes
Other Outcomes (1)
Total Number of Days of Swelling During Each Prophylactic Therapy Period
12 weeks
Study Arms (2)
C1INH-nf First, then Placebo
EXPERIMENTAL1,000 Units (U) of C1INH-nf administered intravenously (IV) every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by matching placebo (saline) administered IV every 3 to 4 days for 12 weeks.
Placebo First, then C1INH-nf
EXPERIMENTALMatching placebo (saline) administered IV every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by 1,000 U of C1INH-nf administered IV every 3 to 4 days for 12 weeks.
Interventions
Eligibility Criteria
You may qualify if:
- Documented HAE
- Normal C1q level
- Relatively frequent angioedema attacks (at least 2 per month on average)
You may not qualify if:
- Low C1q level
- B-cell malignancy
- Presence of anti-C1INH autoantibody
- History of allergic reaction to C1INH or other blood products
- Narcotic addiction
- Current participation in any other investigational drug study or within the past 30 days
- Participation in a C1 esterase inhibitor trial, or received blood or a blood product in the past 90 days
- Pregnancy or lactation
- Any clinically significant medical condition, such as renal failure, that in the opinion of the investigator would interfere with the subject's ability to participate in the study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Shirelead
Study Sites (16)
Allergy and Immunology Associates
Scottsdale, Arizona, 85251, United States
University of California, San Diego
San Diego, California, 92093-0732, United States
Allergy and Asthma Clinical Research, Inc
Walnut Creek, California, 94598, United States
Atlanta Allergy and Asthma Clinic
Suwanee, Georgia, 30024, United States
Hawaii Pacific Health Research Institute
Honolulu, Hawaii, 96826, United States
Welborn Clinic Allergy and Immunology
Evansville, Indiana, 47713, United States
Lake Charles Memorial Hospital
Lake Charles, Louisiana, 70601, United States
Institute for Asthma and Allergy
Wheaton, Maryland, 20902, United States
Libby Clinic
Libby, Montana, 59923, United States
Mount Sinai School of Medicine
New York, New York, 10029, United States
Nationwide Childrens Hospital Clinical Research
Columbus, Ohio, 43205, United States
Allergy Clinic of Tulsa
Tulsa, Oklahoma, 74133, United States
Allergy Asthma and Dermatology Research Center
Lake Oswego, Oregon, 97035, United States
AARA Research Center
Dallas, Texas, 75231, United States
Tyler County Hospital
Woodville, Texas, 75979, United States
St. Joseph's Hospital/Cornerstone Healthcare
Parkersburg, West Virginia, 26101, United States
Related Publications (4)
Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
PMID: 36326435DERIVEDLumry WR, Miller DP, Newcomer S, Fitts D, Dayno J. Quality of life in patients with hereditary angioedema receiving therapy for routine prevention of attacks. Allergy Asthma Proc. 2014 Sep-Oct;35(5):371-6. doi: 10.2500/aap.2014.35.3783.
PMID: 25295804DERIVEDLumry W, Manning ME, Hurewitz DS, Davis-Lorton M, Fitts D, Kalfus IN, Uknis ME. Nanofiltered C1-esterase inhibitor for the acute management and prevention of hereditary angioedema attacks due to C1-inhibitor deficiency in children. J Pediatr. 2013 May;162(5):1017-22.e1-2. doi: 10.1016/j.jpeds.2012.11.030. Epub 2013 Jan 11.
PMID: 23312695DERIVEDZuraw BL, Busse PJ, White M, Jacobs J, Lumry W, Baker J, Craig T, Grant JA, Hurewitz D, Bielory L, Cartwright WE, Koleilat M, Ryan W, Schaefer O, Manning M, Patel P, Bernstein JA, Friedman RA, Wilkinson R, Tanner D, Kohler G, Gunther G, Levy R, McClellan J, Redhead J, Guss D, Heyman E, Blumenstein BA, Kalfus I, Frank MM. Nanofiltered C1 inhibitor concentrate for treatment of hereditary angioedema. N Engl J Med. 2010 Aug 5;363(6):513-22. doi: 10.1056/NEJMoa0805538.
PMID: 20818886DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Shire
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- PREVENTION
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 29, 2009
First Posted
November 2, 2009
Study Start
March 14, 2005
Primary Completion
August 22, 2007
Study Completion
August 22, 2007
Last Updated
June 11, 2021
Results First Posted
June 3, 2010
Record last verified: 2021-05