Clinical trial NCT00462709: Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks
Official title: LEVP2006-4 CHANGE 3 Trial (C1-Inhibitor in Hereditary Angioedema Nanofiltration Generation Evaluating Efficacy): Open-Label Use of C1INH-nf (Human) for the Prophylactic Treatment to Prevent HAE Attacks and as Treatment in Acute HAE Attacks
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Hereditary Angioedema
Keywords: Hereditary angioedema
C1 esterase inhibitor (human)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2007-04-19
Enrollment: 146
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

Brief summary: The study objective was to evaluate the safety and efficacy of prophylactic use of C1INH-nf for the prevention of acute HAE attacks.


Eligibility criteria: Inclusion Criteria:

- History of at least 1 HAE attack per month or any history of laryngeal edema

In addition, this study was open to all subjects who:

* Completed participation in LEVP2005-1/B (NCT01005888) any time after the final prophylactic therapy in Part B
* Were enrolled but not randomized in LEVP2005-1/A (NCT00289211) after Part A was closed
* Were enrolled and randomized in LEVP2005-1/A after LEVP2005-1/B was closed to enrollment, any time after the 3-day telephone follow-up
* Were excluded from LEVP2005-1 for any of the following reasons:

  * Pregnancy or lactation
  * Age less than 6 years
  * Narcotic addiction
  * Presence of anti-C1 inhibitor (C1INH) autoantibodies
* Were not enrolled in LEVP2005-1 after enrollment in LEVP2005-1 was closed, under the following circumstances:

  * Had a diagnosis of HAE: evidence of a low C4 level plus either a low C1INH antigenic level or a low C1INH functional level, or
  * Had a known HAE-causing C1INH mutation, or
  * Had a diagnosis of HAE based on a strong family history of HAE as determined by the principal investigator

Exclusion Criteria:

* History of allergic reaction to C1INH or other blood products
* Participated in any other investigational drug study within the past 30 days other than those sponsored by Lev Pharmaceuticals
* Received blood or a blood product in the past 60 days other than C1INH-nf

Primary outcomes:
measure: Frequency of All HAE Attacks
timeFrame: Duration of the study
Secondary outcomes:
Other outcomes:
measure: Antigenic C1 Inhibitor (C1INH) Serum Levels
timeFrame: Pre-infusion to 1 hour post-infusion
measure: Functional C1INH Serum Levels
timeFrame: Pre-infusion to 1 hour post-infusion
measure: Complement C4 Serum Levels
timeFrame: Pre-infusion to 1 hour post-infusion
Locations:
city: Hoover
country: United States
facility: Clinical Research Consultants, Inc
state: Alabama
city: Scottsdale
country: United States
facility: Allergy and Immunology Associates
state: Arizona
city: Bentonville
country: United States
facility: Allergy and Asthma Clinic of Northwest Arkansas
state: Arkansas
city: Los Angeles
country: United States
facility: UCLA-David Geffen School of Medicine
state: California
city: San Diego
country: United States
facility: University of California, San Diego
state: California
city: San Jose
country: United States
facility: Allergy and Asthma Associates of Santa Clara
state: California
city: Walnut Creek
country: United States
facility: Allergy and Asthma Clinical Research, Inc
state: California
city: Fort Lauderdale
country: United States
facility: Allergy and Asthma Center
state: Florida
city: Orlando
country: United States
facility: Allergy and Asthma Center of East Orlando
state: Florida
city: Weston
country: United States
facility: Cleveland Clinic
state: Florida
city: Atlanta
country: United States
facility: Family Allergy and Asthma Center
state: Georgia
city: Suwanee
country: United States
facility: Atlanta Allergy and Asthma Clinic
state: Georgia
city: Chicago
country: United States
facility: University Consultants in Allergy & Immunology
state: Illinois
city: Evansville
country: United States
facility: Welborn Clinic
state: Indiana
city: Liberal
country: United States
facility: Private Practice
state: Kansas
city: Olathe
country: United States
facility: Olathe Medical Center
state: Kansas
city: Bowling Green
country: United States
facility: Graves-Gilbert Clinic
state: Kentucky
city: Louisville
country: United States
facility: Family Asthma and Allergy Research Center
state: Kentucky
city: Waldorf
country: United States
facility: Asthma Allergy and Sinus Center
state: Maryland
city: Wheaton
country: United States
facility: Institute for Asthma and Allergy
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Traverse City
country: United States
facility: Grand Traverse Allergy
state: Michigan
city: Bemidji
country: United States
facility: MeritCare Clinical Research
state: Minnesota
city: Jackson
country: United States
facility: University of Mississippi Medical Center
state: Mississippi
city: Saint Louis
country: United States
facility: St. Louis University School of Medicine
state: Missouri
city: Las Vegas
country: United States
facility: Nevada Access to Research and Education Society
state: Nevada
city: Newark
country: United States
facility: UMDNJ Asthma and Allergy Research Center
state: New Jersey
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Hastings-on-Hudson
country: United States
facility: Private Practice
state: New York
city: Mineola
country: United States
facility: Winthrop University Hospital
state: New York
city: New York
country: United States
facility: Mount Sinai School of Medicine
state: New York
city: Greenville
country: United States
facility: Allergy Partners of East Carolina
state: North Carolina
city: Bismarck
country: United States
facility: Legacy Pharma Research
state: North Dakota
city: Fargo
country: United States
facility: MeritCare Clinical Research
state: North Dakota
city: Columbus
country: United States
facility: Nationwide Childrens Hospital Clinical Research
state: Ohio
city: Tulsa
country: United States
facility: Allergy Clinic of Tulsa
state: Oklahoma
city: Eugene
country: United States
facility: Oregon Medical Group
state: Oregon
city: Lake Oswego
country: United States
facility: Allergy Asthma and Dermatology Research Center
state: Oregon
city: Hershey
country: United States
facility: Penn State University
state: Pennsylvania
city: Dallas
country: United States
facility: AARA Research Center
state: Texas
city: Galveston
country: United States
facility: University of Texas Medical Branch
state: Texas
city: Houston
country: United States
facility: University of Texas - Pediatric Pulmonary/Allergy and Immunology
state: Texas
city: San Antonio
country: United States
facility: Allergy and Asthma Research Center
state: Texas
city: Woodville
country: United States
facility: Tyler County Hospital
state: Texas
city: Richmond
country: United States
facility: Virginia Adult and Pediatric Allergy and Asthma
state: Virginia
city: Spokane
country: United States
facility: Marycliff Allergy Specialists
state: Washington
city: Tacoma
country: United States
facility: Puget Sound Allergy, Asthma and Immunology
state: Washington
city: Parkersburg
country: United States
facility: St. Joseph's Hospital/Cornerstone Healthcare
state: West Virginia
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: 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.
citation: Baker JW, Craig TJ, Riedl MA, Banerji A, Fitts D, Kalfus IN, Uknis ME. Nanofiltered C1 esterase inhibitor (human) for hereditary angioedema attacks in pregnant women. Allergy Asthma Proc. 2013 Mar-Apr;34(2):162-9. doi: 10.2500/aap.2013.34.3645.
citation: Lumry 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.
citation: Zuraw 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.