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The study objective was to evaluate the safety and efficacy of prophylactic use of C1INH-nf for the prevention of acute HAE attacks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open-label C1INH-nf | Experimental | 1,000 Units (U) of C1INH-nf administered intravenously (IV) every 3 to 7 days. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| C1 esterase inhibitor [human] (C1INH-nf) | Biological |
|
| Measure | Description | Time Frame |
|---|---|---|
| Frequency of All HAE Attacks | A hereditary angioedema (HAE) attack was defined as a discrete episode during which the subject progressed from no angioedema to symptoms of angioedema. | Duration of the study |
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| Measure | Description | Time Frame |
|---|---|---|
| Antigenic C1 Inhibitor (C1INH) Serum Levels | Change from pre-infusion to 1 hour post-infusion in antigenic C1INH serum levels. | Pre-infusion to 1 hour post-infusion |
| Functional C1INH Serum Levels | Change from pre-infusion to 1 hour post-infusion in functional C1INH serum levels. Functional C1INH serum levels are expressed as a percent of total detectable C1INH (i.e., functional C1INH/total detectable C1INH). |
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:
Were not enrolled in LEVP2005-1 after enrollment in LEVP2005-1 was closed, under the following circumstances:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Clinical Research Consultants, Inc | Hoover | Alabama | 35216 | United States | ||
| Allergy and Immunology Associates |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 36326435 | Derived | 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. | |
| 23484892 | Derived | 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. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Open-label C1INH-nf | 1,000 Units (U) of C1 esterase inhibitor (C1INH-nf) administered intravenously (IV) every 3 to 7 days. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Pre-infusion to 1 hour post-infusion |
| Complement C4 Serum Levels | Change from pre-infusion to 1 hour post-infusion in complement C4 serum levels. | Pre-infusion to 1 hour post-infusion |
| Scottsdale |
| Arizona |
| 85251 |
| United States |
| Allergy and Asthma Clinic of Northwest Arkansas | Bentonville | Arkansas | 72712 | United States |
| UCLA-David Geffen School of Medicine | Los Angeles | California | 90095 | United States |
| University of California, San Diego | San Diego | California | 92093 | United States |
| Allergy and Asthma Associates of Santa Clara | San Jose | California | 95117 | United States |
| Allergy and Asthma Clinical Research, Inc | Walnut Creek | California | 94598 | United States |
| Allergy and Asthma Center | Fort Lauderdale | Florida | 33334 | United States |
| Allergy and Asthma Center of East Orlando | Orlando | Florida | 32826 | United States |
| Cleveland Clinic | Weston | Florida | 33331 | United States |
| Family Allergy and Asthma Center | Atlanta | Georgia | 30342 | United States |
| Atlanta Allergy and Asthma Clinic | Suwanee | Georgia | 30024 | United States |
| University Consultants in Allergy & Immunology | Chicago | Illinois | 60612 | United States |
| Welborn Clinic | Evansville | Indiana | 47713 | United States |
| Private Practice | Liberal | Kansas | 67901 | United States |
| Olathe Medical Center | Olathe | Kansas | 66061 | United States |
| Graves-Gilbert Clinic | Bowling Green | Kentucky | 42101 | United States |
| Family Asthma and Allergy Research Center | Louisville | Kentucky | 40215 | United States |
| Asthma Allergy and Sinus Center | Waldorf | Maryland | 20602 | United States |
| Institute for Asthma and Allergy | Wheaton | Maryland | 20902 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Grand Traverse Allergy | Traverse City | Michigan | 49684 | United States |
| MeritCare Clinical Research | Bemidji | Minnesota | 56601 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| St. Louis University School of Medicine | St Louis | Missouri | 63110 | United States |
| Nevada Access to Research and Education Society | Las Vegas | Nevada | 89102 | United States |
| UMDNJ Asthma and Allergy Research Center | Newark | New Jersey | 07103 | United States |
| Private Practice | Hastings-on-Hudson | New York | 10706 | United States |
| Winthrop University Hospital | Mineola | New York | 11501 | United States |
| Mount Sinai School of Medicine | New York | New York | 10029 | United States |
| Montefiore Medical Center | The Bronx | New York | 10461 | United States |
| Allergy Partners of East Carolina | Greenville | North Carolina | 27858 | United States |
| Legacy Pharma Research | Bismarck | North Dakota | 58501 | United States |
| MeritCare Clinical Research | Fargo | North Dakota | 58122 | United States |
| Nationwide Childrens Hospital Clinical Research | Columbus | Ohio | 43205 | United States |
| Allergy Clinic of Tulsa | Tulsa | Oklahoma | 74133 | United States |
| Oregon Medical Group | Eugene | Oregon | 97401 | United States |
| Allergy Asthma and Dermatology Research Center | Lake Oswego | Oregon | 97035 | United States |
| Penn State University | Hershey | Pennsylvania | 17033 | United States |
| AARA Research Center | Dallas | Texas | 75231 | United States |
| University of Texas Medical Branch | Galveston | Texas | 77555-1083 | United States |
| University of Texas - Pediatric Pulmonary/Allergy and Immunology | Houston | Texas | 77030 | United States |
| Allergy and Asthma Research Center | San Antonio | Texas | 78229 | United States |
| Tyler County Hospital | Woodville | Texas | 75979 | United States |
| Virginia Adult and Pediatric Allergy and Asthma | Richmond | Virginia | 23229 | United States |
| Marycliff Allergy Specialists | Spokane | Washington | 99204 | United States |
| Puget Sound Allergy, Asthma and Immunology | Tacoma | Washington | 98405 | United States |
| St. Joseph's Hospital/Cornerstone Healthcare | Parkersburg | West Virginia | 26102 | United States |
| 23312695 | Derived | 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. |
| 20818886 | Derived | 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. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Open-label C1INH-nf | 1,000 U of C1INH-nf administered IV every 3 to 7 days. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Frequency of All HAE Attacks | A hereditary angioedema (HAE) attack was defined as a discrete episode during which the subject progressed from no angioedema to symptoms of angioedema. | Intent-to-treat Efficacy (ITT-E) Population (N=146; the number of subjects who received at least one prophylactic dose of C1INH-nf for the prevention of HAE attacks). HAE attack frequency was reported by 137 subjects at screening (i.e., data were missing for 9 subjects). | Posted | Median | Full Range | attacks per month | Duration of the study |
|
|
| ||||||||||||||||||||||||||||||||
| Other Pre-specified | Antigenic C1 Inhibitor (C1INH) Serum Levels | Change from pre-infusion to 1 hour post-infusion in antigenic C1INH serum levels. | ITT-E subjects with data at both sampling time points (N=137). | Posted | Mean | Standard Deviation | mg/dL | Pre-infusion to 1 hour post-infusion |
|
| |||||||||||||||||||||||||||||||||
| Other Pre-specified | Functional C1INH Serum Levels | Change from pre-infusion to 1 hour post-infusion in functional C1INH serum levels. Functional C1INH serum levels are expressed as a percent of total detectable C1INH (i.e., functional C1INH/total detectable C1INH). | ITT-E subjects with data at both sampling time points (N=132). | Posted | Mean | Standard Deviation | percent | Pre-infusion to 1 hour post-infusion |
|
| |||||||||||||||||||||||||||||||||
| Other Pre-specified | Complement C4 Serum Levels | Change from pre-infusion to 1 hour post-infusion in complement C4 serum levels. | ITT-E subjects with data at both sampling time points (N=134). | Posted | Mean | Standard Deviation | mg/dL | Pre-infusion to 1 hour post-infusion |
|
|
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Presented are treatment-emergent adverse reactions considered to be related to C1INH-nf. There were no serious adverse reactions considered related to C1INH-nf.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Open-label C1INH-nf | 1,000 U of C1INH-nf administered IV every 3 to 7 days. | 0 | 146 | 9 | 146 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | MedDRA (9.0) |
| ||
| Headache | Nervous system disorders | MedDRA (9.0) |
| ||
| Rash | Skin and subcutaneous tissue disorders | MedDRA (9.0) |
| ||
| Phlebitis | Vascular disorders | MedDRA (9.0) |
|
Clinical Study Agreement. Most restrictive provision - PI will not publish results until after first of: multicenter publication is published or 24 months from study end. Thereafter, PI may publish his results. PI must provide copy of proposed publication to sponsor for pre-review. If sponsor requests, PI must delete sponsor confidential information before publication and/or delay publication for 90 days so sponsor can file for patents or take other action to protect its patent rights.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
| ID | Term |
|---|---|
| D054179 | Angioedemas, Hereditary |
| ID | Term |
|---|---|
| D000799 | Angioedema |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D000081208 | Hereditary Complement Deficiency Diseases |
| D000081207 | Primary Immunodeficiency Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014581 | Urticaria |
| D017445 | Skin Diseases, Vascular |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D007153 | Immunologic Deficiency Syndromes |
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| ID | Term |
|---|---|
| D050718 | Complement C1 Inhibitor Protein |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D003174 | Complement C1 Inactivator Proteins |
| D015843 | Serpins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D003169 | Complement Inactivator Proteins |
| D003165 | Complement System Proteins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
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| Title | Denominators | Categories |
|---|
| Pre-infusion |
| |||||
| Increase at 1 hour post-infusion |
|
|
| Title | Denominators | Categories |
|---|
| Pre-infusion |
| |||||
| Change at 1 hour post-infusion |
|