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| ID | Type | Description | Link |
|---|---|---|---|
| 1453 | Other Identifier | CSL Behring |
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Hereditary angioedema (HAE) is a rare disorder characterized by congenital lack of functional C1 esterase inhibitor. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of involvement of the larynx.The planned extension study is designed to enrol subjects that participated in the pivotal study in order to provide them with C1-INH for treatment of acute HAE attacks for 24 months or until the licensing procedure for C1-INH is finalized, whatever comes first.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| C1 Esterase Inhibitor | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| C1 Esterase Inhibitor | Drug | Lyophilisate containing approximately 500 U C1-INH to be reconstituted with 10 mL water for injection; Single Dose: 20 U/kg b.w. iv |
|
| Measure | Description | Time Frame |
|---|---|---|
| Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population) | The start of symptom relief was determined by subject self-assessment. | Up to 24 h after start of study treatment |
| Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population) | The start of symptom relief was determined by subject self-assessment. | Up to 24 h after start of study treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Complete Resolution of All HAE Symptoms (ITT Subject Population) | Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card. | Up to Day 9 following an attack |
| Time to Complete Resolution of All HAE Symptoms (ITT Attack Population) |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Program Director | CSL Behring | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Contact CSL Behring for facility details | Weston | Florida | 33331 | United States | ||
| Contact CSL Behring for facility details |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 20635155 | Background | Craig TJ, Wasserman RL, Levy RJ, Bewtra AK, Schneider L, Packer F, Yang WH, Keinecke HO, Kiessling PC. Prospective study of rapid relief provided by C1 esterase inhibitor in emergency treatment of acute laryngeal attacks in hereditary angioedema. J Clin Immunol. 2010 Nov;30(6):823-9. doi: 10.1007/s10875-010-9442-1. Epub 2010 Jul 16. | |
| 24661784 |
| Label | URL |
|---|---|
| United States Hereditary Angioedema Association | View source |
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This was a multicenter, open-label extension study enrolling subjects at 15 sites in North America that had participated in study CE1145_3001 (NCT00168103). Enrollment occurred between August 2005 and January 2008.
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| ID | Title | Description |
|---|---|---|
| FG000 | C1 Esterase Inhibitor |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card. |
| Up to Day 9 following an attack |
| Atlanta |
| Georgia |
| 30342 |
| United States |
| Contact CSL Behring for facility details | Idaho Falls | Idaho | 83404 | United States |
| Contact CSL Behring for facility details | Chicago | Illinois | 60612-3244 | United States |
| Contact CSL Behring for facility details | Shreveport | Louisiana | 71130 | United States |
| Contact CSL Behring for facility details | Boston | Massachusetts | 02115 | United States |
| Contact CSL Behring for facility details | Plymouth | Minnesota | 55446 | United States |
| Contact CSL Behring for facility details | Omaha | Nebraska | 69131 | United States |
| Contact CSL Behring for facility details | Tulsa | Oklahoma | 74133 | United States |
| Contact CSL Behring for facility details | Eugene | Oregon | 97401 | United States |
| Contact CSL Behring for facility details | Hershey | Pennsylvania | 17033 | United States |
| Contact CSL Behring for facility details | Rapid City | South Dakota | 57702 | United States |
| Contact CSL Behring for facility details | Dallas | Texas | 75230 | United States |
| Contact CSL Behring for facility details | Ottawa | Ontario | KIY 4G2 | Canada |
| Bernstein JA, Machnig T, Keinecke HO, Whelan GJ, Craig TJ. The effect of weight on the efficacy and safety of C1 esterase inhibitor concentrate for the treatment of acute hereditary angioedema. Clin Ther. 2014 Apr 1;36(4):518-25. doi: 10.1016/j.clinthera.2014.02.005. Epub 2014 Mar 21. |
| 25803207 | Background | Bork K, Craig TJ, Bernstein JA, Feuersenger H, Machnig T, Staubach P. Efficacy of C1 esterase inhibitor concentrate in treatment of cutaneous attacks of hereditary angioedema. Allergy Asthma Proc. 2015 May-Jun;36(3):218-24. doi: 10.2500/aap.2015.36.3844. Epub 2015 Mar 23. |
| 21195947 | Result | Wasserman RL, Levy RJ, Bewtra AK, Hurewitz D, Craig TJ, Kiessling PC, Keinecke HO, Bernstein JA. Prospective study of C1 esterase inhibitor in the treatment of successive acute abdominal and facial hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2011 Jan;106(1):62-8. doi: 10.1016/j.anai.2010.10.012. Epub 2010 Nov 20. |
| 21884533 | Result | Craig TJ, Bewtra AK, Bahna SL, Hurewitz D, Schneider LC, Levy RJ, Moy JN, Offenberger J, Jacobson KW, Yang WH, Eidelman F, Janss G, Packer FR, Rojavin MA, Machnig T, Keinecke HO, Wasserman RL. C1 esterase inhibitor concentrate in 1085 Hereditary Angioedema attacks--final results of the I.M.P.A.C.T.2 study. Allergy. 2011 Dec;66(12):1604-11. doi: 10.1111/j.1398-9995.2011.02702.x. Epub 2011 Sep 2. |
| 23987198 | Derived | Craig TJ, Rojavin MA, Machnig T, Keinecke HO, Bernstein JA. Effect of time to treatment on response to C1 esterase inhibitor concentrate for hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2013 Sep;111(3):211-5. doi: 10.1016/j.anai.2013.06.021. Epub 2013 Jul 16. |
| 22856636 | Derived | Craig TJ, Bewtra AK, Hurewitz D, Levy R, Janss G, Jacobson KW, Packer F, Bernstein JA, Rojavin MA, Machnig T, Keinecke HO, Wasserman RL. Treatment response after repeated administration of C1 esterase inhibitor for successive acute hereditary angioedema attacks. Allergy Asthma Proc. 2012 Jul-Aug;33(4):354-61. doi: 10.2500/aap.2012.33.3589. |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | C1 Esterase Inhibitor |
| 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 of HAE | Type I HAE (common form genotype): An impaired synthesis and an elevated turnover of a normal and functionally active C1-Inhibitor (C1-INH) molecule occurs, causing a reduction in the availability of functionally active C1-INH to levels of 5% to 30% of normal. Type II HAE (variant form genotype): Normal levels of a functionally impaired C1-INH molecule are synthesized while the normal form of C1-INH is considerably reduced in the circulation. | Number | 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 | Time to Start of Relief of Symptoms From HAE Attack (Intent to Treat (ITT) Subject Population) | The start of symptom relief was determined by subject self-assessment. | Intent to treat (ITT) subject population: All subjects admitted to the study who received any portion of the study medication. | Posted | Median | 95% Confidence Interval | hours | Up to 24 h after start of study treatment |
|
|
| |||||||||||||||||||||||||
| Secondary | Time to Complete Resolution of All HAE Symptoms (ITT Subject Population) | Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card. | Intent to treat (ITT) subject population: All subjects admitted to the study who received any portion of the study medication. | Posted | Median | 95% Confidence Interval | hours | Up to Day 9 following an attack |
|
| ||||||||||||||||||||||||||
| Primary | Time to Start of Relief of Symptoms From HAE Attack (ITT Attack Population) | The start of symptom relief was determined by subject self-assessment. | ITT attack population: All attacks in subjects admitted to the study for which any portion of study medication was administered. | Posted | Median | 95% Confidence Interval | hours | Up to 24 h after start of study treatment | HAE Attacks | Participants |
|
| ||||||||||||||||||||||||
| Secondary | Time to Complete Resolution of All HAE Symptoms (ITT Attack Population) | Complete resolution of symptoms was determined by subject self-assessment and documented on a diary card. | ITT attack population: All attacks in subjects admitted to the study for which any portion of study medication was administered. | Posted | Median | 95% Confidence Interval | hours | Up to Day 9 following an attack | HAE Attacks | Participants |
|
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For each HAE attack, the AE reporting period comprised the time period from the subject's enrollment (Day 1) until Day 7 to 9. The reporting period for serious adverse events (SAEs) was 12 Weeks from the time of the first HAE attack.
Adverse events were documented in a diary card. Subjects were treated for 1 to 186 attacks during the study. The safety population consisted of enrolled subjects who received study medication.
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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 | C1 Esterase Inhibitor | 1 | 57 | 25 | 57 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hereditary angioedema | Congenital, familial and genetic disorders | MedDRA 13.0 | Systematic Assessment | A hereditary angioedema attack was reported as an adverse event if it represented a worsening of symptoms during a treated attack. |
|
| Staphylococcal infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hereditary angioedema | Congenital, familial and genetic disorders | MedDRA 13.0 | Systematic Assessment | A hereditary angioedema attack was reported as an adverse event if it represented a worsening of symptoms during a treated attack. |
|
| Conjunctivitis | Eye disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
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| Abdominal distension | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Abdominal tenderness | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
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| Dry mouth | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Local swelling | General disorders | MedDRA 13.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 13.0 | Systematic Assessment |
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| Infusion related reaction | General disorders | MedDRA 13.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Vulvovaginal mycotic infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
| |
| Erythema infectiosum | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
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| Herpes zoster | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
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| Periodontal infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
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| Viral infection | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
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| Gastroenteritis bacterial | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
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| Vaginitis bacterial | Infections and infestations | MedDRA 13.0 | Systematic Assessment |
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| Clavicle fracture | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
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| Hand fracture | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 13.0 | Systematic Assessment |
| |
| Blood potassium decreased | Investigations | MedDRA 13.0 | Systematic Assessment |
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| Red blood cells urine positive | Investigations | MedDRA 13.0 | Systematic Assessment |
| |
| Urine leukocyte esterase positive | Investigations | MedDRA 13.0 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
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| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
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| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 13.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 13.0 | Systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA 13.0 | Systematic Assessment |
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| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 13.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 13.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 13.0 | Systematic Assessment |
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The investigator must provide a copy of any results communication to the sponsor for review at least 30 days prior to public release. The sponsor may request any changes necessary to prevent forfeiture of patent rights to data not in the public domain. For a multi-center study, the investigator must wait (i) at least 1 year after the study is completed at all sites or (ii) until notified by the sponsor that no multi-center publication is planned before seeking publication review.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Program Director | CSL Behring | Use email contact | clinicaltrials@cslbehring.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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| Unknown |
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| Title | Denominators | Categories |
|---|
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