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This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema (HAE) patients.
HAE is characterized by recurrent localized angioedema caused by uncontrolled activation of the complement and contact systems due to a congenital deficiency of functional C1 inhibitor.
rhC1INH has been developed to offer a more widely available therapeutic alternative to the existing plasma-derived C1INH (pdC1INH) products that have been used in the treatment of acute angioedema attacks patients with HAE.
Patients who have qualified for enrollment in advance and who present to a study center within 5 hours of onset of an attack will be evaluated for eligibility. 75 eligible patients will be randomized (3:2) to receive an intravenous infusion of rhC1INH or saline in a double-blind fashion. Open-label rhC1INH may be provided as rescue medication to patients who do not experience the beginning of relief within 4 hours or who experience life-threatening oropharyngeal-laryngeal angioedema symptoms.
Any patient having received a randomized treatment will be allowed to receive treatment with rhC1INH in an open-label fashion for subsequent eligible attacks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| rhC1INH | Experimental |
| |
| Placebo (Saline) | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| rhC1INH | Drug | One i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater. |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Beginning of Relief of Symptoms | Time to beginning of relief is the time lapsed from the beginning of the infusion of study medication to the beginning of a beneficial effect based on patient's responses to the Treatmetn Effect Questionnaire (TEQ) for the primary attack location. The beginning of relief is defined as the first timepoint at which
| Patients observed for 24 hours |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Minimal Symptoms | The key secondary efficacy endpoint was the time to minimal symptoms at all locations. The time to achieving minimal symptoms was defined as an answer of "Yes" to TEQ question 3. | 24 hours |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Allergy, Asthma & Immunology, Assoc, Ltd. | Scottsdale | Arizona | 85251 | United States | ||
| Allergy and Asthma Institute of the Valley |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42159953 | Derived | Riedl MA, Narsipur N, Jones D, Tachdjian R, Relan A, Kilvert H, Aiello E, Habimana K, Roskell N, Gough A, Harper JR, Li HH, Harrington A. Post Hoc Analysis of Recombinant C1 Inhibitor Clinical Data Using Contemporary Endpoints for Hereditary Angioedema. Adv Ther. 2026 May 20. doi: 10.1007/s12325-026-03625-0. Online ahead of print. |
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Patients could be enrolled into the open-label phase of the study after the initial randomized treatment.
During the double blind phase of the study, patients were randomized once to receive rhC1INH or Saline in a ratio of 3:2. After the randomized treatment, patiënts with subsequent attacks could be trated with open-label rhC1INH.
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| ID | Title | Description |
|---|---|---|
| FG000 | rhC1INH | rhC1INH: One i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater. |
| FG001 | Placebo (Saline) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Double-blind Phase |
|
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| Placebo (Saline) | Drug | One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment |
|
| Granada Hills |
| California |
| 91344 |
| United States |
| UCLA Department of Medicine Division of Clinical Immunology, David Geffen School of Medicine | Los Angeles | California | 90095 | United States |
| USF Asthma, Allergy and Immunology Clinical Research Unit | Tampa | Florida | 33613 | United States |
| Family Allergy and Asthma Center | Atlanta | Georgia | 30342 | United States |
| Institute for Asthma and Allergy, P.C. | Chevy Chase | Maryland | 20815 | United States |
| Asthma & Allergy Center - Washington University School of Medicine | St Louis | Missouri | 63141 | United States |
| University of Cincinnati Physicians, Inc. | Cincinnati | Ohio | 45267 | United States |
| Optimed Research, LTD | Columbus | Ohio | 43235 | United States |
| Baker Allergy, Asthma and Dermatology Research Center, LLC | Lake Oswego | Oregon | 97035 | United States |
| Pennsylvania State- Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| AARA Research Center | Dallas | Texas | 75231 | United States |
| University of Texas - Medical Branch | Galveston | Texas | 77555 | United States |
| Allergy, Asthma & Immunology Clinic, P.A. | Irving | Texas | 75063 | United States |
| Marycliff Allergy Specialists | Spokane | Washington | 99204 | United States |
| UMHAT "Tsaritsa Yoanna - ISUL"; Clinic of Ear-Nose-Throat Diseases | Sofia | 1527 | Bulgaria |
| Ottawa Allergy Research Corp. | Ottawa | Ontario | KIY 4G2 | Canada |
| Semmelweis University Faculty of Medicine, III Department of Internal Medicine | Budapest | H-1125 | Hungary |
| Allergy, Immunology & Angioedema Center, | Tel Litwinsky | Ramat Gan | 52621 | Israel |
| Bnei-Zion Medical Centre, Clinical Immunology and Allergy Division | Haifa | 31048 | Israel |
| Ospedale Luigi Sacco, Azienda Ospedaliera - Polo Universitario II Divisione di Medicina Interna | Milan | 20157 | Italy |
| P.H.U. Clinic for Dermatology, Medical University Skopje, Unit of Allergology and Clinical Immunology | Skopje | 1000 | North Macedonia |
| Szpital Uniwersytecki w Krakowie, Oddział Kliniczny Klinik Chorób Wewnętrznych, Poradnia Alergologiczna | Krakow | 31-531 | Poland |
| Spitalul Clinic Judeţean Mureş Secţia Clinică Medicină Internă, Compartimentul de Alergologie şi Imunologie | Târgu Mureş | 540103 | Romania |
| Clinic for Immunology and Allergology | Belgrade | 11000 | Serbia |
| Allergy Diagnostic & Clinical Research Unit University of Cape Town Lung Institute | Mowbray | 7700 | South Africa |
| Wits Health Consortium (Pty) Ltd - Wits Donald Gordon Medical Centre | Parktown | 2193 | South Africa |
Placebo (Saline): One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Open Label Phase |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | rhC1INH | rhC1INH: One i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater. |
| BG001 | Placebo (Saline) | Placebo (Saline): One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| 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 | Time to Beginning of Relief of Symptoms | Time to beginning of relief is the time lapsed from the beginning of the infusion of study medication to the beginning of a beneficial effect based on patient's responses to the Treatmetn Effect Questionnaire (TEQ) for the primary attack location. The beginning of relief is defined as the first timepoint at which
| Posted | Median | 95% Confidence Interval | minutes | Patients observed for 24 hours |
|
|
| |||||||||||||||||||||||||||||
| Secondary | Time to Minimal Symptoms | The key secondary efficacy endpoint was the time to minimal symptoms at all locations. The time to achieving minimal symptoms was defined as an answer of "Yes" to TEQ question 3. | Posted | Median | Full Range | minutes | 24 hours |
|
|
From signing of the ICF till 90 days following each treated attack. If multiple attacks were treated, each was followed for 90 days.
In the participants at risk rhC1INH arm group, patients that were randomized to Placebo in the Double Blind phase but received a rescue dose (rhC1INH) are included.
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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 | rhC1INH | rhC1INH: One i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater. | 1 | 56 | 6 | 56 | ||
| EG001 | Placebo (Saline) | Placebo (Saline): One i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment | 0 | 18 | 4 | 18 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal hernia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Vasomotor rhinitis | Respiratory, thoracic and mediastinal disorders | MedDRA | Systematic Assessment |
| |
| Diarrhea | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA | Systematic Assessment |
| |
| Procedural headache | Injury, poisoning and procedural complications | MedDRA | Systematic Assessment |
| |
| Fibrin D-dimer increased | Investigations | MedDRA | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA | Systematic Assessment |
| |
| Lipoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA | Systematic Assessment |
| |
| Skin burning sensation | Skin and subcutaneous tissue disorders | MedDRA | Systematic Assessment |
|
To ensure against inadvertent disclosure of confidential information, the PI will provide Sponsor an opportunity to review any proposed publication/disclosure before it is submitted or otherwise disclosed. The PI will submit proposed manuscript, publication or disclosure to the Sponsor for comment at least 60 days prior to its submission/disclosure. If requested, the publishing party shall remove any Information (other than Study results) prior to submitting or disclosing the materials.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Anurag Relan, Vice President Clinical Research and Medical Affairs | Pharming Technologies | a.relan@pharming.com |
| ID | Term |
|---|---|
| D054179 | Angioedemas, Hereditary |
| D000799 | Angioedema |
| ID | Term |
|---|---|
| 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 |
|---|---|
| D012965 | Sodium Chloride |
| ID | Term |
|---|---|
| D002712 | Chlorides |
| D006851 | Hydrochloric Acid |
| D017606 | Chlorine Compounds |
| D007287 | Inorganic Chemicals |
| D017670 | Sodium Compounds |
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| >=65 years |
|
| Male |
|
|