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Hereditary angioedema ("HAE") is a disease characterized by recurrent tissue swelling affecting various body locations. Recent literature shows that patients with frequent attacks may benefit from long-term prophylaxis. This study aims to evaluate the safety and prophylactic effect of weekly administrations of 50 IU/kg recombinant C1 Inhibitor ("rhC1INH").
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Recombinant Human C1 Inhibitor | Other | Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Recombinant Human C1 Inhibitor | Drug | 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period. |
|
| Measure | Description | Time Frame |
|---|---|---|
| HAE Attacks/Week | Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week). | 8 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters. | "PK/PD" parameters will be based on concentration time curves after the 1st and 8th "rhC1INH" administration.(ratio visit 8/ visit 1, based on the area under the curve from baseline up to 4 hours after administration (AUC 0-4) | 8 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Bruno Giannetti, MD | COO Pharming | Study Chair |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| For information on sites, please contact Pharming Technologies | Leiden | Netherlands | ||||
| Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department |
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Patients with a history of frequent attacks, defined as "HAE" attacks occurring at least every two weeks were included in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Recombinant Human C1 Inhibitor | Weekly administration of 50 IU/kg recombinant human C1 inhibitor Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Recombinant Human C1 Inhibitor | Weekly administration of 50 IU/kg recombinant human C1 inhibitor Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period. |
| 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 |
| 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 | HAE Attacks/Week | Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week). | Posted | Mean | 95% Confidence Interval | attacks/week | 8 weeks |
|
Treatment emergent adverse events ("TEAEs") were defined as events with start from onset of study drug administration. All Serious Adverse Events("SAEs")are listed.
All Adverse Events ("AEs")occurring within 7 days of onset after study drug administration have been listed.
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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 | Recombinant Human C1 Inhibitor | Weekly administration of 50 IU/kg recombinant human C1 inhibitor Recombinant Human C1 Inhibitor : 50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Appendicitis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia-mild | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Affairs Department | Pharming Technologies BV | +31715247400 | medicalinfo@pharming.com |
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| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D054179 | Angioedemas, Hereditary |
| ID | Term |
|---|---|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000799 | Angioedema |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
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| ID | Term |
|---|---|
| D050718 | Complement C1 Inhibitor Protein |
| C571093 | conestat alfa |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D003174 | Complement C1 Inactivator Proteins |
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|
| Târgu Mureş |
| 541103 |
| Romania |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Units | Counts |
|---|
| Participants |
|
|
| Secondary | The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters. | "PK/PD" parameters will be based on concentration time curves after the 1st and 8th "rhC1INH" administration.(ratio visit 8/ visit 1, based on the area under the curve from baseline up to 4 hours after administration (AUC 0-4) | Posted | Geometric Mean | 95% Confidence Interval | ratio | 8 weeks |
|
|
|
| 2 |
| 25 |
| 2 |
| 25 |
| Laryngeal edema | Respiratory, thoracic and mediastinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Anaemia-moderate | Blood and lymphatic system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
The investigator must provide a copy of any results communication to the sponsor for review at least 30 days prior to its submission or disclosure. The sponsor may request to delete information identified by sponsor as confidential information prior to submitting such manuscript and/or abstract for publication. For a multi-center study, the investigator must wait (i) at least 24 months after the study is completed at all sites or (ii) until after the multi-center publication.
| D000081208 | Hereditary Complement Deficiency Diseases |
| D000081207 | Primary Immunodeficiency Diseases |
| 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 |
| 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 |
| Title | Measurements |
|---|---|
|