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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-002453-29 | EudraCT Number | ||
| SHP616-301 | Other Identifier | Shire |
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Primary Objective - To assess the relative efficacy of two dose levels of CINRYZE (500 Units and 1000 Units) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of age with hereditary angioedema (HAE).
Secondary Objectives - To assess the safety and tolerability, characterize the pharmacokinetics (PK) and pharmacodynamics (PD), and assess the immunogenicity of two dose levels of CINRYZE administered by IV injection in children 6 to 11 years of age with HAE.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 500/1000 | Experimental | 500 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks |
|
| 1000/500 | Experimental | 1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 500 Units of CINRYZE administered by IV injection twice per week for 12 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CINRYZE 500 | Biological | 500 Units of CINRYZE administered by IV injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Normalized Number of Angioedema Attacks Per Month in a Treatment Period | Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency. | From start of treatment up to 12 weeks during each treatment period |
| Measure | Description | Time Frame |
|---|---|---|
| Cumulative Attack-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period | Severity of the angioedema attack sign/symptom was characterized as None: no symptom; Mild: noticeable symptom but easily tolerated by the participant and did not interfere with routine activities; Moderate: symptom interfered with the participant's ability to attend school or participate in family life and social/recreational activities; Severe: symptom significantly limited the participant's ability to attend school or participate in family life and social/recreational activities. Symptom severity score was assigned as Mild = 1, Moderate = 2 and Severe = 3. Cumulative attack severity score was the sum of the maximum symptom severity scores recorded for each angioedema attack in a treatment period. Cumulative attack-severity score normalized per month [(raw score/number of days of participation in that treatment period)*30.4] was reported here. Cumulative attack-severity score normalized per month ranged from 0 to 10.4 and higher scores represent worse symptoms. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Asthma and Allergy Associates, P.C | Colorado Springs | Colorado | 80907 | United States | ||
| Nevada Access to Research and Education Society |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28662509 | Result | Aygoren-Pursun E, Soteres D, Moldovan D, Christensen J, Van Leerberghe A, Hao J, Schranz J, Jacobson KW, Martinez-Saguer I. Preventing Hereditary Angioedema Attacks in Children Using Cinryze(R): Interim Efficacy and Safety Phase 3 Findings. Int Arch Allergy Immunol. 2017;173(2):114-119. doi: 10.1159/000477541. Epub 2017 Jun 30. | |
| 36326435 |
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Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
A total of 16 participants were screened and of them, 12 were enrolled into the baseline observational period (12 weeks) and were randomized to receive the treatment in sequence A-B and B-A during this crossover study without a washout period.
The study was conducted in 10 study centers in the United States, European Union, Mexico, and Israel between 20 March 2014 (first participant first visit) and 04 May 2017 (last participant last visit).
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment A-B (500 U/1000 U CINRYZE) | Participants received 500 units (U) of CINRYZE intravenous (IV) injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment A) during intervention period 1 followed by 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment B) during intervention period 2. There was no washout period between the two intervention periods. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Intervention Period 1 |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 26, 2015 | Apr 24, 2018 |
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| CINRYZE 1000 | Biological | 1000 Units of CINRYZE administered by IV injection |
|
| From start of treatment up to 12 weeks during each treatment period |
| Cumulative Daily-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period | Severity of the angioedema attack sign/symptom was characterized as None: no symptom; Mild: noticeable but easily tolerated by the participant and did not interfere with routine activities; Moderate: interfered with the participant's ability to attend school or participate in family life and social/recreational activities; Severe: significantly limited the participant's ability to attend school or participate in family life and social/recreational activities. Symptom severity score was assigned as Mild = 1, Moderate = 2 and Severe = 3. Cumulative daily-severity score was the sum of the severity scores recorded for every day of reported symptoms in a treatment period. Cumulative daily-severity score normalized per month [(raw score/number of days of participation in that treatment period)*30.4] was reported here. Cumulative daily-severity score normalized per month ranged from 0 to 15.6 and higher scores represent worse symptoms. | From start of treatment up to 12 weeks during each intervention period |
| Normalized Number of Angioedema Attacks Per Month Requiring Acute Treatment in a Treatment Period | Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks requiring acute treatment was normalized for the number of days participants participated in a given period and expressed as the monthly frequency. | From start of treatment up to 12 weeks during each intervention period |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Dose Group | An adverse event (AE) was any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurring in a participant participating in a clinical study with the sponsor's product, regardless of causal relationship. TEAEs were defined as events that started or worsened on or after the date and time of the first dose of investigational product and up to 7 days after the last dose of investigational product. | From start of study treatment up to 25 weeks |
| Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen | C1 INH antigen concentration in plasma was determined using an automated nephelometric assay. | Pre-dose and 1 hour (h) post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention period |
| C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma | The functional activity of C1 INH in plasma samples was determined by a chromogenic assay. | Pre-dose and 1 h post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention period |
| Plasma Concentration of Complement C4 | Concentration of Complement C4 in plasma was determined using an automated nephelometric assay. | Pre-dose and 1 h post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention period |
| Number of Participants With C1 Esterase Inhibitor (C1 INH) Antibodies in Plasma | The presence of C1 INH antibodies in plasma samples was determined using a proprietary enzyme-linked-immunosorbent-assay. Number of participants with C1 INH Antibodies was reported. | Pre-dose, 1 week post treatment (Week 13, Week 25) and 1 month post treatment follow-up (Week 28) |
| Las Vegas |
| Nevada |
| 89106 |
| United States |
| Oregon Allergy Associates | Eugene | Oregon | 97401 | United States |
| Klinikum der J.W. Goethe Universitat | Frankfurt | 60590 | Germany |
| HZRM Hamophilie Zentrum Rhein Main GmbH | Mörfelden-Walldorf | 64546 | Germany |
| Tel Aviv Sourasky Medical Center | Tel Aviv | 64239 | Israel |
| Instituto Nacional de Pediatria | Mexico City | 04530 | Mexico |
| Clinical County Hospital Mures | Târgu Mureş | 540072 | Romania |
| 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. |
| 30968444 | Derived | Aygoren-Pursun E, Soteres DF, Nieto-Martinez SA, Christensen J, Jacobson KW, Moldovan D, Van Leerberghe A, Tang Y, Lu P, Vardi M, Schranz J, Martinez-Saguer I. A randomized trial of human C1 inhibitor prophylaxis in children with hereditary angioedema. Pediatr Allergy Immunol. 2019 Aug;30(5):553-561. doi: 10.1111/pai.13060. Epub 2019 May 29. |
| FG001 | Treatment B-A (1000 U/500 U CINRYZE) | Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment B) during intervention period 1 followed by 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment A) during intervention period 2. There was no washout period between the two intervention periods. |
| COMPLETED |
|
| NOT COMPLETED |
|
| Intervention Period 2 |
|
Safety set included all participants who took at least 1 dose of investigational product.
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| ID | Title | Description |
|---|---|---|
| BG000 | Treatment A-B (500 U/1000 U CINRYZE) | Participants received 500 units (U) of CINRYZE intravenous (IV) injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment A) in Intervention period 1 followed by 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment B) in Intervention period 2. There was no washout period between two intervention periods. |
| BG001 | Treatment B-A (1000 U/500 U CINRYZE) | Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment B) in Intervention period 1 followed by 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 12 weeks (Treatment A) in Intervention period 2. There was no washout period between two intervention periods. |
| 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, Continuous | Age was calculated as the difference between date of birth and date of informed consent, truncated to years. | Mean | Standard Deviation | Years |
| ||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | 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 | Normalized Number of Angioedema Attacks Per Month in a Treatment Period | Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency. | Full Analysis Set (FAS) included all participants in the safety set who had at least 1 post-baseline primary efficacy assessment. | Posted | Mean | Standard Deviation | Angioedema attacks per month | From start of treatment up to 12 weeks during each treatment period |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Cumulative Attack-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period | Severity of the angioedema attack sign/symptom was characterized as None: no symptom; Mild: noticeable symptom but easily tolerated by the participant and did not interfere with routine activities; Moderate: symptom interfered with the participant's ability to attend school or participate in family life and social/recreational activities; Severe: symptom significantly limited the participant's ability to attend school or participate in family life and social/recreational activities. Symptom severity score was assigned as Mild = 1, Moderate = 2 and Severe = 3. Cumulative attack severity score was the sum of the maximum symptom severity scores recorded for each angioedema attack in a treatment period. Cumulative attack-severity score normalized per month [(raw score/number of days of participation in that treatment period)*30.4] was reported here. Cumulative attack-severity score normalized per month ranged from 0 to 10.4 and higher scores represent worse symptoms. | FAS included all participants in the safety set who had at least 1 post-baseline primary efficacy assessment. | Posted | Mean | Standard Deviation | Score on a scale | From start of treatment up to 12 weeks during each treatment period |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Cumulative Daily-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period | Severity of the angioedema attack sign/symptom was characterized as None: no symptom; Mild: noticeable but easily tolerated by the participant and did not interfere with routine activities; Moderate: interfered with the participant's ability to attend school or participate in family life and social/recreational activities; Severe: significantly limited the participant's ability to attend school or participate in family life and social/recreational activities. Symptom severity score was assigned as Mild = 1, Moderate = 2 and Severe = 3. Cumulative daily-severity score was the sum of the severity scores recorded for every day of reported symptoms in a treatment period. Cumulative daily-severity score normalized per month [(raw score/number of days of participation in that treatment period)*30.4] was reported here. Cumulative daily-severity score normalized per month ranged from 0 to 15.6 and higher scores represent worse symptoms. | FAS included all participants in the safety set who had at least 1 post-baseline primary efficacy assessment. | Posted | Mean | Standard Deviation | Score on a scale | From start of treatment up to 12 weeks during each intervention period |
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| Secondary | Normalized Number of Angioedema Attacks Per Month Requiring Acute Treatment in a Treatment Period | Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks requiring acute treatment was normalized for the number of days participants participated in a given period and expressed as the monthly frequency. | FAS included all participants in the safety set who had at least 1 post-baseline primary efficacy assessment. | Posted | Mean | Standard Deviation | Angioedema attacks per month | From start of treatment up to 12 weeks during each intervention period |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Dose Group | An adverse event (AE) was any untoward, undesired, unplanned clinical event in the form of signs, symptoms, disease, or laboratory or physiological observations occurring in a participant participating in a clinical study with the sponsor's product, regardless of causal relationship. TEAEs were defined as events that started or worsened on or after the date and time of the first dose of investigational product and up to 7 days after the last dose of investigational product. | Safety set included all participants who received at least 1 dose of investigational product. | Posted | Count of Participants | Participants | From start of study treatment up to 25 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen | C1 INH antigen concentration in plasma was determined using an automated nephelometric assay. | Pharmacokinetic (PK) set consisted of all pariticipants in the safety set with no major deviations related to investigational product intake and evaluable PK profiles. | Posted | Mean | Standard Deviation | Gram per liter (g/L) | Pre-dose and 1 hour (h) post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention period |
|
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| Secondary | C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma | The functional activity of C1 INH in plasma samples was determined by a chromogenic assay. | PK set consisted of all pariticipants in the safety set with no major deviations related to investigational product intake and evaluable PK profiles. | Posted | Mean | Standard Deviation | Units per milliliter (U/mL) | Pre-dose and 1 h post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention period |
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| Secondary | Plasma Concentration of Complement C4 | Concentration of Complement C4 in plasma was determined using an automated nephelometric assay. | PK set consisted of all pariticipants in the safety set with no major deviations related to investigational product intake and evaluable PK profiles. | Posted | Mean | Standard Deviation | Milligram per liter (mg/L) | Pre-dose and 1 h post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention period |
|
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| Secondary | Number of Participants With C1 Esterase Inhibitor (C1 INH) Antibodies in Plasma | The presence of C1 INH antibodies in plasma samples was determined using a proprietary enzyme-linked-immunosorbent-assay. Number of participants with C1 INH Antibodies was reported. | Safety set included all participants who received at least 1 dose of investigational product. | Posted | Count of Participants | Participants | Pre-dose, 1 week post treatment (Week 13, Week 25) and 1 month post treatment follow-up (Week 28) |
|
|
From start of study drug administration up to Week 25
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Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Treatment A (500 U CINRYZE) | Participants received 500 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 1 in sequence A-B and ntervention period 2 in sequence B-A). Each intervention period was of 12 weeks. | 0 | 12 | 0 | 12 | 10 | 12 |
| EG001 | Treatment B (1000 U CINRYZE) | Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks. | 0 | 12 | 0 | 12 | 11 | 12 |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Coccydynia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Vascular pain | Vascular disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Facial pain | General disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Infusion site pain | General disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Hereditary angioedema | Congenital, familial and genetic disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Gingivitis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| H1n1 influenza | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Paronychia | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Tinea pedis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Viral pharyngitis | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 20.0 | Non-systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Excoriation | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Face injury | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Joint injury | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Lip injury | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Multiple injuries | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Post-Traumatic neck syndrome | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Sunburn | Injury, poisoning and procedural complications | MedDRA 20.0 | Non-systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Erythema marginatum | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Prurigo | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 20.0 | Non-systematic Assessment |
|
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | May 25, 2017 | Apr 24, 2018 | SAP_001.pdf |
| 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 |
Not provided
Not provided
| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Treatment B (1000 U CINRYZE) |
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks. |
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Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks. |
|
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|
Participants received 1000 U CINRYZE IV injection twice weekly (every 3 or 4 days) for 24 weeks (Intervention period 2 in sequence A-B and ntervention period 1 in sequence B-A). Each intervention period was of 12 weeks. |
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| Units | Counts |
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| Participants |
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