Trial · NCT02865720
Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)
NCT02865720, Phase 3, Completed, Shire; results first posted 2018-12-31
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE). Trials, record NCT02865720. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02865720
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)
- NCT number
- NCT02865720
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 8 participants, actual
- Lead sponsor
- Shire
- Conditions
- Hereditary Angioedema (HAE)
- Interventions
- CINRYZE 500 U, CINRYZE 1000 U
- First posted
- 2016-08-12
- Last updated
- 2021-06-02
- Results first posted
- 2018-12-31
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From start of study drug administration up to Week 12 | An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered an investigational product and that did not necessarily have a causal relationship with the treatment. TEAEs were defined as all AEs that started during the treatment period and up to 7 days after the last dose of investigational product, or AEs that were seen at baseline but worsened in frequency and/or severity during the treatment period and up to 7 days after the last dose of investigational product. |
| Primary | Number of Participants With Clinically Significant Abnormalities in Physical Examination Reported as Adverse Events (AEs) | From start of study drug administration up to Week 12 | Physical examinations included measurement of body weight and height. Clinically significant abnormalities related to physical examination as determined by investigator were recorded and reported as AE. |
| Primary | Number of Participants With Potentially Clinically Important (PCI) Vital Signs Reported as Adverse Events (AEs) | Baseline up to Week 12 | Vital sign assessments included systolic blood pressure (SBP), diastolic blood pressure (DBP) and pulse rate. Investigator used both absolute values and change from baseline values to determine if the vital sign was potentially clinically important. Criteria for the potential clinical importance of both absolute and change from baseline values were pre-specified as: SBP (less than \[\<\] 90 millimeter of mercury \[mmHg\]; greater than or equal to \[\>=\] 140 mmHg), DBP (\< 60 mmHg; \>=90 mmHg) and pulse (less than or equal to \[\<=\] 50 beats per minute \[bpm\]; \>= 100 bpm. A participant's vital sign had to meet both the absolute and change from baseline criteria to be considered as potentially clinically important. |
| Primary | Number of Participants With Potentially Clinically Important (PCI) Clinical Laboratory Assessments Reported as Adverse Events (AEs) | Baseline up to Week 12 | Number of participants with potentially clinically important (PCI) clinical laboratory assessments reported as adverse events were reported. |
| Primary | Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 1 | Week 1: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 hours (h) post-dose | C1 INH antigen concentration in plasma was determined using an automated nephelometric assay. |
| Primary | Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 12 | Week 12: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 h post-dose | C1 INH antigen concentration in plasma was determined using an automated nephelometric assay. |
| Primary | Concentration of Plasma Complement C4 at Week 1 | Week 1: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 h post-dose | Concentration of plasma complement C4 was reported. |
| Primary | Concentration of Plasma Complement C4 at Week 12 | Week 12: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 h post-dose | Concentration of plasma complement C4 was reported. |
| Primary | Concentration of Plasma Complement C1q at Week 1 | Baseline (Week 1) | Concentration of plasma complement C1q was reported. |
| Primary | Normalized Number of Angioedema Attacks (NNA) Per Month | Baseline up to Week 12 | Angioedema attack was defined as any participant-reported (or caregiver-reported) indication of swelling or pain at any location following a report of no swelling or pain on the previous day (that is, there must have been a full symptom-free calendar day preceding the onset of symptoms for an attack to be considered a new attack). NNA was calculated as the overall number of angioedema attacks recorded during the period divided by the number of days in the period and multiplied by 30.4.Number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency as compared to the historical data where, NNA was the number of angioedema attacks during 3 months prior to study drug administration. Historical data was obtained from medical or angioedema history electronic case report forms (eCRF). |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | From start of study drug administration up to Week 12 | ITT-S set included participants who received any amount of investigational product. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Clinically Significant Abnormalities in Physical Examination Reported as Adverse Events (AEs) | From start of study drug administration up to Week 12 | ITT-S set included participants who received any amount of investigational product. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Important (PCI) Vital Signs Reported as Adverse Events (AEs) | Baseline up to Week 12 | ITT-S set included participants who received any amount of investigational product. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Potentially Clinically Important (PCI) Clinical Laboratory Assessments Reported as Adverse Events (AEs) | Baseline up to Week 12 | ITT-S set included participants who received any amount of investigational product. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 1 | Week 1: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 hours (h) post-dose | Pharmacokinetic (PK) set included all participants with evaluable PK profiles. | Gram per liter (g/L) | MEAN |
| PRIMARY | Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 12 | Week 12: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 h post-dose | PK set included all participants with evaluable PK profiles. | gram per liter (g/L) | MEAN |
| PRIMARY | Concentration of Plasma Complement C4 at Week 1 | Week 1: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 h post-dose | Pharmacodynamic (PD) set included all participants with evaluable PD profiles. | Milligram per liter (mg/L) | MEAN |
| PRIMARY | Concentration of Plasma Complement C4 at Week 12 | Week 12: Pre-dose, 0.5, 1, 2, 6, 24, 48, 72 and 96 h post-dose | PD set included all participants with evaluable PD profiles. | mg/L | MEAN |
| PRIMARY | Concentration of Plasma Complement C1q at Week 1 | Baseline (Week 1) | PD set included all participants with evaluable PD profiles. | International units per milliliter | MEAN |
| PRIMARY | Normalized Number of Angioedema Attacks (NNA) Per Month | Baseline up to Week 12 | Full analysis set (FAS) included all participants who had at least 1 post-baseline efficacy assessment. | Angioedema attacks per month | MEAN |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group98
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | CINRYZE 1000 U | 7 | Participants | ||
| Number of Participants With Clinically Significant Abnormalities in Physical Examination Reported as Adverse Events (AEs) | CINRYZE 1000 U | 0 | Participants | ||
| Number of Participants With Potentially Clinically Important (PCI) Vital Signs Reported as Adverse Events (AEs) | CINRYZE 1000 U | 0 | Participants | ||
| Number of Participants With Potentially Clinically Important (PCI) Clinical Laboratory Assessments Reported as Adverse Events (AEs) | CINRYZE 1000 U | 0 | Participants | ||
| Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 1 | CINRYZE 1000U | Pre-dose | 0.0581 | Standard Deviation 0.04877 | Gram per liter (g/L) |
| Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 1 | CINRYZE 1000U | 0.5 h post-dose | 0.1463 | Standard Deviation 0.04210 | Gram per liter (g/L) |
| Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 1 | CINRYZE 1000U | 1 h post-dose | 0.1426 | Standard Deviation 0.05795 | Gram per liter (g/L) |
| Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 1 | CINRYZE 1000U | 2 h post-dose | 0.1436 | Standard Deviation 0.04524 | Gram per liter (g/L) |
| Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 1 | CINRYZE 1000U | 6 h post-dose | 0.1413 | Standard Deviation 0.04376 | Gram per liter (g/L) |
| Concentration of C1 Esterase Inhibitor (C1 INH) Antigen (Protein Volume) at Week 1 | CINRYZE 1000U | 24 h post-dose | 0.1147 | Standard Deviation 0.03825 | Gram per liter (g/L) |
1 to 10 of 98Source ClinicalTrials.gov
See all 98 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | CINRYZE 500 U | 0 | |
| Overall Study | STARTED | CINRYZE 1000 U | 8 | |
| Overall Study | COMPLETED | CINRYZE 500 U | 0 | |
| Overall Study | COMPLETED | CINRYZE 1000 U | 8 | |
| Overall Study | NOT COMPLETED | CINRYZE 500 U | 0 | |
| Overall Study | NOT COMPLETED | CINRYZE 1000 U | 0 |
Source ClinicalTrials.gov
Adverse events21
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Arthralgia | Musculoskeletal and connective tissue disorders | CINRYZE 1000 U | 2 | 8 | 2 |
| other | Intervertebral disc disorder | Musculoskeletal and connective tissue disorders | CINRYZE 1000 U | 1 | 8 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | CINRYZE 1000 U | 1 | 8 | 1 |
| other | Constipation | Gastrointestinal disorders | CINRYZE 1000 U | 1 | 8 | 1 |
| other | Chest pain | General disorders | CINRYZE 1000 U | 1 | 8 | 1 |
| other | Injection site pain | General disorders | CINRYZE 1000 U | 1 | 8 | 3 |
| other | Seasonal allergy | Immune system disorders | CINRYZE 1000 U | 1 | 8 | 1 |
| other | Herpes virus infection | Infections and infestations | CINRYZE 1000 U | 1 | 8 | 2 |
| other | Nasopharyngitis | Infections and infestations | CINRYZE 1000 U | 3 | 8 | 4 |
| other | Arthropod bite | Injury, poisoning and procedural complications | CINRYZE 1000 U | 1 | 8 | 1 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | CINRYZE 1000 U | 0 | 8 |
| Total, other adverse events | CINRYZE 1000 U | 7 | 8 |
| Total, serious adverse events | CINRYZE 1000 U | 2 | 8 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02865720
GET https://conductscience.org/api/trials/v1/NCT02865720/results?page=2