Trial · NCT00438815
Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks
NCT00438815, Phase 3, Completed, Shire; results first posted 2010-06-09
3 sourcestrials-2026-11-12-0.4.0
Cite this record
ConductScience Library. Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks. Trials, record NCT00438815. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00438815
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks
- NCT number
- NCT00438815
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 113 participants, actual
- Lead sponsor
- Shire
- Conditions
- Hereditary Angioedema
- Interventions
- C1 esterase inhibitor [human] (C1INH-nf)
- First posted
- 2007-02-22
- Last updated
- 2021-06-08
- Results first posted
- 2010-06-09
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Hereditary Angioedema (HAE) Attacks Treated With C1INH-nf | Duration of the study (2.5 years) | |
| Primary | Percent of HAE Attacks With Substantial Relief of the Defining Symptom | Within 4 hours after initial treatment | Subjects were to assess their symptoms every 15 minutes up to 4 hours after the initial dose or until substantial relief of the defining symptom was achieved. The conservative analysis defined substantial relief as 3 consecutive assessments of improvement of the defining symptom; any attack that did not have 3 consecutive documented reports of improvement was considered a treatment failure. In the less conservative analysis, attacks also were considered to have responded if clinical improvement of the defining symptom occurred but data were incomplete due to cessation of symptom assessments. |
| Secondary | Time to Beginning of Substantial Relief of the Defining Symptom | Within 4 hours after initial treatment | Subjects were to assess their symptoms every 15 minutes up to 4 hours after the initial dose or until substantial relief of the defining symptom was achieved. Substantial relief was defined as 3 consecutive assessments of improvement of the defining symptom. Beginning of substantial relief was considered the first of the 3 consecutive assessments. |
| Secondary | Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments | Within 4 hours after initial treatment | For attack number 1, the number of censored observations precluded estimation of the 95% confidence interval (CI) upper bound for median time to event (subjects who did not experience beginning of substantial relief of the defining symptom within 4 hours after initial treatment were included in the analysis as censored observations). Entry of 4.0 hours indicates that data were not estimable (NE); as non-numeric data are not supported by the 95% CI field, entry of the actual result (ie, NE or \>4.0) was not possible. |
| Secondary | Antigenic C1 Inhibitor (C1INH) Serum Levels | Pre-infusion to 1 hour post-infusion | Change in antigenic C1INH serum levels from pre-infusion to 1 hour after the initial dose of study drug. |
| Secondary | Functional C1INH Serum Levels | Pre-infusion to 1 hour post-infusion | Percent change in functional C1INH serum levels from pre-infusion to 1 hour after the initial dose of study drug. Functional C1INH serum levels are expressed as a percent of total detectable C1INH (ie, functional C1INH/total detectable C1INH). |
| Secondary | Complement C4 Serum Levels | Pre-infusion to 1 hour post-infusion | Change in complement C4 serum levels from pre-infusion to 1 hour after the initial dose of study drug. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Hereditary Angioedema (HAE) Attacks Treated With C1INH-nf | Duration of the study (2.5 years) | Intent-to-treat Efficacy (ITT-E) Population (N=101; the number of subjects who received at least one dose of C1INH-nf for the treatment of an acute HAE attack). | attacks | NUMBER |
| SECONDARY | Time to Beginning of Substantial Relief of the Defining Symptom | Within 4 hours after initial treatment | ITT-E Population. | hours | MEDIAN |
| SECONDARY | Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments | Within 4 hours after initial treatment | ITT-E subjects with at least 10 HAE attacks during the study (N=15). | hours | MEDIAN |
| SECONDARY | Antigenic C1 Inhibitor (C1INH) Serum Levels | Pre-infusion to 1 hour post-infusion | ITT-E subjects with data at both sampling time points (N=84). | mg/dL | MEAN |
| SECONDARY | Functional C1INH Serum Levels | Pre-infusion to 1 hour post-infusion | ITT-E subjects with data at both sampling time points (N=82). | percent of functional C1INH | MEAN |
| SECONDARY | Complement C4 Serum Levels | Pre-infusion to 1 hour post-infusion | ITT-E subjects with data at both sampling time points (N=74). | mg/dL | MEAN |
| PRIMARY | Percent of HAE Attacks With Substantial Relief of the Defining Symptom | Within 4 hours after initial treatment | ITT-E Population (N=101; the number of subjects who received at least one dose of C1INH-nf for the treatment of an acute HAE attack). | percent of attacks | NUMBER |
Source ClinicalTrials.gov
Results by group21
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Hereditary Angioedema (HAE) Attacks Treated With C1INH-nf | Open-label C1INH-nf | Conservative Analysis | 609 | attacks | |
| Number of Hereditary Angioedema (HAE) Attacks Treated With C1INH-nf | Open-label C1INH-nf | Less Conservative Analysis | 598 | attacks | |
| Time to Beginning of Substantial Relief of the Defining Symptom | Open-label C1INH-nf | 0.75 | 95% Confidence Interval 0.50 to 1.00 | hours | |
| Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments | Open-label C1INH-nf | Attack number 1 | 1.50 | 95% Confidence Interval 0.75 to 4.00 | hours |
| Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments | Open-label C1INH-nf | Attack number 2 | 0.50 | 95% Confidence Interval 0.25 to 2.50 | hours |
| Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments | Open-label C1INH-nf | Attack number 3 | 0.50 | 95% Confidence Interval 0.50 to 0.75 | hours |
| Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments | Open-label C1INH-nf | Attack number 4 | 0.50 | 95% Confidence Interval 0.25 to 1.00 | hours |
| Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments | Open-label C1INH-nf | Attack number 5 | 0.75 | 95% Confidence Interval 0.50 to 1.25 | hours |
| Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments | Open-label C1INH-nf | Attack number 6 | 0.50 | 95% Confidence Interval 0.25 to 1.25 | hours |
| Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments | Open-label C1INH-nf | Attack number 7 | 0.75 | 95% Confidence Interval 0.50 to 0.75 | hours |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 results by groupParticipant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Open-label C1INH-nf | 113 | |
| Overall Study | COMPLETED | Open-label C1INH-nf | 43 | |
| Overall Study | NOT COMPLETED | Open-label C1INH-nf | 70 |
Source ClinicalTrials.gov
Adverse events2
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 | Infusion site pain | General disorders | Open-label C1INH-nf | 1 | 113 | |
| other | Rash | Skin and subcutaneous tissue disorders | Open-label C1INH-nf | 1 | 113 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Open-label C1INH-nf | ||
| Total, other adverse events | Open-label C1INH-nf | 1 | 113 |
| Total, serious adverse events | Open-label C1INH-nf | 0 | 113 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
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.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone 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/NCT00438815
GET https://conductscience.org/api/trials/v1/NCT00438815/results?page=2