A Phase 3 interventional study of C1 esterase inhibitor [human] (C1INH-nf) in Hereditary Angioedema, sponsored by Shire. Completed at 48 sites in United States. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2021-06-08.
Sponsored by Shire · Phase 3, Interventional, and Prevention
The study objective was to evaluate the safety and efficacy of prophylactic use of C1INH-nf for the prevention of acute HAE attacks.
164 studies on the registry are indexed under Angioedema; 19 are open to participants now.
This study's enrollment of 146 is above the median of 44 across 111 interventional studies indexed under Angioedema.
Browse Angioedema studies →Shire is the lead sponsor of 346 studies on the registry; 2 are open to participants now.
Of its 47 completed or terminated interventional studies of FDA-regulated products, 47 (100%) have results posted.
Counted across the registry records on this site, refreshed daily.
In addition, this study was open to all subjects who:
Were excluded from LEVP2005-1 for any of the following reasons:
Were not enrolled in LEVP2005-1 after enrollment in LEVP2005-1 was closed, under the following circumstances:
Exclusion Criteria:
1,000 Units (U) of C1INH-nf administered intravenously (IV) every 3 to 7 days.
Biological: C1 esterase inhibitor [human] (C1INH-nf)
Frequency of All HAE Attacks
A hereditary angioedema (HAE) attack was defined as a discrete episode during which the subject progressed from no angioedema to symptoms of angioedema.
Time frame: Duration of the study
Antigenic C1 Inhibitor (C1INH) Serum Levels
Change from pre-infusion to 1 hour post-infusion in antigenic C1INH serum levels.
Time frame: Pre-infusion to 1 hour post-infusion
Functional C1INH Serum Levels
Change from pre-infusion to 1 hour post-infusion in functional C1INH serum levels. Functional C1INH serum levels are expressed as a percent of total detectable C1INH (i.e., functional C1INH/total detectable C1INH).
Time frame: Pre-infusion to 1 hour post-infusion
Complement C4 Serum Levels
Change from pre-infusion to 1 hour post-infusion in complement C4 serum levels.
Time frame: Pre-infusion to 1 hour post-infusion
| Milestone | Open-label C1INH-nf |
|---|---|
| Started | 146 |
| Completed | 79 |
| Not completed | 67 |
| Withdrew: Transitioned to commercial c1inh-nf | 40 |
| Withdrew: Lost to follow-up | 10 |
| Withdrew: Withdrawal by subject | 8 |
| Withdrew: Transferred to levp2006-1 (nct00438815) | 3 |
| Withdrew: Death | 2 |
| Withdrew: Logistical reasons | 2 |
| Withdrew: Physician decision | 1 |
| Withdrew: Withdrew to start treatment with cetor | 1 |
A hereditary angioedema (HAE) attack was defined as a discrete episode during which the subject progressed from no angioedema to symptoms of angioedema.
| attacks per month | Open-label C1INH-nf |
|---|---|
| At Screening (N=137) | 3.00 (0.08 to 28.00) |
| During Prophylactic Therapy with C1INH-nf (N=146) | 0.21 (0.00 to 4.56) |
Change from pre-infusion to 1 hour post-infusion in antigenic C1INH serum levels.
| mg/dL | Open-label C1INH-nf |
|---|---|
| Pre-infusion | 17.1 ± 19.67 |
| Increase at 1 hour post-infusion | 8.6 ± 11.89 |
Change from pre-infusion to 1 hour post-infusion in functional C1INH serum levels. Functional C1INH serum levels are expressed as a percent of total detectable C1INH (i.e., functional C1INH/total detectable C1INH).
| percent | Open-label C1INH-nf |
|---|---|
| Pre-infusion | 42.0 ± 25.85 |
| Percent increase at 1 hour post-infusion | 30.4 ± 18.05 |
Change from pre-infusion to 1 hour post-infusion in complement C4 serum levels.
| mg/dL | Open-label C1INH-nf |
|---|---|
| Pre-infusion | 10.1 ± 8.17 |
| Change at 1 hour post-infusion | -0.1 ± 3.61 |
Non-serious events are listed at a 1% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Open-label C1INH-nf | — | 0/146 (0%) | 9/146 (6.2%) |
| Event | Open-label C1INH-nf |
|---|---|
| HeadacheNervous system disorders | 3/146 |
| RashSkin and subcutaneous tissue disorders | 3/146 |
| NauseaGastrointestinal disorders | 2/146 |
| PhlebitisVascular disorders | 2/146 |
| Age, Continuous(years) | Open-label C1INH-nf |
|---|---|
| Mean | 36.5 ± 16.48 |
| Sex: Female, Male(Participants) | Open-label C1INH-nf |
|---|---|
| Female | 112 |
| Male | 34 |
This study is completed, as verified in May 2021. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Shire