A Phase 2 interventional study of Recombinant human C1 inhibitor and Placebo in Hereditary Angioedema, sponsored by Pharming Technologies B.V.. Completed at 10 sites in 7 countries. Open to participants aged 13 Years and older. Per ClinicalTrials.gov, last updated 2017-12-08.
Sponsored by Pharming Technologies B.V. · Phase 2, Interventional, and Prevention
Primary Objective:
To evaluate the efficacy of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
Secondary Objective:
To evaluate the safety and immunogenicity of recombinant human C1 inhibitor (rhC1INH) in the prophylaxis of angioedema attacks in patients with HAE
Study Design:
This is a multi-center, randomized, double-blind, placebo-controlled, 3-period crossover study of rhC1INH in prophylaxis of angioedema attacks in patients with HAE.
Medical screening (clinical and laboratory parameters) will be performed and patient medical history specific to HAE attacks will be collected to assess eligibility. Each patient will receive three 4 week periods of treatment twice weekly.
164 studies on the registry are indexed under Angioedema; 19 are open to participants now.
This study's enrollment of 32 is below the median of 44 across 111 interventional studies indexed under Angioedema.
Browse Angioedema studies →Pharming Technologies B.V. is the lead sponsor of 22 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients fulfilling the following criteria at Screening are eligible for participation in the study:
Exclusion Criteria:
Patients who meet any of the following criteria at Screening are to be excluded from study participation:
rhC1INH administered twice weekly
Biological: Recombinant human C1 inhibitor
rhC1INH administered once weekly
Biological: Recombinant human C1 inhibitor
Placebo (Saline) administered twice weekly
Other: Placebo
Also known as: rhC1INH, Ruconest, Conestat alfa
Also known as: Saline
Number of HAE Attacks
Average number of HAE attacks normalized to a 28 day period
Time frame: 28 days
Number of Participants With Adverse Events
Number of participants that experienced Treatment Emergent Adverse Events observed in safety population
Time frame: 20 weeks
Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks
Percentage of participants achieving at least 50% reduction in the number of attacks normalized to a 28-day period as compared to the placebo treatment period
Time frame: 28 days
Immunogenicity
Number of participants analyzed for neutralizing C1INH-specific antibodies and neutralizing rhC1INH-specific antibodies after confirmed anti-C1INH and anti rhC1INH IgM or IgG antibodies
Time frame: 20 weeks
| Milestone | Treatment Sequence A | Treatments Sequence B | Treatment Sequesce C | Treatment Sequence D | Treatment Sequence E | Treatment Sequence F |
|---|---|---|---|---|---|---|
| Started | 5 | 6 | 5 | 5 | 6 | 5 |
| Completed | 5 | 3 | 4 | 5 | 5 | 4 |
| Not completed | 0 | 3 | 1 | 0 | 1 | 1 |
Average number of HAE attacks normalized to a 28 day period
| attacks | rhC1INH Twice Weekly | rhC1INH Once Weekly | Placebo (Saline) Twice Weekly |
|---|---|---|---|
| Number of HAE Attacks | 2.74 (1.8 to 3.7) | 4.36 (3.1 to 5.6) | 7.18 (5.8 to 8.6) |
Number of participants that experienced Treatment Emergent Adverse Events observed in safety population
| Participants | rhC1INH Twice Weekly | rhC1INH Once Weekly | Placebo (Saline) Twice Weekly |
|---|---|---|---|
| Number of Participants With Adverse Events | 10 | 13 | 8 |
Percentage of participants achieving at least 50% reduction in the number of attacks normalized to a 28-day period as compared to the placebo treatment period
| percentage of participants | rhC1INH Twice Weekly | rhC1INH Once Weekly |
|---|---|---|
| Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks | 74 (57 to 86) | 42 (26 to 59) |
Number of participants analyzed for neutralizing C1INH-specific antibodies and neutralizing rhC1INH-specific antibodies after confirmed anti-C1INH and anti rhC1INH IgM or IgG antibodies
| Participants | rhC1INH Twice Weekly | rhC1INH Once Weekly | Placebo (Saline) Twice Weekly |
|---|---|---|---|
| Immunogenicity | 0 | 0 | 0 |
Collected over 20 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| rhC1INH Twice Weekly | — | 1/29 (3.4%) | 5/29 (17.2%) |
| rhC1INH Once Weekly | — | 0/29 (0%) | 7/29 (24.1%) |
| Placebo (Saline) Twice Weekly | — | 0/28 (0%) | 2/28 (7.1%) |
| Event | rhC1INH Twice Weekly | rhC1INH Once Weekly | Placebo (Saline) Twice Weekly |
|---|---|---|---|
| PhimosisCongenital, familial and genetic disorders | 1/29 | 0/29 | 0/28 |
| Event | rhC1INH Twice Weekly | rhC1INH Once Weekly | Placebo (Saline) Twice Weekly |
|---|---|---|---|
| HeadacheNervous system disorders | 5/29 | 2/29 | 0/28 |
| NasopharyngitisInfections and infestations | 0/29 | 3/29 | 2/28 |
| AnxietyPsychiatric disorders | 0/29 | 2/29 | 0/28 |
Population is intention to treat (ITT) population
| Age, Categorical(Participants) | All Study Participants |
|---|---|
| <=18 years | 1 |
| Between 18 and 65 years | 28 |
| >=65 years | 3 |
| Age, Continuous(years) | All Study Participants |
|---|---|
| Mean | 46 (17 to 74) |
| Sex: Female, Male(Participants) | All Study Participants |
|---|---|
| Female | 26 |
| Male | 6 |
| Region of Enrollment(participants) | All Study Participants |
|---|---|
| Canada | 2 |
| Czech Republic | 2 |
| Romania | 6 |
| United States | 7 |
| Macedonia, The Former Yugoslav Republic of | 8 |
| Italy | 1 |
| Serbia | 2 |
| Israel | 4 |
This study is completed, as verified in Nov 2017. You cannot join it, but the record below documents what was studied.
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Pharming Technologies B.V.