Phase 2
N=32
A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor
Hereditary Angioedema
Bottom Line
View on ClinicalTrials.gov: NCT02247739 ↗Enrolled (actual)
32
Serious AEs
1.2%
Results posted
Dec 2017
Primary outcome: Primary: Number of HAE Attacks — 2.74; 4.36; 7.18 attacks
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Recombinant human C1 inhibitor (Biological); Placebo (Other)
- Age
- Pediatric, Adult, Older Adult · 13+ yrs
- Sex
- All
- Sponsor
- Pharming Technologies B.V.
- Primary completion
- May 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of HAE Attacks |
2.74; 4.36; 7.18 | — |
| SECONDARY Number of Participants With Adverse Events |
10; 13; 8 | — |
| SECONDARY Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks |
74; 42 | — |
Summary
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
Eligibility Criteria
Inclusion Criteria
Patients fulfilling the following criteria at Screening are eligible for participation in the study:
- Age 13 years or older
- Laboratory confirmed diagnosis of HAE
- A history of frequent HAE attacks (at least 4 attacks per month across a minimum of 3 consecutive months).
- Female patients of childbearing potential who are sexually active must be willing to use an acceptable form of contraception.
- Provided written informed consent (and written assent for minors)
- Willingness and ability to comply with all protocol procedures
Exclusion Criteria
Patients who meet any of the following criteria at Screening are to be excluded from study participation:
- Patients with medical history of allergy to rabbits or rabbit-derived products (including rhC1INH)
- Diagnosis of acquired angioedema (AAE)
- Patients who are pregnant, or breastfeeding, or are currently intending to become pregnant
- Treatment with any investigational drug in the past 30 days
- Patients with any condition or treatment that, in the opinion of the Investigator, might interfere with the evaluation of study objectives
- Patients currently treated with angiotensin-converting enzyme (ACE) inhibitors
Data sourced from ClinicalTrials.gov (NCT02247739). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.