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Phase 2 Completed N=32 Randomized Quadruple-blind Prevention

A Phase 2 HAE Prophylaxis Study With Recombinant Human C1 Inhibitor

Source: ClinicalTrials.gov NCT02247739 ↗
Enrolled (actual)
32
Serious AEs
1.2%
Results posted
Dec 2017
Primary outcomePrimary: Number of HAE Attacks — 2.74; 4.36; 7.18 attacks

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

Outcome Measures

OutcomeResultp-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

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
View full record on ClinicalTrials.gov →

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. Informational only — not medical advice.

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