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Table 1 Features of COMPACT [14] and CHANGE [11, 17] studies

From: Indirect comparison of intravenous vs. subcutaneous C1-inhibitor placebo-controlled trials for routine prevention of hereditary angioedema attacks

  COMPACT CHANGE
C1-INH formulation Subcutaneous Intravenous
Study phase III III
Study design Twice-weekly, dosed by body-weight, C1-INH(SC), 40 or 60 IU/kg for 16 weeks, preceded or followed by placebo for 16 weeks Twice-weekly, fixed dose, C1-INH(IV) 1000 U for 12 weeks, preceded or followed by placebo for 12 weeks
Inclusion Criteria Age ≥ 12 years
C1-INH-HAE type I/II confirmed by central laboratory
≥ 4 HAE attacks over consecutive 2 months within 3 months before screening
Stable oral HAE prophylaxis regimen permitteda
Age ≥ 6 years
C1-INH-HAE confirmed by laboratory analysis
Frequent HAE attacks ≥ 2 per month
Normal C1q level
Exclusion Criteria History of arterial/venous thrombosis requiring anticoagulant therapy or current prothrombotic risk
History of poor response to C1-INH therapy for the management of HAE
Incurable malignancies
C1-INH(IV) routine HAE prophylaxis within 3 months of screeninga
Pregnant, nursing, or plan to become pregnant during the study
Alcohol, drug, or medication abuse within 1 year of screening
Known or suspected hypersensitivity to the investigational product
Participated in another interventional clinical study within 30 days of screening
Unable to have HAE adequately managed with on-demand treatment
Any clinically significant medical condition, e.g., renal failure, that would interfere with participation
Presence of anti-C1-INH autoantibody
B cell malignancy
Participation in a C1 esterase inhibitor trial, or received blood/blood product in the past 90 days
Pregnancy or lactation
Narcotic addiction
History of allergic reaction to C1-INH or other blood products
Participation in any other investigational drug study within the past 30 days
Low C1q level
  1. HAE hereditary angioedema, IU international units, IV intravenous, SC subcutaneous, U units
  2. aAmendment implemented during the study