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Phase 3 N=75 Randomized Double-blind Treatment

Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks

Hereditary Angioedema

Enrolled (actual)
75
Serious AEs
1.4%
Results posted
Aug 2015
Primary outcome: Primary: Time to Beginning of Relief of Symptoms — 90; 152 minutes

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
rhC1INH (Drug); Placebo (Saline) (Drug)
Age
Pediatric, Adult, Older Adult · 13+ yrs
Sex
All
Sponsor
Pharming Technologies B.V.
Primary completion
Dec 2012

Outcome Measures

OutcomeResultp-value
PRIMARY
Time to Beginning of Relief of Symptoms
90; 152
SECONDARY
Time to Minimal Symptoms
303; 483

Summary

This study is being conducted to confirm the efficacy, safety, and immunogenicity of recombinant human C1 inhibitor (rhC1INH) at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in Hereditary Angioedema (HAE) patients.

Eligibility Criteria

Inclusion Criteria

  • Aged at least 13 years
  • Signed written informed consent
  • Clear clinical and laboratory diagnosis of HAE with baseline plasma level of functional C1INH of less than 50% of normal
  • Willingness and ability to comply with all protocol procedures
  • Clinical symptoms of an eligible HAE attack with onset less than 5 hours before the time of initial evaluation

Exclusion Criteria

  • Medical history of allergy to rabbits or rabbit-derived products (including rhC1INH), or positive anti-rabbit dander IgE test (cut off >0.35 kU/L; ImmunoCap® assay; Phadia or equivalent).
  • A diagnosis of acquired C1INH deficiency (AAE)
  • Pregnancy, or breastfeeding, or current intention to become pregnant
  • Treatment with any investigational drug in the past 30 days
  • Known or suspected addiction to drug and/or alcohol abuse
  • Suspicion for an alternate explanation of the symptoms other than acute HAE attack
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01188564). 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.

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