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Treatment of Hereditary Angioedema Prodrome with Recombinant C1-esterase Inhibitor (Ruconest)

Phase 4
Completed
Conditions
Hereditary Angioedema
Interventions
Registration Number
NCT06690047
Lead Sponsor
Bernstein Clinical Research Center
Brief Summary

To assess the efficacy of recombinant human C1-esterase inhibitor in the management of HAE prodrome for preventing the progression from prodrome to an acute angioedema attacks. Subjects will either receive Ruconest after the first 2 prodromes or during the last 2 prodromes. 5 clinic visits will occur within 24 hours of a prodrome. Subjects will complete prodrome severity and angioedema attack diaries

Detailed Description

After screening, subjects will be followed in the study for four consecutive prodromes. For prodromes designed to be treated with Ruconest, the drug will be administered within 12 hours of prodromal onset and prior to the onset of objective selling. Ruconset will be self-administered at home or by a healthcare professional at the recommended dose of 50 IU/kg body weight with maximum dose of 4200 IU as a slow IV injection over 5 minutes Subjects will be randomized into two arms after enrollment. Subjects in Arm A receives Ruconest after 1st and 2nd prodrome and no Ruconest after 3rd and 4th prodromes. Subjects in Arm B receive no Ruconest after 1st and 2nd prodrome and Ruconest after 3rd and 4th prodromes.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
5
Inclusion Criteria
  • Prior diagnosis of HAE Type 1 and 2,
  • One or more HAE attacks per month,
  • History of 4 prodromes that proceed to angioedema attacks
Exclusion Criteria
  • History of thrombosis or arterial/venous thromboembolic attacks
  • History of atherosclerosis, morbid obesity, immobility
  • History of allergy to rabbits or products from rabbits
  • History of life-threatening immediate allergic reactions to C1 esterase inhibitor preparations

Study & Design

Study Type
INTERVENTIONAL
Study Design
CROSSOVER
Arm && Interventions
GroupInterventionDescription
RuconestRuconestopen-label Ruconest
Primary Outcome Measures
NameTimeMethod
The primary endpoint was the difference in response rates in preventing HAE attacks between Conestat Alfa®-treated vs. untreated HAE prodrome events.up to 12 months from start of study

A mixed model for repeated measures was used to determine the statistical significance of Conestat Alfa®'s clinical efficacy for treating the HAE prodrome versus the acute attack.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (2)

Clinical Research Center of Alabama

🇺🇸

Birmingham, Alabama, United States

Bernstein Clinical Research Center, LLC

🇺🇸

Cincinnati, Ohio, United States

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