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

NCT06690047 | Completed Phase 4 FDA Drug

• 1 other identifier: Ruc01
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 2

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

Conditions

Hereditary Angioedema

Keywords

prodrome

Outcome Measures

Primary Outcomes (1)

• The primary endpoint was the difference in response rates in preventing HAE attacks between Conestat Alfa®-treated vs. untreated HAE prodrome events.

  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.

  up to 12 months from start of study

Study Arms (2)

Ruconest

ACTIVE COMPARATOR

open-label Ruconest

Biological: Ruconest

No Ruconest

NO INTERVENTION

No Ruconest given

Interventions

Ruconest BIOLOGICAL

recombinant human C1-esterase inhibitor

Ruconest

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Prior diagnosis of HAE Type 1 and 2,
• One or more HAE attacks per month,
• History of 4 prodromes that proceed to angioedema attacks

You may not qualify if:

• 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

Sponsors & Collaborators

• Bernstein Clinical Research Center: lead

Study Sites (2)

Clinical Research Center of Alabama

Birmingham, Alabama, 35209, United States

Location

Bernstein Clinical Research Center, LLC

Cincinnati, Ohio, 45236, United States

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

conestat alfa

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Study Officials

• Jonathan Bernstein, MD

  Bernstein Clinical Research Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PI

Study Record Dates

• First Submitted: November 12, 2024
• First Posted: November 15, 2024
• Study Start: August 14, 2018
• Primary Completion: April 28, 2022
• Study Completion: April 28, 2022
• Last Updated: November 15, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)