Global Registry to Gather Data on Natural History of Patients With Hereditary Angioedema Type I and II

NCT03828279 | Unknown DMC

• 1 other identifier: HAERegistry
• Study Type: observational
• Target: 220
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this international hereditary angioedema (HAE) register is to collect homogeneous clinical and laboratory data on patients with HAE type I and II gathering better information on the natural course of the disease and detecting therapeutic options to manage it.

Conditions

Hereditary Angioedema Type I and II

Keywords

angioedema; disease registry; C1 inhibitor; anti bradykinin treatments

Outcome Measures

Primary Outcomes (3)

• Number of angioedema events

  Number of angioedema events

  Through study completion, an average of 5 years

• Time of angioedema events

  Time in hours of presence of angioedema symptoms

  Through study completion, an average of 5 years

• Severity of angioedema events

  Number of severe, moderate, mild angioedema symptoms based on a three point scale patient reported outcome

  through study completion, an average of 5 years

Secondary Outcomes (3)

• Comorbidities

  Through study completion , an average of 5 years

• Treatment-Emergent comorbidities

  Through study completion , an average of 5 years

• Treatment efficacy

  Through study completion , an average of 5 years

Study Arms (2)

hereditary angioedema type I

Patients were diagnosed as C1 inhibitor HAE type I when functional and antigenic C1 inhibitor were ≤ 50% of normal

Diagnostic Test: functional and antigenic C1 inhibitor

hereditary angioedema type II

Patients were diagnosed as type II when functional C1 inhibitor was ≤50% and antigenic was \>50% of normal

Diagnostic Test: functional and antigenic C1 inhibitor

Interventions

functional and antigenic C1 inhibitor DIAGNOSTIC_TEST

Diagnosis of HAE is based on personal and/or family history of angioedema and on C1 inhibitor functional or antigenic plasma levels ≤50% of normal.

Also known as: genetic analysis

hereditary angioedema type I; hereditary angioedema type II

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

patients diagnosed with HAE type I and II and followed by a referral center for angioedema

You may qualify if:

• patients diagnosed with HAE type I and II with signed informed consent
• laboratory diagnostic criteria documenting plasma levels of C1 inhibitor antigen and function and of C4 antigen

You may not qualify if:

• patients without HAE type I and II
• patients without documented laboratory diagnostic criteria
• patients not capable to give informed consent

Sponsors & Collaborators

• HAE Global Registry Foundation: lead

Study Sites (1)

ASST FBF Sacco

Milan, 20157, Italy

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Sodium Citrate samples are collected form patients for testing plasma levels of C1 inhibitor antigen and function and C4 antigen. Cells are collected for DNA extraction and detection of mutations in SERPING1

MeSH Terms

Conditions

Hereditary Angioedema Types I and II; Angioedema; Angioedemas, Hereditary

Interventions

Genetic Testing

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Genetic Techniques; Genetic Services; Health Services; Health Care Facilities Workforce and Services; Diagnostic Services; Preventive Health Services

Study Officials

• marco cicardi, MD

  HAE Global Registry Foundation

  PRINCIPAL INVESTIGATOR

Central Study Contacts

marco cicardi, MD

CONTACT

+39 0239042516; marco.cicardi@unimi.it

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 1 Year
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2019
• First Posted: February 4, 2019
• Study Start: October 1, 2017
• Primary Completion: December 31, 2019
• Study Completion: December 31, 2023
• Last Updated: February 5, 2019

Record last verified: 2019-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: entire study period
• Access Criteria: to be a member of the HAERegistry and have the approval of the HGRSC to analyze data

Locations

IT (1)