Italian iTTP Registry

NCT ID: NCT06376786

Last Updated: 2024-07-10

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 132 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-06-20
• Study Completion Date: 2030-05-31

Brief Summary

ItaliTTP is an observational, prospective, single-arm, national, multicenter, non-pharmacological cohort study aimed at better defining and understanding the natural history, disease severity, and clinical outcomes of patients with immune-mediated thrombotic thrombocytopenic purpura (iTTP) in Italy.

A minimum of 132 consecutive patients with acute iTTP (first event or relapse) will be enrolled for 3 years, with the possibility of extension, with a follow-up period of 3 years.

Detailed Description

Acquired immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening thrombotic microangiopathy characterized by episodes of thrombocytopenia, microangiopathic hemolytic anemia, and extensive microvascular thrombosis leading to multiorgan involvement. Despite advances in understanding iTTP etiology and management in the acute phase, significant gaps in knowledge about its progression, particularly during clinical remission and concerning long-term complications, persist.

ItaliTTP, a national, multicenter, observational, prospective, non-pharmacological cohort study, aims to elucidate the natural history, severity, and outcomes of iTTP in Italy. The study will enroll hospitalized iTTP patients (experiencing either initial or recurrent episodes) and follow them in outpatient settings across participating Italian centers. The study plans to include at least 132 patients of any gender, aged 12 to 99, over a three-year period, with an option for extension, and a three-year follow-up. During hospitalization and subsequent outpatient visits, participants will undergo routine clinical assessments and laboratory tests. In addition to these data, peripheral blood samples will be collected for ADAMTS13 analysis and potential future research.

Conditions

TTP - Thrombotic Thrombocytopenic Purpura

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

iTTP patients

iTTP patients will be treated and followed-up as per standard clinical practice.

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• Patients with an acute iTTP episode (first event or relapse), defined by thrombocytopenia and microangiopathic hemolytic anemia, in the absence of alternative causes, and the presence of severe deficiency of ADAMTS13 activity (< 10 IU/dL or <10% of normal value) and anti-ADAMTS13 autoantibodies
• Both male and female patients, aged 12 years or older
• Patients who have signed the informed consent for the participation to the study

Exclusion Criteria

• Patients who have not signed the informed consent for the participation to the study

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 99 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fondazione Luigi Villa

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Flora Peyvandi, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy

Locations

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, MI, Italy

Site Status: RECRUITING

Countries

Italy

Central Contacts

Sandra Maccarone

Role: CONTACT

contact@fondazioneluigivilla.org

+39 02 551 0709

Ilaria Mancini, MSc, PhD

Role: CONTACT

ilaria.mancini@guest.unimi.it

+39 02 5503 5414

Other Identifiers

ItaliTTP

Identifier Type: -

Identifier Source: org_study_id