Clinical trial NCT06376786: Italian iTTP Registry
Official title: Italian iTTP Registry (a Prospective Observational Study)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: TTP - Thrombotic Thrombocytopenic Purpura
Keywords: ADAMTS13
Thrombotic microangiopathy
Lead sponsor: Fondazione Luigi Villa
Sponsor class: OTHER
First posted: 2024-04-19
Enrollment: 132
Sex: ALL
Minimum age (years): 12
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Hospitalized patients with an acute event of iTTP (first event or relapse), then followed-up in outpatient clinics of the participating centers.

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.

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

Primary outcomes:
measure: Age at onset
timeFrame: 3 years
measure: Sex
timeFrame: 3 years
measure: Birth Country/Region
timeFrame: 3 years
measure: Race
timeFrame: 3 years
measure: Blood group
timeFrame: 3 years
measure: BMI
timeFrame: 3 years
measure: Proportion of patients with comorbidities, including: autoimmune diseases, cancer, HIV infection, hypertension, type 2 diabetes, hypercholesterolemia, cardiovascular disease, chronic renal failure, liver disease, depression.
timeFrame: 3 years
measure: Proportion of acute iTTP episodes preceded by potential triggering factors including: infections, pregnancy, surgery, psychological trauma, vaccination, drugs
timeFrame: 3 years
measure: Incidence, type and severity of clinical manifestations, including: bleeding, cardiovascular, neurological, renal and systemic signs and symptoms
timeFrame: 3 years
measure: Platelet count lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
timeFrame: 3 years
measure: Hemoglobin lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
timeFrame: 3 years
measure: Lactate dehydrogenase (LDH) lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
timeFrame: 3 years
measure: Creatinine lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
timeFrame: 3 years
measure: Cardiac troponin
timeFrame: 3 years
measure: ADAMTS13 activity
timeFrame: 6 years
measure: Anti-ADAMTS13 antibodies
timeFrame: 6 years
measure: Number of daily therapeutic plasma exchange procedures
timeFrame: 3 years
measure: Proportion of acute iTTP patients treated with rituximab
timeFrame: 6 years
measure: Proportion of acute iTTP patients treated with immunosuppressors other than steroids and rituximab
timeFrame: 6 years
measure: Proportion of iTTP patients treated with caplacizumab
timeFrame: 3 years
measure: Incidence, type and severity of TTP-related drugs adverse events
timeFrame: 6 years
measure: Proportion of iTTP patients achieving clinical remission
timeFrame: 6 years
measure: Proportion of iTTP patients refractory to acute iTTP treatment
timeFrame: 6 years
measure: Proportion of iTTP patients experiencing complications during hospitalization, including: bleeding, thrombosis, neurological, renal, cardiac complications
timeFrame: 6 years
measure: Proportion of iTTP patients experiencing clinical exacerbation
timeFrame: 6 years
measure: Proportion of iTTP patients achieving ADAMTS13 remission
timeFrame: 6 years
measure: Time to clinical response
timeFrame: 6 years
measure: Time to clinical remission
timeFrame: 6 years
measure: Time to ADAMTS13 remission
timeFrame: 6 years
measure: Proportion of iTTP patients with a clinical relapse
timeFrame: 6 years
measure: Proportion of iTTP patients with an ADAMTS13 relapse
timeFrame: 6 years
measure: Time to clinical relapse
timeFrame: 6 years
measure: Time to ADAMTS13 relapse
timeFrame: 6 years
measure: Incidence, type and severity of pregnancy complications in iTTP pregnant women
timeFrame: 6 years
Secondary outcomes:
measure: iTTP incidence in Italy
timeFrame: 3 years
Other outcomes:
Locations:
city: Milan
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
state: MI
Study officials:
affiliation: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy
name: Flora Peyvandi, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: