Italian iTTP Registry

NCT06376786 | Recruiting

• 1 other identifier: ItaliTTP
• Study Type: observational
• Target: 132
• Locations: 1 country
• Sites: 1

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.

Conditions

TTP - Thrombotic Thrombocytopenic Purpura

Keywords

ADAMTS13; Thrombotic microangiopathy

Outcome Measures

Primary Outcomes (34)

• Age at onset

  Age at the first acute iTTP episode in years

  3 years

• Sex

  3 years

• Birth Country/Region

  3 years

• Race

  3 years

• Blood group

  ABO/Rh blood group

  3 years

• BMI

  Body mass index in kg/m\^2

  3 years

• Proportion of patients with comorbidities, including: autoimmune diseases, cancer, HIV infection, hypertension, type 2 diabetes, hypercholesterolemia, cardiovascular disease, chronic renal failure, liver disease, depression.

  Proportion of iTTP patients with comorbidities

  3 years

• Proportion of acute iTTP episodes preceded by potential triggering factors including: infections, pregnancy, surgery, psychological trauma, vaccination, drugs

  Proportion of potential triggering conditions/events/drugs occured/taken in the 3 months prior the acute iTTP episode

  3 years

• Incidence, type and severity of clinical manifestations, including: bleeding, cardiovascular, neurological, renal and systemic signs and symptoms

  Incidence, type and severity of clinical manifestations at presentation of the acute iTTP episode

  3 years

• Platelet count lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin

  Platelet count at presentation of the acute iTTP episode, expressed in number x 10\^9/L

  3 years

• Hemoglobin lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin

  Hemoglobin level at presentation of the acute iTTP episode, expressed in g/dL

  3 years

• Lactate dehydrogenase (LDH) lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin

  LDH level at presentation of the acute iTTP episode, expressed in IU/L

  3 years

• Creatinine lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin

  Creatinine level at presentation of the acute iTTP episode, expressed in mg/dL

  3 years

• Cardiac troponin

  Cardiac troponin level at presentation of the acute iTTP episode, expressed in ng/L

  3 years

• ADAMTS13 activity

  Level of functional ADAMTS13 activity expressed in IU/dL or %

  6 years

• Anti-ADAMTS13 antibodies

  Concentration or presence/absence of anti-ADAMTS13 antibodies

  6 years

• Number of daily therapeutic plasma exchange procedures

  Number of daily therapeutic plasma exchange procedures to achieve clinical response of the acute iTTP episode

  3 years

• Proportion of acute iTTP patients treated with rituximab

  6 years

• Proportion of acute iTTP patients treated with immunosuppressors other than steroids and rituximab

  6 years

• Proportion of iTTP patients treated with caplacizumab

  3 years

• Incidence, type and severity of TTP-related drugs adverse events

  Incidence, type and severity of TTP-related drugs adverse events recorded during the acute iTTP episode and disease remission of iTTP patients

  6 years

• Proportion of iTTP patients achieving clinical remission

  Proportion of iTTP patients achieving clinical remission defined as sustained clinical response with either no therapeutic plasma exchange (TPE) and no anti-von Willebrand factor (VWF) therapy for ≥ 30 days or with attainment of ADAMTS13 remission, whichever occurs first.

  6 years

• Proportion of iTTP patients refractory to acute iTTP treatment

  Proportion of iTTP patients refractory to acute iTTP treatment. Refractoriness defined as persistent thrombocytopenia and a persistently raised LDH level despite treatment.

  6 years

• Proportion of iTTP patients experiencing complications during hospitalization, including: bleeding, thrombosis, neurological, renal, cardiac complications

  Proportion of patients who experience complications during the hospitalization for acute iTTP

  6 years

• Proportion of iTTP patients experiencing clinical exacerbation

  Proportion of iTTP patients experiencing clinical exacerbation defined as sustained platelet count ≥ 150 × 109/L (or above the local lower limit of normal \[LLN\]) and LDH \< 1.5 times hte upper limit of normal (ULN) and no clinical evidence of new or progressive ischemic organ injury.

  6 years

• Proportion of iTTP patients achieving ADAMTS13 remission

  Proportion of iTTP patients achieving ADAMTS13 remission defined as ADAMTS13 activity ≥ 20% to \< LLN (partial) or ADAMTS13 activity ≥ LLN (complete).

  6 years

• Time to clinical response

  6 years

• Time to clinical remission

  6 years

• Time to ADAMTS13 remission

  6 years

• Proportion of iTTP patients with a clinical relapse

  Proportion of iTTP patients with a clinical relapse defined as a platelet count decrease to \< 150 × 109/L (with other causes of thrombocytopenia ruled out), with or without clinical evidence of new ischemic organ injury, after a clinical remission.

  6 years

• Proportion of iTTP patients with an ADAMTS13 relapse

  Proportion of iTTP patients with an ADAMTS13 relapse defined as a decrease of ADAMTS13 activity to \< 20% after a partial or complete ADAMTS13 remission.

  6 years

• Time to clinical relapse

  6 years

• Time to ADAMTS13 relapse

  6 years

• Incidence, type and severity of pregnancy complications in iTTP pregnant women

  6 years

Secondary Outcomes (1)

• iTTP incidence in Italy

  3 years

Study Arms (1)

iTTP patients

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

Eligibility Criteria

• Age: 12 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Fondazione Luigi Villa: lead

Study Sites (1)

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, MI, 20122, Italy

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic; Thrombotic Microangiopathies

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Thrombophilia; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Flora Peyvandi, MD, PhD

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

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sandra Maccarone

CONTACT

+39 02 551 0709; contact@fondazioneluigivilla.org

Ilaria Mancini, MSc, PhD

CONTACT

+39 02 5503 5414; ilaria.mancini@guest.unimi.it

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 3 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2024
• First Posted: April 19, 2024
• Study Start: June 20, 2024
• Primary Completion (Estimated): May 31, 2030
• Study Completion (Estimated): May 31, 2030
• Last Updated: July 10, 2024

Record last verified: 2024-04

Locations

IT (1)