Italian iTTP Registry

TTP - Thrombotic Thrombocytopenic Purpura Clinical Trial

Official title:

Italian iTTP Registry (a Prospective Observational Study)

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06376786
• Other study ID #: ItaliTTP
• Status: Not yet recruiting
• Start date: June 1, 2024
• Est. completion date: May 31, 2030

Study information

• Verified date: April 2024
• Source: Fondazione Luigi Villa
• Contact: Sandra Maccarone
• Phone: +39 02 551 0709
• Email: contact[@]fondazioneluigivilla.org
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 132
• Est. completion date: May 31, 2030
• Est. primary completion date: May 31, 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 99 Years

Eligibility

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

Related Conditions & MeSH terms

• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombotic Thrombocytopenic
• TTP - Thrombotic Thrombocytopenic Purpura

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Fondazione Luigi Villa

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Age at onset	Age at the first acute iTTP episode in years	3 years
Primary	Sex		3 years
Primary	Birth Country/Region		3 years
Primary	Race		3 years
Primary	Blood group	ABO/Rh blood group	3 years
Primary	BMI	Body mass index in kg/m^2	3 years
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	Cardiac troponin	Cardiac troponin level at presentation of the acute iTTP episode, expressed in ng/L	3 years
Primary	ADAMTS13 activity	Level of functional ADAMTS13 activity expressed in IU/dL or %	6 years
Primary	Anti-ADAMTS13 antibodies	Concentration or presence/absence of anti-ADAMTS13 antibodies	6 years
Primary	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
Primary	Proportion of acute iTTP patients treated with rituximab		6 years
Primary	Proportion of acute iTTP patients treated with immunosuppressors other than steroids and rituximab		6 years
Primary	Proportion of iTTP patients treated with caplacizumab		3 years
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	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
Primary	Time to clinical response		6 years
Primary	Time to clinical remission		6 years
Primary	Time to ADAMTS13 remission		6 years
Primary	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
Primary	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
Primary	Time to clinical relapse		6 years
Primary	Time to ADAMTS13 relapse		6 years
Primary	Incidence, type and severity of pregnancy complications in iTTP pregnant women		6 years
Secondary	iTTP incidence in Italy	The number of all TTP events (first events and relapses) and first TTP events will be divided by the number of people at risk multiplied by the observation time to estimate the incidence rate of iTTP events and iTTP incident cases, respectively (in persons-years).	3 years