TTP - Thrombotic Thrombocytopenic PurpuraNCT06376786
Italian iTTP Registry
An observational study of TTP - Thrombotic Thrombocytopenic Purpura, run by Fondazione Luigi Villa at 1 site in Italy.
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. Read more about this study
- Who is it for?
- Ages 12 to 99 years
- Conditions the study is for
- TTP - Thrombotic Thrombocytopenic Purpura
- How many people
- 132 participants
- Where
- 1 siteItaly
- Study dates
- 20 Jun 2024to 31 May 2030
- How long will I take part?
- 3 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Hospitalized patients with an acute event of iTTP (first event or relapse), then followed-up in outpatient clinics of the participating centers.
Who may be able to join
- 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
Who cannot join
- Patients who have not signed the informed consent for the participation to the study
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site, 1 recruiting
- Recruiting
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, MI, 20122
About this study
Official title: Italian iTTP Registry (a Prospective Observational Study)
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.
Read the 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.
- Keywords
- ADAMTS13, Thrombotic microangiopathy
- Medical subject headings
- Purpura, Thrombotic Thrombocytopenic, Thrombotic Microangiopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| iTTP patients | iTTP patients will be treated and followed-up as per standard clinical practice. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Age at onset
Age at the first acute iTTP episode in years
Measured: 3 years
Sex
Measured: 3 years
Birth Country/Region
Measured: 3 years
Race
Measured: 3 years
Blood group
ABO/Rh blood group
Measured: 3 years
BMI
Body mass index in kg/m^2
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 3 years
Cardiac troponin
Cardiac troponin level at presentation of the acute iTTP episode, expressed in ng/L
Measured: 3 years
ADAMTS13 activity
Level of functional ADAMTS13 activity expressed in IU/dL or %
Measured: 6 years
Anti-ADAMTS13 antibodies
Concentration or presence/absence of anti-ADAMTS13 antibodies
Measured: 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
Measured: 3 years
Proportion of acute iTTP patients treated with rituximab
Measured: 6 years
Proportion of acute iTTP patients treated with immunosuppressors other than steroids and rituximab
Measured: 6 years
Proportion of iTTP patients treated with caplacizumab
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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).
Measured: 6 years
Time to clinical response
Measured: 6 years
Time to clinical remission
Measured: 6 years
Time to ADAMTS13 remission
Measured: 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.
Measured: 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.
Measured: 6 years
Time to clinical relapse
Measured: 6 years
Time to ADAMTS13 relapse
Measured: 6 years
Incidence, type and severity of pregnancy complications in iTTP pregnant women
Measured: 6 years
Secondary outcomes (1)
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).
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Apr 2024
- Study start20 Jun 2024
- Primary completion31 May 2030 (estimated)
- Study completion31 May 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for TTP - Thrombotic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 132 participants (estimated)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- Fondazione Luigi Villa
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Flora Peyvandi, MD, PhD, Principal investigator, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06376786
- Sponsor's protocol ID
- ItaliTTP
- Status last verified
- Apr 2024
- First submitted
- 15 Mar 2024
- Last updated
- 10 Jul 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Italian iTTP Registry (a Prospective Observational Study)
- ClinicalTrials.gov ID
- NCT06376786
- Acronym / sponsor ID
- ItaliTTP
- Keywords
- ADAMTS13, Thrombotic microangiopathy
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 132 participants (estimated)
- Lead sponsor
- Fondazione Luigi VillaUniversity, hospital or other
- Study start
- 20 Jun 2024 (actual)
- Primary completion
- 31 May 2030 (estimated)
- Study completion
- 31 May 2030 (estimated)
- First posted
- 19 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 3 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| iTTP patients | – | –iTTP patients will be treated and followed-up as per standard clinical practice. |
Who may qualify
- Ages
- 12 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Hospitalized patients with an acute event of iTTP (first event or relapse), then followed-up in outpatient clinics of the participating centers.
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (1 listed)
- Patients who have not signed the informed consent for the participation to the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Age at onset
Time frame: 3 years
Age at the first acute iTTP episode in years
Sex
Time frame: 3 years
Birth Country/Region
Time frame: 3 years
Race
Time frame: 3 years
Blood group
Time frame: 3 years
ABO/Rh blood group
BMI
Time frame: 3 years
Body mass index in kg/m^2
Proportion of patients with comorbidities, including: autoimmune diseases, cancer, HIV infection, hypertension, type 2 diabetes, hypercholesterolemia, cardiovascular disease, chronic renal failure, liver disease, depression.
Time frame: 3 years
Proportion of iTTP patients with comorbidities
Proportion of acute iTTP episodes preceded by potential triggering factors including: infections, pregnancy, surgery, psychological trauma, vaccination, drugs
Time frame: 3 years
Proportion of potential triggering conditions/events/drugs occured/taken in the 3 months prior the acute iTTP episode
Incidence, type and severity of clinical manifestations, including: bleeding, cardiovascular, neurological, renal and systemic signs and symptoms
Time frame: 3 years
Incidence, type and severity of clinical manifestations at presentation of the acute iTTP episode
Platelet count lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
Time frame: 3 years
Platelet count at presentation of the acute iTTP episode, expressed in number x 10^9/L
Hemoglobin lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
Time frame: 3 years
Hemoglobin level at presentation of the acute iTTP episode, expressed in g/dL
Lactate dehydrogenase (LDH) lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
Time frame: 3 years
LDH level at presentation of the acute iTTP episode, expressed in IU/L
Creatinine lactate dehydrogenase (LDH), total and indirect bilirubin, liver transaminases, creatinine, troponin
Time frame: 3 years
Creatinine level at presentation of the acute iTTP episode, expressed in mg/dL
Cardiac troponin
Time frame: 3 years
Cardiac troponin level at presentation of the acute iTTP episode, expressed in ng/L
ADAMTS13 activity
Time frame: 6 years
Level of functional ADAMTS13 activity expressed in IU/dL or %
Anti-ADAMTS13 antibodies
Time frame: 6 years
Concentration or presence/absence of anti-ADAMTS13 antibodies
Number of daily therapeutic plasma exchange procedures
Time frame: 3 years
Number of daily therapeutic plasma exchange procedures to achieve clinical response of the acute iTTP episode
Proportion of acute iTTP patients treated with rituximab
Time frame: 6 years
Proportion of acute iTTP patients treated with immunosuppressors other than steroids and rituximab
Time frame: 6 years
Proportion of iTTP patients treated with caplacizumab
Time frame: 3 years
Incidence, type and severity of TTP-related drugs adverse events
Time frame: 6 years
Incidence, type and severity of TTP-related drugs adverse events recorded during the acute iTTP episode and disease remission of iTTP patients
Proportion of iTTP patients achieving clinical remission
Time frame: 6 years
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.
Proportion of iTTP patients refractory to acute iTTP treatment
Time frame: 6 years
Proportion of iTTP patients refractory to acute iTTP treatment. Refractoriness defined as persistent thrombocytopenia and a persistently raised LDH level despite treatment.
Proportion of iTTP patients experiencing complications during hospitalization, including: bleeding, thrombosis, neurological, renal, cardiac complications
Time frame: 6 years
Proportion of patients who experience complications during the hospitalization for acute iTTP
Proportion of iTTP patients experiencing clinical exacerbation
Time frame: 6 years
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.
Proportion of iTTP patients achieving ADAMTS13 remission
Time frame: 6 years
Proportion of iTTP patients achieving ADAMTS13 remission defined as ADAMTS13 activity ≥ 20% to < LLN (partial) or ADAMTS13 activity ≥ LLN (complete).
Time to clinical response
Time frame: 6 years
Time to clinical remission
Time frame: 6 years
Time to ADAMTS13 remission
Time frame: 6 years
Proportion of iTTP patients with a clinical relapse
Time frame: 6 years
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.
Proportion of iTTP patients with an ADAMTS13 relapse
Time frame: 6 years
Proportion of iTTP patients with an ADAMTS13 relapse defined as a decrease of ADAMTS13 activity to < 20% after a partial or complete ADAMTS13 remission.
Time to clinical relapse
Time frame: 6 years
Time to ADAMTS13 relapse
Time frame: 6 years
Incidence, type and severity of pregnancy complications in iTTP pregnant women
Time frame: 6 years
1 secondary outcome
iTTP incidence in Italy
Time frame: 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Flora Peyvandi, MD, PhD principal investigator
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milano, Italy
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of TTP - Thrombotic Thrombocytopenic Purpura, run by Fondazione Luigi Villa at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sandra Maccarone (+39 02 551 0709, contact@fondazioneluigivilla.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Age at onset (time frame: 3 years). The study lists 34 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jul 2024.