MICROVASCULAR DAMAGE IN THROMBOTIC THROMBOCYTOPENIC PURPURA

NCT07615244 — Not applicable, Active not recruiting, 79 participants, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

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STUDY OF MICROVASCULAR DAMAGE IN PATIENTS WITH THROMBOTIC THROMBOCYTOPENIC PURPURA

Registry	NCT07615244, acronym MITHO
Status	Active not recruiting
Phase	Not applicable
Study type	Interventional; non randomized, parallel, masking: none, diagnostic
Enrollment	79 (Estimated)
Sponsor	Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico (Other); collaborators: Istituto Auxologico Italiano, Centro Cardiologico Monzino, Politecnico di Milano
Conditions	TTP - Thrombotic Thrombocytopenic Purpura
Interventions	Factor VIII (FVIII), Von Willebrand factor (VWF) antigen, VWF:RCo, VWF-propeptide, VWF multimers, sP-selectin, sCD40L, F1+2, VEGF-A, VCAM, ICAM, IL-1β, IL-6, PTX3, BlyS, Neutrophil elastase and myeloperoxidase (NETs) markers, soluble C5b-9, C5a, NFL, GFAP, evaluation of thrombus formation with Total Thrombus formation Analysis System (T-TAS), Microfluidic Flow Chamber, Whole exome sequencing - WES, Proteomic Analysis (Diagnostic test, genetic)
Dates	start 2026-02-28, primary completion 2031-01-31 (estimated), completion 2032-01-31 (estimated), first posted 2026-05-29, last updated 2026-06-09
Locations	1 site in Italy

-Summary

The goal of this interventional, non-pharmacological clinical study is to assess microcirculatory dysfunction in patients with thrombotic thrombocytopenic purpura (TTP), both during the acute phase and in remission, compared with the general population.

Microcirculatory damage will be defined as the presence of at least one abnormal instrumental finding among brain magnetic resonance imaging (MRI), optical coherence tomography angiography (OCT-A), and nailfold videocapillaroscopy.

This is a monocentric, national study including two patient cohorts:

Cohort 1: patients enrolled during the acute phase Cohort 2: patients enrolled during remission Participants will be followed for three years with a total of five study visits.

Researchers will not test any drug but will perform detailed clinical, laboratory, and instrumental assessments to evaluate microvascular involvement over time.

Participants will:

* Undergo clinical evaluation, including internal medicine and neurological assessments
* Provide blood and urine samples for laboratory analyses
* Undergo instrumental examinations such as brain MRI, OCT-A, and nailfold videocapillaroscopy
* Receive psychological/neuropsychological evaluations
* Attend follow-up visits over a 3-year period (at baseline, 3 months \[cohort 1 only\], 12, 24, and 36 months)

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+Arms and interventions

Arm	Type	Interventions	Description
Acute TTP	Other	Diagnostic Test: Factor VIII (FVIII); Diagnostic Test: Von Willebrand factor (VWF) antigen; Diagnostic Test: VWF:RCo; Diagnostic Test: VWF-propeptide; Diagnostic Test: VWF multimers; Diagnostic Test: sP-selectin; Diagnostic Test: sCD40L; Diagnostic Test: F1+2; Diagnostic Test: VEGF-A; Diagnostic Test: VCAM; Diagnostic Test: ICAM; Diagnostic Test: IL-1β; Diagnostic Test: IL-6; Diagnostic Test: PTX3; Diagnostic Test: BlyS; Diagnostic Test: Neutrophil elastase and myeloperoxidase (NETs) markers; Diagnostic Test: soluble C5b-9; Diagnostic Test: C5a; Diagnostic Test: NFL; Diagnostic Test: GFAP; Genetic: Whole exome sequencing - WES; Diagnostic Test: Proteomic Analysis	Patients enrolled during acute phase (cohort 1), T0= within 15 days from hospital admission.
Remission TTP	Other	Diagnostic Test: Factor VIII (FVIII); Diagnostic Test: Von Willebrand factor (VWF) antigen; Diagnostic Test: VWF:RCo; Diagnostic Test: VWF-propeptide; Diagnostic Test: VWF multimers; Diagnostic Test: sP-selectin; Diagnostic Test: sCD40L; Diagnostic Test: F1+2; Diagnostic Test: VEGF-A; Diagnostic Test: VCAM; Diagnostic Test: ICAM; Diagnostic Test: IL-1β; Diagnostic Test: IL-6; Diagnostic Test: PTX3; Diagnostic Test: Neutrophil elastase and myeloperoxidase (NETs) markers; Diagnostic Test: soluble C5b-9; Diagnostic Test: C5a; Diagnostic Test: NFL; Diagnostic Test: GFAP; Diagnostic Test: evaluation of thrombus formation with Total Thrombus formation Analysis System (T-TAS); Diagnostic Test: Microfluidic Flow Chamber; Genetic: Whole exome sequencing - WES; Diagnostic Test: Proteomic Analysis	Patients enrolled during clinical remission (cohort 2), T0= check-up visit performed at more than 3 months from clinical response of the previous episode

Factor VIII (FVIII) (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
Von Willebrand factor (VWF) antigen (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
VWF:RCo (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
VWF-propeptide (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
VWF multimers (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
sP-selectin (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
sCD40L (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
F1+2 (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
VEGF-A (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
VCAM (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
ICAM (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
IL-1β (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
IL-6 (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
PTX3 (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
BlyS (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
Neutrophil elastase and myeloperoxidase (NETs) markers (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
soluble C5b-9 (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
C5a (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
NFL (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
GFAP (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
evaluation of thrombus formation with Total Thrombus formation Analysis System (T-TAS) (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
Microfluidic Flow Chamber (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
Whole exome sequencing - WES (Genetic): The study is defined as interventional because it includes assessments that are not part of routine clinical practice
Proteomic Analysis (Diagnostic test): The study is defined as interventional because it includes assessments that are not part of routine clinical practice

+Outcome measures

Primary outcomes

Prevalence [From enrollment to the last visit (after 36 months from Time 0)]: Prevalence of microcirculation damage in iTTP patients compared to general population

Secondary outcomes

Cerebral microcirculation lesions [From enrollment to the last visit (after 36 months from Time 0)]: Evaluate cerebral microcirculation lesions (via brain MRI) in both acute and remission phases of TTP to monitor organ damage progression over time
Retinal microcirculation changes [From enrollment to the last visit (after 36 months from Time 0)]: Assess retinal microcirculation changes (using OCT and OCT-A) during acute and remission phases of TTP to track organ damage evolution
Peripheral microcirculation [From enrollment to the last visit (after 36 months from Time 0)]: Analyze peripheral microcirculation (with nailfold videocapillaroscopy) in acute and remission phases to evaluate progression of organ involvement in TTP
Association between cerebral MRI lesions and cognitive or psychological symptoms [From enrollment to the last visit (36 months from Time 0)]: Explore the relationship between cerebral MRI lesions and cognitive or psychological symptoms during TTP remission, assessing symptom progression
Modification of preclinical biomarkers [From enrollment to the last visit (36 months from Time 0)]: Explore the modification of preclinical biomarkers over time at different stage of the disease
Associations between preclinical biomarkers and microcirculatory damage [From enrollment to the last visit (36 months from Time 0)]: Identify associations between preclinical biomarkers and microcirculatory damage in both acute and remission phases of TTP to predict risk and progression

+Eligibility

Sex: All, ages: 18 Years to any

Inclusion Criteria:

* aged ≥18 years
* diagnosis of TTP (either congenital or acquired immune-mediated TTP)
* referred at our hospital (Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, S.C. Medicina - Emostasi e Trombosi)
* providing a signed informed consent

Exclusion Criteria:

* Patients who do not possess the above inclusion criteria
* Patients who have known contraindications to the instrumental exam included in the study.

+Locations (1)

Facility	City	Country	Status
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico	Milan, Milano	Italy