NCT07205861 Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center
| NCT ID | NCT07205861 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Assistance Publique - Hôpitaux de Paris |
| Condition | Thrombotic Thrombocytopenic Purpura (TTP) |
| Study Type | OBSERVATIONAL |
| Enrollment | 1,200 participants |
| Start Date | 2024-12-02 |
| Primary Completion | 2027-12 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 1,200 participants in total. It began in 2024-12-02 with a primary completion date of 2027-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening disorder characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and ischemic organ damage due to microvascular thrombosis. It results from a severe deficiency in the von Willeband factor (vWF)-cleaving protease ADAMTS13, primarily caused by autoantibodies that inhibit its activity. This deficiency leads to accumulation of ultra-large vWF multimers, triggering pathological platelet aggregation and widespread microthrombi. iTTP typically presents with acute neurological symptoms (e.g., confusion, seizures, coma), cardiac events (e.g., myocardial infarction), and multiorgan dysfunction. Without prompt treatment-plasma exchange, immunosuppression, and the vWF inhibitor caplacizumab-mortality exceeds 90%. Survivors face long-term risks, including cardiovascular complications, cognitive impairment, and reduced life expectancy. The TWI-LIGHT protocol is a national retrospective epidemiological study coordinated by the French Reference Center for Thrombotic Microangiopathies (CNR-MAT). It aims to analyze long-term outcome in \>1,200 iTTP patients diagnosed between October 2000 and June 2024. The study leverages pseudonymized data from the CNR-MAT registry, collected via a secure REDCap database. Key Objectives: 1. Primary: Assess the impact of cardiovascular risk factors (e.g., hypertension, diabetes) and ADAMTS13 activity on life expectancy in iTTP survivors. 2. Secondary: * Evaluate disease burden in underrepresented groups (pregnant/postpartum women, children, elderly patients). * Analyze the influence of new therapies (caplacizumab, rituximab, recombinant ADAMTS13) on care pathways. * Identify prognostic factors and treatment practices. * Characterize neurocognitive outcomes and quality of life post-iTTP. Methodology: * Design: Non-interventional, retrospective (MR-004 compliance), using data from standard care. * Inclusion: Patients with confirmed iTTP (thrombocytopenia, hemolytic anemia, ADAMTS13 \<10%), diagnosed within the study period, and ≥1 year of follow-up. * Exclusion: Cancer-associated iTTP, severe sepsis, or patient opposition to data reuse. * Data Collection: Clinical, biological, and therapeutic variables from hospital/consultation records, including cardiovascular events, ADAMTS13 activity, and neurocognitive assessments. * Analysis: Kaplan-Meier survival curves and Cox regression models to identify risk factors for non-iTTP-related death. Expected Outcomes: * Prevalence of cardiovascular comorbidities and their correlation with ADAMTS13 activity. * Insights into iTTP subtypes (e.g., gestational, pediatric) and therapeutic efficacy. * Evidence-based strategies for personalized long-term management. Ethical Framework: * AP-HP-sponsored, with oversight from Sorbonne University's ethics committee. * Patients informed of data reuse with opt-out rights; data archived for 15 years. This landmark study will inform clinical guidelines, optimize survivor care, and address unmet needs in iTTP management through comprehensive, real-world data analysis.
Eligibility Criteria
Inclusion Criteria: -Patients with a diagnosis of immune mediated TTP Exclusion Criteria: * Cancer- associated iTTP and HIV-associated iTTP * Severe sepsis * Disseminated intravascular coagulation with consumption of coagulation factors; * Transplant-associated TTP * HIV-associated TTP (AIDS stage) * Patient not affiliated with a social security scheme * Patient or parent's objection to the reuse of their healthcare data for research
Contact & Investigator
Paul COPPO, MD, PHD
PRINCIPAL INVESTIGATOR
Assistance Publique - Hôpitaux de Paris
Frequently Asked Questions
Who can join the NCT07205861 clinical trial?
This trial is open to participants of all sexes, studying Thrombotic Thrombocytopenic Purpura (TTP). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07205861 currently recruiting?
Yes, NCT07205861 is actively recruiting participants. Contact the research team at aul.coppo@aphp.fr for enrollment information.
Where is the NCT07205861 trial being conducted?
This trial is being conducted at Paris, France.
Who is sponsoring the NCT07205861 clinical trial?
NCT07205861 is sponsored by Assistance Publique - Hôpitaux de Paris. The principal investigator is Paul COPPO, MD, PHD at Assistance Publique - Hôpitaux de Paris. The trial plans to enroll 1,200 participants.