ClinicalTrials.gov record NCT07205861

Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

Auto-immune Thrombotic Thrombocytopenic Purpura : Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center, TWI-LIGHT

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
SexAll
LocationFrance
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a retrospective, non-interventional study using the French CNR-MAT registry to analyze outcomes in people with autoimmune (immune) TTP diagnosed between October 1, 2000 and June 1, 2024. It plans to include data from over 1,200 patients and focuses on cardiovascular events, ADAMTS13 activity, neurocognitive outcomes, and treatment patterns. The study uses existing hospital and consultation records and does not assign any treatments.

What it is studying. Whether cardiovascular risk factors and ADAMTS13 activity are associated with life expectancy and cardiovascular events after iTTP, and how subgroups (pregnancy-related, pediatric, elderly) and newer therapies relate to long-term outcomes.

What the researchers hope to learn. To describe the prevalence of cardiovascular comorbidities after iTTP; to examine links between remission ADAMTS13 activity and later events or life expectancy; to characterize pregnancy-, childhood-, and elderly-onset iTTP; to evaluate real-world use of newer therapies (caplacizumab, rituximab, recombinant ADAMTS13); and to provide data to inform long-term monitoring and management recommendations.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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:

  • Primary: Assess the impact of cardiovascular risk factors (e.g., hypertension, diabetes) and ADAMTS13 activity on life expectancy in iTTP survivors.
  • 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

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

The TWI-LIGHT protocol is a retrospective, non-interventional epidemiological study coordinated by the French National Reference Center for Thrombotic Microangiopathies (CNR-MAT). It leverages the CNR-MAT registry to analyze long-term outcomes in over 1,200 patients diagnosed with autoimmune thrombotic thrombocytopenic purpura (iTTP) between October 1, 2000, and June 1, 2024. Background and Rationale iTTP is a rare, life-threatening disorder caused by severe ADAMTS13 deficiency, leading to microvascular thrombosis, multiorgan damage, and high mortality without prompt treatment. Survivors face long-term risks, including cardiovascular complications, cognitive impairment, and reduced life expectancy. Despite therapeutic advances (e.g., caplacizumab, rituximab), data on late complications, risk factors, and disease burden in underrepresented subgroups (e.g., pregnant women, children, elderly patients) remain limited. This study addresses critical gaps in understanding iTTP's natural history, treatment efficacy, and survival. Study Design

  • Type: Retrospective, multicenter, non-interventional (MR-004 compliance).
  • Data Collected:
  • Acute Phase: Clinical presentation, lab results (standard biology, including ADAMTS13 activity, troponin), treatments (plasma exchange, immunosuppressants, caplacizumab).
  • Long-Term Follow-Up: Cardiovascular events (major adverse cardiovascular events, including ischemic stroke, myocardial infarction and angioplasty) and cardiovascular risk factors (hypertension, diabetes, weight and body mass index, dyslipidemia, smoking), ADAMTS13 activity trends, neurocognitive outcomes (quality-of-life scales: SF36, HADS), relapse rates, and mortality.
  • Biobanking: ADAMTS13 samples stored at Hôpital Lariboisière (Prof. Veyradier's lab) under ethical approval (AC-2023-6021).

Expected Outcomes and Impact

  • Clinical Insights:
  • Personalized cardiovascular risk profiles for iTTP survivors.
  • Evidence-based guidelines for pregnancy, elderly, and pediatric iTTP management.
  • Optimization of long-term monitoring (e.g., rituximab frequency).
  • Health System Impact:
  • Cost-benefit analysis of novel therapies (e.g., caplacizumab, recombinant ADAMTS13).
  • Strategies to reduce diagnostic delays and hospitalizations. Real-World Relevance: Focus on underrepresented groups and long-term survival, with the goal to improve life expectancy of iTTP patients. This protocol represents a landmark effort to transform iTTP management through comprehensive real-world data, directly impacting clinical practice.
  • Methodological Rigor: Centralized data auditing, ISO/IEC 17025-compliant biobanking.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a retrospective, non-interventional study that uses existing hospital and clinic records and stored biological samples already in the CNR‑MAT registry. Data will be pseudonymized and entered into a secure REDCap database; some ADAMTS13 samples are stored in a central biobank. No study-assigned treatments or extra clinic visits are required as part of this research. Patients are informed about data reuse and have the right to object; study data will be archived for the period specified in the record.

How the study is structured. A retrospective, non-interventional review of existing medical records in a single national cohort. The study analyzes pseudonymized registry data from diagnosed iTTP patients, performs subgroup analyses (pregnancy/postpartum, children, elderly), and uses survival analysis and regression to identify factors linked to outcomes.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in France. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on thrombotic thrombocytopenic purpura

Clinical trials18 trials listed for thrombotic thrombocytopenic purpuraEvery trial the registry lists for thrombotic thrombocytopenic purpura, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in thrombotic thrombocytopenic purpura researchLatest monthly update: September 2026. Includes: Caplacizumab use. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Dec 2, 2024
Primary completion
December 2027 (estimated)
Study completion
December 2028 (estimated)
Target enrollment
1200 participants (estimated)
Last updated on registry
Oct 3, 2025
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Assistance Publique - Hôpitaux de Paris

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

life expectancy
Some studies require that, in the investigator's judgement, a participant is likely to live long enough to complete the study period. It is a study requirement, not a prediction about any individual.
registry
An organised collection of information about people with a particular condition, gathered over time for research. Joining a registry usually means sharing medical information, not receiving a treatment.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
natural history
A study that follows how a condition changes over time without assigning an experimental treatment. It may involve study visits and tests, and is often used to plan future trials.
immunosuppressants
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
Long-Term Follow-Up
Observation that continues for years after the study treatment, often required for gene and cell therapies.
relapse
A condition that improved with treatment and has since returned or worsened.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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