Study of Patients With Thrombotic Microangiopathy Associated With Mitomycin C, Treated or Not Wit… (NCT06098378) | Clinical Trial Compass
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Study of Patients With Thrombotic Microangiopathy Associated With Mitomycin C, Treated or Not With Eculizumab
France30 participantsStarted 2023-06-07
Plain-language summary
Thrombotic microangiopathies (TMA) are defined as a triad combining mechanical hemolytic anemia, peripheral thrombocytopenia and ischemic organ damage.
Mitomycin C is an alkylating agent used as chemotherapy in adenocarcinomas of the breast, lung, pancreas, rectum and anal carcinoma. Mitomycin-C-induced TMA (m-TMA) is a potentially serious complication of chemotherapy: its estimated incidence ranges from 4 to 15% and its mortality exceeds 70%, with an estimated median survival of 2 months. This can also be responsible for kidney failure, sometimes requiring hemodialysis. The time to onset of m-TMA varies from one week to 15 months after the last infusion and is believed to depend on the cumulative dose of mitomycin C.
Eculizumab is a monoclonal antibody that binds to complement protein C5, blocking activation of the terminal complement pathway and formation of the membrane attack complex. This therapy has significantly changed the prognosis of patients with atypical hemolytic uremic syndrome (HUS), a disease in which complement activation plays a central role in TMA. Recently, a retrospective study suggested efficacy of eculizumab in TMA induced by gemcitabine, another chemotherapy, with normalization of platelets and LDH in 83% of patients, and partial or complete renal recovery in 67% and 17% of patients. These results provided arguments in favor of a potential benefit of complement-targeted therapies in TMA induced by certain chemotherapies. However, data on eculizumab in m-TMA remain extremely limited to date.
The objective of this study is to describe the clinical, biological and histological presentation of patients with m-TMA and their evolution after treatment with or without eculizumab.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Adult patients (\>=18 years)
* Having received treatment with mitomycin C (regardless of the method of administration and indication) between 01/01/1990 and 12/31/2023
* and having developed a picture of thrombotic microangiopathy attributed to mitomycin C:
* biological: defined as: thrombocytopenia \<150G/L and mechanical hemolytic anemia (at least 3 out of 4 criteria: hemoglobin \< 12g/dL, presence of schistocytes in blood smear, LDH \> 1N, collapsed haptoglobin (\< lower limit of the limit of laboratory normal)
* or renal: pathological diagnosis of thrombotic microangiopathy on renal biopsy
* having received or not treatment for the episode of microangiopathy, including or not complement inhibitors (Eculizumab).
* Subject not opposing, after information, the reuse of their data for the purposes of this research
Exclusion Criteria:
* Subject having expressed opposition to participating in the study
* Test for positive Shiga toxin
* ADAMST13 activity \<10%
* Thrombotic microangiopathy attributed to metastatic cancer (infiltration of bone marrow or circulating erythroblasts)
* Impossibility of providing the subject with informed information (difficulties in understanding the subject, etc.)
* Subject under judicial protection
* Subject under guardianship or curatorship
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Overall and renal survival after the m-TMA episode
Timeframe: Files analysed retrospectively from January 01, 1990 to December 31, 2023 will be examined