Study of Patients With Thrombotic Microangiopathy Associated With Mitomycin C, Treated or Not With Eculizumab

NCT ID: NCT06098378

Last Updated: 2023-10-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

30 participants

Study Classification

OBSERVATIONAL

Study Start Date

2023-06-07

Study Completion Date

2025-06-07

Brief Summary

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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.

Detailed Description

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Conditions

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Thrombotic Microangiopathies

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

RETROSPECTIVE

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospital, Strasbourg, France

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Service de Néphrologie, Dialyse et Transplantation - CHU de Strasbourg - France

Strasbourg, , France

Site Status RECRUITING

Countries

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France

Central Contacts

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Anna DUVAL, MD

Role: CONTACT

33 3 69 55 05 11

Facility Contacts

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Anna DUVAL, MD

Role: primary

33 3 69 55 05 11

Other Identifiers

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8957

Identifier Type: -

Identifier Source: org_study_id

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