Thrombotic microangiopathies induced by gemcitabine MedDRA version: 20.0 Level: PT Classification code 10043645 Term: Thrombotic microangiopathy System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Interventions
Trade Name: SOLIRIS®
Product Name: SOLIRIS®
Product Code: EU/1/07/393/001
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Eculizumab
CAS Number: 219685-50-4
Concentration unit: mg mil
Sponsors
Rouen University Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age = 18 years old 2. Previous treatment with gemcitabine within the last 18 months (duration of treatment = 3 consecutive months and cumulative dose should be = 10 grams). 3. Neoplasia in remission or not in remission but with an estimated life expectancy > 6 months 4. Acute renal failure defined by 1 of the following 2 criteria : Creatinine > 2 times baseline creatinine and/or diuresis =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Progressive neoplasia with a life expectancy of <6 months 2. Patient with a contraindication to the administration of the treatment experienced: SOLIRIS® 300 mg concentrate for solution for infusion 3. Contraindication to antibiotic prophylaxis 4. Thrombotic microangiopathy associated with cancer (metastatic adenocarcinoma with bone marrow invasion, erythromyelemia, disseminated intravascular coagulation) 5. Active systemic bacterial infection, untreated or confirmed sepsis (positive blood cultures within 7 days of patient inclusion and not treated with effective antibiotic therapy) 6. Unresolved meningococcal infection 7. Patient not vaccinated against meningococcal infection 8. Pregnant or breastfeeding woman or proven lack of contraception 9. Known systemic lupus erythematosus 10. Person deprived of liberty by an administrative or judicial decision or person placed under judicial protection, under guardianship or curatorship 11. Patient participating in another interventional clinical trial / having participated in another interventional clinical trial within 1 month
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The main objective is to study the evolution of renal function (occurrence of partial or total renal remission) in patients with gemcitabine-induced thrombotic microangiopathy treated with eculizumab.;Secondary Objective: The secondary objectives of the study are : 1. to describe the clinical and biological evolution (time to hematological and renal remission) of gemcitabine-induced thrombotic microangiopathy 2. to clarify the role of the alternating complement pathway by assaying C5 and C5b9 in plasma 3. to clarify the role and incidence of the mutations involved in the alternate complement pathway 4. to evaluate the tolerance to eculizumab treatment in this indication 5. To evaluate the quality of life of patients treated with eculizumab in this indication.;Primary end point(s): Time from initiation of eculizumab treatment to renal remission;Timepoint(s) of evaluation of this end point: During the 12 months of participation | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1- Number of patients in haematological remission: • Normalization of the platelet count> 150 giga / L and decrease in LDH, on 2 successive samples over a period of 4 weeks. • Time between diagnosis of TAD secondary to gemcitabine and start of treatment with eculizumab • Time between the start of treatment with eculizumab and haematological remission: normalization of the platelet count> 150 giga / L and decrease in LDH, on 2 successive samples over a period of 4 weeks. • Value of biological parameters: Haptoglobin, schizocytes, Hematocrit, Hemoglobin, reticulocytes, serum creatinine at M0, M1, M2, M3, M6 and M12 2- Number of patients with overexpression of C5b9 on the renal biopsy (if performed during treatment) and percentage of patients with overexpression of C5 and C5b9 in plasma 3- Number of patients with a mutation involved in the alternate complement pathway 4- Tolerance will be assessed by: • Number of red blood cells transfused before and after treatment with eculizumab • Duration of hospitalization in an intensive care unit / Resuscitation and total duration of hospitalization • Number of EvI and EvIG 5- Quality of life assessed by the SF-36 questionnaire ;Timepoint(s) of evaluation of this end point: During the 12 months of participation | — |
Countries
France
Contacts
Public ContactBLOT
University hospital center of Rouen
Outcome results
None listed