Study evaluating the efficacy of eculizumab in the treatment of gemcitabine-induced thrombotic microangiopathies.

Phase 1

• Conditions: Thrombotic microangiopathies induced by gemcitabine; MedDRA version: 20.0Level: PTClassification code 10043645Term: Thrombotic microangiopathySystem Organ Class: 10005329 - Blood and lymphatic system disorders; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2020-003168-22-FR
• Lead Sponsor: Rouen University Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-01
• Last Update Posted: 13 September 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 10

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 < 0.5 ml/kg/h for 12 hours.
5.Clinical and biological criteria for thrombotic microangiopathy: mechanical hemolytic anemia and/or tThrombocytopeniaPatient affiliated with a social security system,
6.Adult patient or representative of the adult patient who has read and understood the information letter and signed the consent form.
7.Woman of childbearing age with effective contraception as defined by the WHO (estrogen-progestin or intrauterine device or tubal ligation) for more than one month and a negative -HCG pregnancy test at baseline, for the duration of the study and for at least 5 months after the end of the treatment, or Postmenopausal woman (non-medically induced amenorrhea for at least 12 months prior to the baseline visit) or For men, use of protection during sexual intercourse for the duration of the study and for at least 60 days after the end of study treatment
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 :<br>1. to describe the clinical and biological evolution (time to hematological and renal remission) of gemcitabine-induced thrombotic microangiopathy<br>2. to clarify the role of the alternating complement pathway by assaying C5 and C5b9 in plasma<br>3. to clarify the role and incidence of the mutations involved in the alternate complement pathway<br>4. to evaluate the tolerance to eculizumab treatment in this indication<br>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