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Multicenter, Uncontrolled Pilot Study Evaluating the Efficacy of Eculizumab in the Treatment of Gemcitabine-induced Thrombotic Microangiopathies (GEMECULI)

Primary Purpose

Thrombotic Microangiopathies

Status
Not yet recruiting
Phase
Phase 3
Locations
France
Study Type
Interventional
Intervention
Eculizumab administration
Sponsored by
University Hospital, Rouen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Thrombotic Microangiopathies focused on measuring Eculizumab, gemcitabine

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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 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

Sites / Locations

  • Amiens University Hospital
  • Rouen University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Patient with Thrombotic microangiopathies induced by gemcitabine

Arm Description

Patient with Thrombotic microangiopathies induced by gemcitabine will be treat with Eculizumab

Outcomes

Primary Outcome Measures

Time from initiation of eculizumab treatment to renal remission

Secondary Outcome Measures

Number of AE and SAE during the study
Number of red blood cells transfused before and after treatment with eculizumab

Full Information

First Posted
January 18, 2023
Last Updated
January 18, 2023
Sponsor
University Hospital, Rouen
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1. Study Identification

Unique Protocol Identification Number
NCT05702996
Brief Title
Multicenter, Uncontrolled Pilot Study Evaluating the Efficacy of Eculizumab in the Treatment of Gemcitabine-induced Thrombotic Microangiopathies
Acronym
GEMECULI
Official Title
Multicenter, Uncontrolled Pilot Study Evaluating the Efficacy of Eculizumab in the Treatment of Gemcitabine-induced Thrombotic Microangiopathies
Study Type
Interventional

2. Study Status

Record Verification Date
January 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
March 2023 (Anticipated)
Primary Completion Date
September 2025 (Anticipated)
Study Completion Date
September 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Rouen

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
The main objective of this prospective multisite trial is to study the evolution of TMA (thrombotic microangiopathy) induced by gemcitabin and treated by eculizumab.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thrombotic Microangiopathies
Keywords
Eculizumab, gemcitabine

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Patient with Thrombotic microangiopathies induced by gemcitabine
Arm Type
Experimental
Arm Description
Patient with Thrombotic microangiopathies induced by gemcitabine will be treat with Eculizumab
Intervention Type
Drug
Intervention Name(s)
Eculizumab administration
Intervention Description
Patient with Thrombotic microangiopathies induced by gemcitabine will be treat with Eculizumab
Primary Outcome Measure Information:
Title
Time from initiation of eculizumab treatment to renal remission
Time Frame
maximum 12 months
Secondary Outcome Measure Information:
Title
Number of AE and SAE during the study
Time Frame
12 months
Title
Number of red blood cells transfused before and after treatment with eculizumab
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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 < 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 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Steven GRANGE, MD
Phone
+3323288
Ext
8261
Email
steven.grange@chu-rouen.fr
First Name & Middle Initial & Last Name or Official Title & Degree
Florian VALLIN
Phone
+3323288
Ext
8265
Email
florian.vallin@chu-rouen.fr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Steven GRANGE, MD
Organizational Affiliation
Rouen University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Amiens University Hospital
City
Amiens
Country
France
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Claire PRESNE, MD
Facility Name
Rouen University Hospital
City
Rouen
Country
France
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Steven GRANGE, MD

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Multicenter, Uncontrolled Pilot Study Evaluating the Efficacy of Eculizumab in the Treatment of Gemcitabine-induced Thrombotic Microangiopathies

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