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

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

Study Overview

• Status: Not yet recruiting
• Conditions: Thrombotic Microangiopathies
• Intervention / Treatment: Drug: Eculizumab administration

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Steven GRANGE, MD; Phone Number: 8261 +3323288; Email: steven.grange@chu-rouen.fr
• Study Contact Backup: Name: Florian VALLIN; Phone Number: 8265 +3323288; Email: florian.vallin@chu-rouen.fr

Study Locations

• France
  • Amiens, France
    • Amiens university hospital
    • Contact: Claire PRESNE, MD
  • Rouen, France
    • Rouen University Hospital
    • Contact: Steven GRANGE, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Patient with Thrombotic microangiopathies induced by gemcitabine; Patient with Thrombotic microangiopathies induced by gemcitabine will be treat with Eculizumab	Drug: Eculizumab administration; Patient with Thrombotic microangiopathies induced by gemcitabine will be treat with Eculizumab

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Time from initiation of eculizumab treatment to renal remission	maximum 12 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of AE and SAE during the study	12 months
Number of red blood cells transfused before and after treatment with eculizumab	12 months

Collaborators and Investigators

• Sponsor: University Hospital, Rouen
• Investigators: Principal Investigator: Steven GRANGE, MD, Rouen University Hospital

Study record dates

Study Major Dates

• Study Start (Anticipated): March 1, 2023
• Primary Completion (Anticipated): September 1, 2025
• Study Completion (Anticipated): September 1, 2025

Study Registration Dates

• First Submitted: January 18, 2023
• First Submitted That Met QC Criteria: January 18, 2023
• First Posted (Actual): January 27, 2023

Study Record Updates

• Last Update Posted (Actual): January 27, 2023
• Last Update Submitted That Met QC Criteria: January 18, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Keywords: gemcitabine; Eculizumab
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Hematologic Diseases; Thrombocytopenia; Blood Platelet Disorders; Vascular Diseases; Thrombotic Microangiopathies; Physiological Effects of Drugs; Immunosuppressive Agents; Immunologic Factors; Complement Inactivating Agents; Eculizumab
• Other Study ID Numbers: 2020/0206/HP

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No