Clinical trial • Phase IV • Haematology|Rare Disease

Exagamglogene autotemcel for Sickle cell disease|Transfusion-dependent β-thalassemia

Phase IV trial of Exagamglogene autotemcel for Sickle cell disease|Transfusion-dependent β-thalassemia. open-label, none/not specified-controlled.

Overview

open-label, none/not specified-controlled Phase IV trial across 5 sites in Italy, Belgium, Germany.

Recruits 53 paediatric patients.

Vulnerable Population: Consent is provided by subjects or legal representative or guardian (if applicable). Child, adolescent and parent subject information and informed consent form (ICF) documents are provided (country-specific child/adolescent/parent ICFs listed for Italy, Belgium and Germany).

{"criterion_text":"- Subjects or legal representative or guardian (if applicable) must sign and date informed consent form (ICF).\n- Subjects must have received CTX001 infusion."}

{"endpoint_text":"- New Malignancies","definition_or_measurement_approach":""}
{"endpoint_text":"- New or worsening hematologic disorders (e.g. immune-mediated cytopenias, aplastic anemia, primary immunodeficiencies)","definition_or_measurement_approach":""}
{"endpoint_text":"- All-cause mortality","definition_or_measurement_approach":""}
{"endpoint_text":"- All serious adverse events (SAEs) occurring up to 15 years after CTX001 infusion","definition_or_measurement_approach":"SAEs monitoring up to 15 years after CTX001 infusion"}
{"endpoint_text":"- CTX001-related AE's","definition_or_measurement_approach":""}

{"endpoint_text":"- TDT and SCD: Total Hemoglobin (Hb) concentration over time","definition_or_measurement_approach":""}
{"endpoint_text":"- TDT and SCD: Fetal Hemoglobin (HbF) concentration over time","definition_or_measurement_approach":""}
{"endpoint_text":"- TDT and SCD: Proportion of alleles with intended genetic modification present in peripheral blood over time","definition_or_measurement_approach":""}
{"endpoint_text":"- TDT and SCD: Proportion of alleles with intended genetic modification present in CD34+ cells of the bone marrow over time","definition_or_measurement_approach":""}
{"endpoint_text":"- TDT and SCD: Change in patient-reported outcome (PRO) over time in participants ≥18 years of age assessed using EuroQol quality of life scale (EQ-5D-5L) for participants from study 111,121 and 171 only","definition_or_measurement_approach":"Assessed using EuroQol quality of life scale (EQ-5D-5L) for specified participants (≥18 years) from studies 111, 121 and 171"}

Planned Sample Size: 53
Recruitment Window Months: 228
Consent Approach: Subjects or legal representative or guardian (if applicable) must sign and date an informed consent form (ICF). Country-specific subject information and ICF documents exist for adults, adolescents, children and parents (Italy: child/adolescent/parent/adult ICFs in Italian; Belgium: adult and pregnancy ICFs in English and French; Germany: adult, adolescent, child and parent ICFs in German).

Site Name: Association Hospitaliere De Bruxelles Hopital Universitaire Des Enfants Reine Fabiola
Department Name: Pediatric Hemato-Oncology
Principal Investigator Name: Laurence Dedeken
Principal Investigator Email: laurence.dedeken@huderf.be
Contact Person Name: Laurence Dedeken
Contact Person Email: laurence.dedeken@huderf.be

Site Name: Universitaetsklinikum Regensburg AöR
Department Name: Pediatric Hematology, Oncology and Stem cell transplantation
Principal Investigator Name: Selim Corbacioglu
Principal Investigator Email: selim.corbacioglu@klinik.uni-regensburg.de
Contact Person Name: Selim Corbacioglu
Contact Person Email: selim.corbacioglu@klinik.uni-regensburg.de

Site Name: Universitaetsklinikum Duesseldorf AöR
Department Name: Department of Pediatric Oncology, Hematology and Clinical Immunology
Principal Investigator Name: Roland Meisel
Principal Investigator Email: Meisel@med.uni-duesseldorf.de
Contact Person Name: Roland Meisel
Contact Person Email: Meisel@med.uni-duesseldorf.de

Investigational Product Name: Casgevy 4 - 13 x 10^6 cells/mL dispersion for infusion
Active Substance: Exagamglogene autotemcel
Modality: Cell therapy|Gene therapy
Routes Of Administration: Intravenous infusion
Route: Intravenous infusion
Authorisation Status: Marketing authorisation EU/1/23/1787/001
Orphan Designation: Yes
Maximum Dose: 20 (dose unit: Other)

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