Clinical trial • Phase IV • Immunology|Oncology

Autologous enriched T cells retrovirally transduced to express two chimeric antigen receptors targeting CD19 and CD22 for Secondary malignancy following CAR T-cell therapy|Acute lymphoblastic leukemia|Non-Hodgkin's lymphoma|Systemic lupus erythematosus

Phase IV trial of Autologous enriched T cells retrovirally transduced to express two chimeric antigen receptors targeting CD19 and CD22 for Secondary mali…

Overview

Trial Therapeutic Area
Immunology|Oncology
Trial Disease
Secondary malignancy following CAR T-cell therapy|Acute lymphoblastic leukemia|Non-Hodgkin's lymphoma|Systemic lupus erythematosus
Trial Stage
Phase IV
Drug Modality
Cell therapy|Monoclonal antibody
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
13-03-2024
First CTIS Authorization Date
19-07-2024

Trial design

open-label, none/not specified-controlled Phase IV trial across 4 sites in Spain.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
90
Trial Duration For Participant
5475

Eligibility

Recruits 90 paediatric patients.

Vulnerable Population
Vulnerable populations included: children/adolescents. Study materials include child assent form, age-specific subject information and informed consent forms for 8 to 11 years and 12 to 17 years, Parent/Guardian ICFs and adolescent-to-adult re-consent letters; consent is obtained from the participant or from parent/guardian as applicable, with child assent for minors.

Inclusion criteria

  • {"criterion_text":"- Patients must have received an AUTO CAR T cell therapy on a previous treatment study.\n- Patients must have provided informed consent for long-term follow-up study prior to participation.\n- Patients must be able to comply with the study requirements."}

Exclusion criteria

  • {"criterion_text":"- There are no exclusion criteria for this study."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Incidence of serious adverse events related to AUTO CAR T cell therapy","definition_or_measurement_approach":"Measured as the incidence (occurrence) of serious adverse events judged related to AUTO CAR T cell therapy over follow-up."}
  • {"endpoint_text":"- New malignancies","definition_or_measurement_approach":"Identification and recording of any new malignancies occurring during follow-up."}
  • {"endpoint_text":"- Other designated adverse events of special interest related to AUTO CAR T cell therapy","definition_or_measurement_approach":"Recording and monitoring of predefined adverse events of special interest related to AUTO CAR T cell therapy."}

Secondary endpoints

  • {"endpoint_text":"- Overall survival for up to 15 years after the first AUTO CAR T cell therapy infusion","definition_or_measurement_approach":"Overall survival measured up to 15 years from the first AUTO CAR T infusion."}
  • {"endpoint_text":"- Duration of supportive care","definition_or_measurement_approach":"Length of time patients require supportive care interventions (no further detail provided)."}
  • {"endpoint_text":"- Duration of response, progression-free survival","definition_or_measurement_approach":"Duration of clinical response and progression-free survival as recorded during follow-up."}
  • {"endpoint_text":"- Proportion of patients with detectable vector copy number in peripheral blood for up to 15 years from first AUTO CAR T cell therapy infusion","definition_or_measurement_approach":"Proportion of patients with detectable vector copy number in peripheral blood, assessed over a 15-year period from first infusion."}
  • {"endpoint_text":"- Confirm / monitor for absence of detectable RCR/RCL for up to 15 years from first AUTO CAR T cell therapy infusion","definition_or_measurement_approach":"Periodic testing to confirm absence (or detect presence) of replication competent retrovirus (RCR) or lentivirus (RCL) up to 15 years."}
  • {"endpoint_text":"- In case of new malignancy: Insertion site analysis to determine insertional mutagenesis as potential cause/contributor","definition_or_measurement_approach":"If a new malignancy arises, perform insertion site analysis to assess whether insertional mutagenesis may be causative or contributory."}

Recruitment

Planned Sample Size
90
Recruitment Window Months
181
Consent Approach
Informed consent obtained prior to participation; age-specific information and consent/assent documents available (Parent/Guardian ICF, child assent form, SIS and ICF for 8-11 years and 12-17 years, adolescent-to-adult re-consent letters). There are additional ICFs for patient becoming pregnant and pregnant partner. Materials and translations include English and Spanish protocol synopses; consent is provided by participants or parent/guardian as applicable, with assent for minors.

Methods

  • Patients may be enrolled following their AUTO CAR T cell therapy treatment (clinic-based enrolment of previously treated AUTO CAR T cell therapy patients).

Geography

Total Number Of Sites
4
Total Number Of Participants
90

Spain

Earliest CTIS Part Ii Submission Date
11-07-2024
Latest Decision Or Authorization Date
26-06-2025
Processing Time Days
350
Number Of Sites
4
Number Of Participants
21

Sites

Site Name
Hospital Universitari Vall D Hebron
Department Name
Paediatric Haemato-Oncology
Contact Person Name
Gloria Lacoboni
Contact Person Email
giacoboni@vhio.net
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Hematology
Contact Person Name
Manuel Guerreiro
Contact Person Email
Manuel_direito@iislafe.es
Site Name
Hospital Universitari Vall d'Hebron
Department Name
Haematology
Contact Person Name
Josefina Cortes
Contact Person Email
fina.cortes@vhir.org
Site Name
Hospital Infantil Universitario Nino Jesus
Department Name
Paediatric Haemato-Oncology
Contact Person Name
Alba Rubio
Contact Person Email
alba.rubio@salud.madrid.org

Sponsor

Primary sponsor

Full Name
Autolus Limited
Organisation Type
Pharmaceutical company
Country Of Registered Address
United Kingdom

Investigational products

Investigational Product Name
AUTO1
Active Substance
Autologous enriched T cells retrovirally transduced to express two chimeric antigen receptors targeting CD19 and CD22
Modality
Cell therapy
Routes Of Administration
INTRAVENOUS INFUSION
Route
INTRAVENOUS INFUSION
Authorisation Status
1
Orphan Designation
Yes
Maximum Dose
100 U/g unit(s)/gram
Investigational Product Name
AUTO4
Active Substance
AUTO4
Modality
Cell therapy
Routes Of Administration
INTRAVENOUS INFUSION
Route
INTRAVENOUS INFUSION
Authorisation Status
1
Maximum Dose
25 U/g unit(s)/gram
Investigational Product Name
MabThera 100 mg concentrate for solution for infusion
Active Substance
Rituximab
Modality
Monoclonal antibody
Routes Of Administration
INTRAVENOUS INFUSION
Route
INTRAVENOUS INFUSION
Authorisation Status
2
Maximum Dose
375 mg/m2
Investigational Product Name
MabThera 500 mg concentrate for solution for infusion
Active Substance
Rituximab
Modality
Monoclonal antibody
Routes Of Administration
INTRAVENOUS INFUSION
Route
INTRAVENOUS INFUSION
Authorisation Status
2
Maximum Dose
375 mg/m2

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