Residual Disease Driven Strategy for CARCIK (CD19) in Adults/Pediatric BCP-ALL

Acute Lymphoblastic Leukemia Clinical Trial

Official title:

Measurable Residual Disease Driven Strategy for One or Two Infusions of Non- Viral, Transposon-manipulated CARCIK (CD19) Cells: A Phase II Study in Pediatric and Adult Patients With Relapsed/Refractory B Cell Precursor ALL (BCP-ALL)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05252403
• Other study ID #: FT03CARCIK
• Status: Recruiting
• Phase: Phase 2
• Start date: December 1, 2021
• Est. completion date: March 2025

Study information

• Verified date: May 2023
• Source: Fondazione Matilde Tettamanti Menotti De Marchi Onlus
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a single arm, open-label, multi-center, phase II study to determine the activity and the safety of a therapeutic strategy that allows a second CARCIK-CD19 cells infusion, driven by the status of disease from one month after the first infusion, in adult and pediatric patients with r/r BCP- ALL.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 35
• Est. completion date: March 2025
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 75 Years

Eligibility

Inclusion Criteria:

Children (1-17) and adults (18-75 years old);

• Relapsed or refractory adult and pediatric B- ALL as defined for the presence of bone marrow with = 5% lymphoblasts by morphologic assessment, or if <5%, with at least 1% of molecular disease at PCR;
• Evidence of CD19 tumor expression in bone marrow and/or peripheral blood by flow cytometry;
• Diagnosis of CD19 positive ALL in the bone marrow, and/or peripheral blood and/or extramedullary sites with the exclusion of Central Nervous System (CNS) if CNS-3 disease.

Exclusion Criteria:

• GVHD Grades II-IV for patients who had previously been transplanted; 2. Any cell therapy in the previous 30 days;
• Patient with concomitant life-threatening infectious disease;
• Lansky/Karnofsky score <60;
• Patients with hepatic or renal disease as specific above;
• Pregnant or breast-feeding females;
• Rapidly progressive disease that in the estimation of the investigator and sponsor would compromise ability to complete study therapy;

Related Conditions & MeSH terms

• Acute Lymphoblastic Leukemia
• Precursor Cell Lymphoblastic Leukemia-Lymphoma

Intervention

Genetic:
• PTG-CARCIK-CD19
PTG-CARCIK-CD19 cells is a gene therapy medicinal product (GTMP) composed of genetically modified allogeneic T lymphocytes formulated as cell suspension in normal saline solution and freezing media

Locations

Country	Name	City	State
Italy	Ospedale PG23	Bergamo
Italy	Fondazione MBBM	Monza

Sponsors (1)

Lead Sponsor	Collaborator
Fondazione Matilde Tettamanti Menotti De Marchi Onlus

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the overall response rate: change from baseline at day 28 after the first CARCIK-CD19 infusion	At baseline and at Day 28 after infusion: lymphoblasts < 5% at morphological evaluation of bone marrow aspirate	Baseline and day 28
Primary	To evaluate the overall response rate: change from baseline at day 28 after the first CARCIK-CD19 infusion	At baseline and at Day 28 after infusion: lymphoblasts < 5% at flow cytometry bone marrow evaluation	Baseline and day 28
Primary	To evaluate the overall response rate: change from baseline at day 28 after the first CARCIK-CD19 infusion	At baseline and at Day 28 after infusion: molecular evaluation of disease positivity <0.01%	Baseline and day 28
Primary	To evaluate the duration of response of patients treated with CARCIK - CD19 cells. Change from day 28 at month 2, 3, 4, 5, 6, 9 and 12	Day 28 and month 2, 3, 4, 5, 6, 9 and 12: lymphoblasts < 5% at morphological evaluation of bone marrow aspirate	Day 28 and month 2, 3, 4, 5, 6, 9 and 12
Primary	To evaluate the duration of response of patients treated with CARCIK - CD19 cells. Change from day 28 at month 2, 3, 4, 5, 6, 9 and 12	Day 28 and month 2, 3, 4, 5, 6, 9 and 12: lymphoblasts < 5% at flow cytometry bone marrow evaluation	Day 28 and month 2, 3, 4, 5, 6, 9 and 12
Primary	To evaluate the duration of response of patients treated with CARCIK - CD19 cells. Change from day 28 at month 2, 3, 4, 5, 6, 9 and 12	Day 28 and month 2, 3, 4, 5, 6, 9 and 12: molecular evaluation of disease positivity <0.01%	Day 28 and month 2, 3, 4, 5, 6, 9 and 12