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

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)

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.

Study Overview

• Status: Recruiting
• Conditions: Acute Lymphoblastic Leukemia
• Intervention / Treatment: Genetic: PTG-CARCIK-CD19

• Study Type: Interventional
• Enrollment (Anticipated): 35
• Phase: Phase 2

Contacts and Locations

Study Locations

• Italy
  • Bergamo, Italy
    • Recruiting
    • Ospedale PG23
    • Contact: Alessandroi Rambaldi, MD; Phone Number: + 39 035 2673683; Email: arambaldi@asst-pg23.it
  • Monza, Italy
    • Recruiting
    • Fondazione MBBM
    • Contact: Andrea Biondi, MD; Phone Number: +39 039 2333661; Email: abiondi.unimib@gmail.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 75 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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;

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: CARCIK-CD19	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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
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
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
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

Collaborators and Investigators

• Sponsor: Fondazione Matilde Tettamanti Menotti De Marchi Onlus

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2021
• Primary Completion (Anticipated): December 1, 2024
• Study Completion (Anticipated): March 1, 2025

Study Registration Dates

• First Submitted: January 24, 2022
• First Submitted That Met QC Criteria: February 12, 2022
• First Posted (Actual): February 23, 2022

Study Record Updates

• Last Update Posted (Estimate): May 4, 2023
• Last Update Submitted That Met QC Criteria: May 3, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: ALL
• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Leukemia, Lymphoid; Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma
• Other Study ID Numbers: FT03CARCIK

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