A Study to Evaluate the Long-term Safety of Patients With Advanced Lymphoid Leukemia Who Have Been Previously Administered With UCART19

Long-term Follow-up Study of Patients Who Have Previously Been Exposed to UCART19 (Allogeneic Engineered T-cells Expressing a Lentiviral-based Anti-CD19 Chimeric Antigen Receptor)

The purpose of this study is to evaluate the long-term safety and efficacy of UCART19 administration to patients with advanced lymphoid leukemia.

Study Overview

• Status: Active, not recruiting
• Conditions: Advanced Lymphoid Leukemia
• Intervention / Treatment: Biological: UCART19 follow-up

• Study Type: Interventional
• Enrollment (Actual): 28
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Institut de Recherches Internationales Servier; Phone Number: +33 1 55 72 43 66; Email: scientificinformation@servier.com

Study Locations

• France
  • PARIS Cedex 12, France, 75571
    • Hôpital Saint-Antoine
  • Paris, France, 75010
    • Hôpital Saint-Louis
  • Paris, France, 75019
    • Hôpital Robert-Debré
• Japan
  • Fukuoka, Japan, 812-8582
    • Kyushyu University Hospital
  • Sapporo, Japan, 060-8648
    • Hokkaido University Hospital
• Spain
  • Barcelona, Spain, 08950
    • Hospital San Juan de Dios
• United Kingdom
  • London, United Kingdom, SE5 9RS
    • King's College Hospital NHS Foundation Trust
  • London, United Kingdom
    • UCL Great Ormond Hospital
  • Manchester, United Kingdom, M20 4BX
    • The Christie NHS Foundation Trust
• United States
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital Los Angeles
  • Colorado
    • Denver, Colorado, United States, 80219
      • Colorado Blood Cancer Institute
  • Florida
    • Tampa, Florida, United States, 33612
      • Moffit Cancer Center
  • Massachusetts
    • Charlestown, Massachusetts, United States, 02129
      • Massachussetts General Hospital
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Texas
    • Dallas, Texas, United States, 75235
      • University of Texas Southwestern Medical Center
    • Houston, Texas, United States, 77030
      • University of Texas MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Written informed consent obtained prior any study-specific procedure (patient or parent(s) or legal representative)
• Patient dosed with UCART19 who completed or discontinued early from a sponsored or from any investigator-initiated study that tested UCART19, or patients who were administered UCART19 under a special access scheme (compassionate use);
• Female patients of childbearing potential and male patients with partners of childbearing potential must continue to use an effective method of birth control as well as their partners for a 12-month duration after the last UCART19 administration.

Exclusion Criteria:

- No exclusion criteria for this study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• 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: UCART19 follow-up	Biological: UCART19 follow-up; UCART19 will not be administered during the study period. Patients who will be rolled-over from the parent study to this long term follow-up study, have previously received UCART19. The roll-over occurs at the end of the parent study, or at any time after UCART19 administration, in case of premature discontinuation from the parent study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Long-term safety of UCART19 with or without alemtuzumab	Number, duration, outcome of all adverse events (AE) within 12 months post last UCART19 infusion; Number, duration, outcome of adverse events of special interest (AESI) up to the end of the study; Proportion of patients with adverse events leading to death up to the end of the study; For paediatric patients: assesment of the potential impact on growth curve and puberty	Up to 15 Year

Secondary Outcome Measures

Outcome Measure	Time Frame
Assessment of long-term anti tumor activity of UCART19	Month 3, Month 6, Month 12 during Year 1 following last UCART19 dosing then every 6 months up to Year 3, then yearly up to Year 15
Proportion of patients who underwent allogeneic HSCT (Hematopoietic Stem Cell Transplantation) for patients treated with UCART19	Month 3, Month 6, Month 12 during Year 1 following last UCART19 dosing, then every 6 months up to Year 3, then yearly up to Year 15
Time to transplant for patients treated with UCART19	Month 3, Month 6, Month 12 during Year 1 following last UCART19 dosing, then every 6 months up to Year 3, then yearly up to Year 15
Assessment of overall survival	Month 3, Month 6, Month 12 during Year 1 following last UCART19 dosing, then every 6 months up to Year 3, then yearly up to Year 15
Proportion of patients with detectable UCART19 levels in blood	Month 3, Month 6, Month 12 during Year 1 following last UCART19 dosing (If no more UCART19 detectable at the Month 12 visit), then every 6 months up to Year 3, then yearly up to Year 15 or the first evidence of no more detectable UCART19 (if earlier)
Proportion of patients with detectable UCART19 levels in bone marrow	Month 3, Month 6, Month 12 during Year 1 following last UCART19 dosing, then every 6 months up to Year 3, then yearly up to Year 15

Collaborators and Investigators

• Sponsor: Institut de Recherches Internationales Servier
• Collaborators: ADIR, a Servier Group company
• Investigators: Principal Investigator: Reuben Benjamin, MD, PhD, King's College Hospital NHS Trust

Publications and helpful links

Helpful Links

• Find Results on Servier Clinical Trial Data

Study record dates

Study Major Dates

• Study Start (Actual): March 10, 2021
• Primary Completion (Estimated): February 1, 2025
• Study Completion (Estimated): February 1, 2025

Study Registration Dates

• First Submitted: March 14, 2016
• First Submitted That Met QC Criteria: April 6, 2016
• First Posted (Estimated): April 12, 2016

Study Record Updates

• Last Update Posted (Actual): September 13, 2023
• Last Update Submitted That Met QC Criteria: September 12, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Leukemia; Leukemia, Lymphoid
• Other Study ID Numbers: CL1-68587-003; 2016-000297-38 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data.

Access can be requested for all interventional clinical studies:

• used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
• where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope.

In addition, access can be requested for all interventional clinical studies in patients:

• sponsored by Servier
• with a first patient enrolled as of 1 January 2004 onwards
• for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.

• IPD Sharing Time Frame: After Marketing Authorisation in EEA or US if the study is used for the approval.
• IPD Sharing Access Criteria: Researchers should register on Servier Data Portal and fill in the research proposal form. This form in four parts should be fully documented. The Research Proposal Form will not be reviewed until all mandatory fields are completed.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Study Data/Documents

1. Individual Participant Data Set
2. Study Protocol
3. Statistical Analysis Plan
4. Informed Consent Form
5. Clinical Study Report
6. Study-level clinical trial data

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No