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

• ClinicalTrials.gov Identifier: NCT02735083
• Recruitment Status: Enrolling by invitation
• First Posted: April 12, 2016
• Last Update Posted: October 19, 2020
• Sponsor: Institut de Recherches Internationales Servier
• Collaborator: ADIR, a Servier Group company
• Information provided by (Responsible Party): Servier ( Institut de Recherches Internationales Servier )

Tracking Information

• First Submitted Date: March 14, 2016
• First Posted Date: April 12, 2016
• Last Update Posted Date: October 19, 2020
• Actual Study Start Date: November 9, 2016
• Estimated Primary Completion Date: March 2040 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 14, 2020)

Long-term safety of UCART19 with or without alemtuzumab [ Time Frame: Up to 15 Year ]

• 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

Original Primary Outcome Measures (submitted: April 6, 2016)

Long-term safety of UCART19 [ Time Frame: Up to 15 Year ]

- Adverse events assessed as related to UCART19

Current Secondary Outcome Measures (submitted: October 14, 2020)

• Assessment of long-term anti tumor activity of UCART19 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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 bone marrow [ Time Frame: 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 ]

Original Secondary Outcome Measures (submitted: April 6, 2016)

• Assessment of long-term anti leukemic activity of UCART19. [ Time Frame: onth 3, Month 6, Month 12 during Year 1 following UCART19 dosing, then every 6 months up to Year 3, then yearly up to Year 15 if the follow-up period is prolonged ]
• Proportion of patients who underwent allogeneic HSCT (Hematopoietic Stem Cell Transplantation) [ Time Frame: Month 3, Month 6, Month 12 during Year 1 following UCART19 dosing, then every 6 months up to Year 3, then yearly up to Year 15 if the follow-up period is prolonged ]
• -Proportion of patients with detectable UCART19 levels in peripheral blood [ Time Frame: Month 3, Month 6, Month 12 during Year 1 following UCART19 dosing, then every 6 months up to Year 3, then yearly up to Year 15 if the follow-up period is prolonged ]
• Time to transplant [ Time Frame: Month 3, Month 6, Month 12 during Year 1 following UCART19 dosing, then every 6 months up to Year 3, then yearly up to Year 15 if the follow-up period is prolonged ]
• Proportion of patients with detectable RCL (Replication Competent Lentivirus) in peripheral blood [ Time Frame: Month 3, Month 6, Month 12 during Year 1 following UCART19 dosing, then every 6 months up to Year 3, then yearly up to Year 15 if the follow-up period is prolonged ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Study to Evaluate the Long-term Safety of Patients With Advanced Lymphoid Malignancies Who Have Been Previously Administered With UCART19
• Official Title: 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)
• Brief Summary: The purpose of this study is to evaluate the long-term safety and efficacy of UCART19 administration to patients with advanced lymphoid malignancies.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Other
• Condition: Advanced Lymphoid Malignancies

Intervention

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.

Study Arms

Experimental: UCART19 follow-up

Intervention: Biological: UCART19 follow-up

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Enrolling by invitation
• Estimated Enrollment (submitted: July 10, 2019): 300
• Original Estimated Enrollment (submitted: April 6, 2016): 200
• Estimated Study Completion Date: March 2040
• Estimated Primary Completion Date: March 2040 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: France, Japan, Spain, United Kingdom, United States
• Removed Location Countries: Belgium

Administrative Information

• NCT Number: NCT02735083
• Other Study ID Numbers: CL1-68587-003; 2016-000297-38 ( EudraCT Number )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: Yes

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.

• Supporting Materials: Study Protocol
• Supporting Materials: Statistical Analysis Plan (SAP)
• Supporting Materials: Informed Consent Form (ICF)
• Supporting Materials: Clinical Study Report (CSR)
• Time Frame: After Marketing Authorisation in EEA or US if the study is used for the approval.
• 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.
• URL: https://clinicaltrials.servier.com/

• Responsible Party: Servier ( Institut de Recherches Internationales Servier )
• Study 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

• PRS Account: Servier
• Verification Date: October 2020