Study of Efficacy and Safety of Reinfusion of Tisagenlecleucel in Pediatric and Young Adult Patients With Acute Lymphoblastic Leukemia (ALL)

January 27, 2023 updated by: Novartis Pharmaceuticals

A Phase II, Open Label, Multi-center Trial to Determine the Efficacy and Safety of Tisagenlecleucel Re-infusion in Pediatric and Adolescent Young Adult (AYA) Patients With Acute Lymphoblastic Leukemia Experiencing Loss of B Cell Aplasia

This was a multi-center Phase II study investigating the efficacy and safety of reinfusion of tisagenlecleucel in pediatric and young adult patients with acute lymphoblastic leukemia (ALL) who were treated with tisagenlecleucel and experience B cell recovery.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

This trial was a phase II, open label, multi-center trial to determine the efficacy and safety of tisagenlecleucel re-infusion in pediatric and adolescent young adult (AYA) patients with acute lymphoblastic leukemia (ALL) experiencing loss of B cell aplasia. Loss of B-cell aplasia is defined as: peripheral blood (PB) absolute B lymphocyte count ≥ 50/µL, OR PB B lymphocyte ≥ 10% of the total lymphocytes. B-cell aplasia is defined as PB absolute B lymphocyte count <50/µL.

The study had the following phases for all patients: Screening, Treatment and Follow-up. The total duration of the study was about 12 months. After tisagenlecleucel re-infusion, efficacy was assessed at months 1, 3, 6, and End of Study at which time blood samples were obtained.

The study stopped early due to slow enrollment into the trial. The rate of enrollment made the trial no longer feasible to continue.

The patients were able to voluntarily withdraw from the study for any reason, at any time. Patients who received commercial tisagenlecleucel had to be followed for up to 15 years post-infusion. Patients could have been followed under the Center for International Blood and Marrow Transplant Research (CIBMTR) cellular therapy registry if consented for participation. For patients who do not provide consent for participation in the Center for International Blood and Marrow Transplant Research (CIBMTR) registry, adverse events were to be reported for 15 years or until the patient enrolls in the registry.

Study Type

Interventional

Enrollment (Actual)

5

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Childrens Hospital Los Angeles Divisionof Hematology/Oncology
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Ann and Robert H Lurie Childrens Hospital of Chicago
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Children s Mercy Hospital
    • Texas
      • Dallas, Texas, United States, 75235
        • UT Southwestern Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 25 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Signed informed consent must be obtained prior to participation in the study
  • Must have an additional dose of unexpired, commercial tisagenlecleucel available and prescribed by a physician in the course of medical practice
  • Age up to and including 25 years
  • Patients must have CD-19+ Leukemia
  • Patients who were previously treated with tisagenlecleucel and present with evidence of B-cell recovery as defined by: Peripheral blood (PB) absolute B lymphocyte count ≥ 50/µL, OR PB B lymphocyte ≥ 10% of the total lymphocytes

Exclusion Criteria:

  • Prior gene therapy other than tisagenlecleucel
  • Prior adoptive T cell therapy other than tisagenlecleucel
  • Active CNS involvement by malignancy
  • Active or latent hepatitis B or active hepatitis C, or any uncontrolled infection at screening
  • HIV positive test within 8 weeks of screening

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Tisagenlecleucel

Tisagenlecleucel Cell Dispersion for Infusion given once during the study.

The approved dose range for tisagenlecleucel is: 0.2 to 5.0×106 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×108 CAR-positive viable T cells for patients > 50 kg body weight.
Biological: Tisagenlecleucel

Tisagenlecleucel Cell Dispersion for Infusion given once during the study.

The approved dose range for tisagenlecleucel is: 0.2 to 5.0×106 CAR positive viable T cells / kg for patients' ≤ 50 kg body weight or 0.1 to 2.5×108 CAR-positive viable T cells for patients > 50 kg body weight.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Patients Who Establish B Cell Aplasia Within 9 Months of Reinfusion
Time Frame: Post-reinfusion up to 9 months (Day 1 is excluded)
Percentage of patients who establish B-cell aplasia at any visit following re-infusion with tisagenlecleucel. B-cell aplasia is defined as peripheral blood (PB) absolute B lymphocyte count <50/μL. Planned timeframe was 12 months but actual timeframe was approximately 9 months due to early termination of the trial. Day 1 is post lymphodepleting chemotherapy and pre-reinfusion of tisagenlecleucel.
Post-reinfusion up to 9 months (Day 1 is excluded)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Time Frame: Post-reinfusion up to 9 months
Overall remission rate (ORR) was defined as the percentage of participants with a best overall disease response of complete remission (CR) or CR with incomplete blood count recovery (CRi). However, the rate was not calculated due to low enrollment. Participants' best responses have been listed by day and participants may be counted more than once.
Post-reinfusion up to 9 months
Participants With an Event
Time Frame: Reinfusion up to 9 months
An event was defined as one of the following: death from any cause after remission, relapse, treatment failure (defined as no response in the study or discontinuation from the study for death, adverse event, lack of efficacy or progressive disease or new cancer therapy).
Reinfusion up to 9 months
Overall Survival (OS)
Time Frame: Reinfusion up to 9 months
Deaths due to any reason.
Reinfusion up to 9 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 19, 2020

Primary Completion (Actual)

October 19, 2021

Study Completion (Actual)

October 19, 2021

Study Registration Dates

First Submitted

January 9, 2020

First Submitted That Met QC Criteria

January 9, 2020

First Posted (Actual)

January 13, 2020

Study Record Updates

Last Update Posted (Estimate)

January 30, 2023

Last Update Submitted That Met QC Criteria

January 27, 2023

Last Verified

January 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CCTL019BUS03

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Yes

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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