Study Stopped
Terminated due to slow enrollment
Study of Efficacy and Safety of Reinfusion of Tisagenlecleucel in Pediatric and Young Adult Patients With Acute Lymphoblastic Leukemia (ALL)
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
1 other identifier
interventional
5
1 country
4
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Oct 2020
Shorter than P25 for phase_2
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 9, 2020
CompletedFirst Posted
Study publicly available on registry
January 13, 2020
CompletedStudy Start
First participant enrolled
October 19, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 19, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
October 19, 2021
CompletedResults Posted
Study results publicly available
July 15, 2022
CompletedJanuary 30, 2023
January 1, 2023
1 year
January 9, 2020
April 14, 2022
January 27, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage of Patients Who Establish B Cell Aplasia Within 9 Months of Reinfusion
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 Outcomes (3)
Complete Response (CR) or Complete Response With Incomplete Blood Count Recovery (CRi) by Day
Post-reinfusion up to 9 months
Participants With an Event
Reinfusion up to 9 months
Overall Survival (OS)
Reinfusion up to 9 months
Study Arms (1)
Tisagenlecleucel
EXPERIMENTALTisagenlecleucel 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.
Interventions
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.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (4)
Childrens Hospital Los Angeles Divisionof Hematology/Oncology
Los Angeles, California, 90027, United States
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611, United States
Children s Mercy Hospital
Kansas City, Missouri, 64108, United States
UT Southwestern Medical Center
Dallas, Texas, 75235, United States
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Novartis Pharmaceuticals
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 9, 2020
First Posted
January 13, 2020
Study Start
October 19, 2020
Primary Completion
October 19, 2021
Study Completion
October 19, 2021
Last Updated
January 30, 2023
Results First Posted
July 15, 2022
Record last verified: 2023-01
Data Sharing
- IPD Sharing
- Will share
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.