Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients (NCT03876769) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Study of Efficacy and Safety of Tisagenlecleucel in HR B-ALL EOC MRD Positive Patients
United States, Belgium, Canada121 participantsStarted 2019-06-24
Plain-language summary
This is a single arm, open-label, multi-center, phase II study to determine the efficacy and safety of tisagenlecleucel in de novo HR pediatric and young adult B-ALL patients who received first-line treatment and are EOC MRD positive. The study will have the following sequential phases: screening, pre-treatment, treatment \& follow-up, and survival. After tisagenlecleucel infusion, patient will have assessments performed more frequently in the first month and then at Day 29, then every 3 months for the first year, every 6 months for the second year, then yearly until the end of the study. Efficacy and safety will be assessed at study visits and as clinically indicated throughout the study. The study is expected to end in approximately 8 years after first patient first treatment (FPFT). A post-study long term follow-up safety will continue under a separate protocol per health authority guidelines.
Who can participate
Age range
1 Year – 25 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
. De novo NCI HR B-ALL who received first-line treatment and are MRD ≥ 0.01% at EOC. EOC bone marrow MRD will be collected prior to screening and will be assessed by multi-parameter flow cytometry using central laboratory analysis.
. Age 1 to 25 years at the time of screening
. Lansky (age \< 16 years) or Karnofsky (age ≥ 16 years) performance status ≥ 60%
. Adequate organ function during the screening period:
. Prior induction and consolidation chemotherapy allowed: 1st line subjects: ≤ 3 blocks of standard chemotherapy for first-line B-ALL, defined as 4-drug induction, Berlin-Frankfurt-Münster (BFM) consolidation or Phase 1b, and interim maintenance with high-dose methotrexate.
Exclusion criteria
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial is testing tisagenlecleucel as a CAR-T cell therapy for B-cell ALL patients who still have measurable residual disease after end-of-consolidation treatment — does my situation match that profile, and is my MRD status the kind this trial was designed for?
2Since this is a Phase 2 trial that is no longer actively enrolling, what does that mean for my chances of participating, and are there any related or successor trials studying tisagenlecleucel that might still be open to me?
3The trial is measuring Disease Free Survival both with and without accounting for stem cell transplant — can you explain how that matters for me, and would a stem cell transplant likely still be part of my treatment plan whether or not I pursued a CAR-T approach like this?
4CAR-T therapies like tisagenlecleucel can carry serious risks such as cytokine release syndrome — given that this is still a Phase 2 study where the full safety profile is still being characterized, how do those risks compare to the standard treatment options available to me right now?
5Since the trial tracks Overall Survival as a key outcome, what do you know so far about how patients in this study have fared, and how does that compare to what I might expect from a more conventional treatment path?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Disease Free Survival (DFS) rate without censoring for new anticancer therapy, including Stem Cell Transplantation (SCT) while in remission
Timeframe: 5 years after tisagenlecleucel infusion
. M3 marrow at the completion of 1st line induction therapy
. M2 or M3 marrow or persistent extramedullary disease at the completion of first-line consolidation therapy or evidence of disease progression in the peripheral blood or new extramedullary disease prior to enrollment. Patients with previous CNS disease are eligible if there is no active CNS involvement of leukemia at the time of screening.
. Philadelphia chromosome positive ALL
. Hypodiploid: less than 44 chromosomes and/or DNA index \< 0.81, or other clear evidence of a hypodiploid clone
. Prior tyrosine kinase inhibitor therapy
. Subjects with concomitant genetic syndromes associated with bone marrow failure states: such as subjects with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome. Subjects with Down syndrome will not be excluded.
. Subjects with Burkitt's lymphoma/leukemia (i.e. subjects with mature B-ALL, leukemia with B-cell \[sIg positive and kappa or lambda restricted positivity\] ALL, with FAB L3 morphology and /or a MYC translocation)
. Has had treatment with any prior anti-CD19 therapy 9. Treatment with any prior gene or engineered T cell therapy