Study on the Effectiveness and Safety of Tisagenlecleucel for High-Risk B-Cell Acute Lymphoblastic Leukemia in Young Patients
Sponsor: Novartis Pharmaceuticals
ClinicalTrials ID:NCT03876769
This clinical trial is testing the safety and effectiveness of a treatment called tisagenlecleucel in children and young adults with high-risk B-cell Acute Lymphoblastic Leukemia (B-ALL) who have not fully responded to initial treatments.
Patient Parameters
| Parameter | Options |
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Program Overview
This study aims to evaluate how well the treatment tisagenlecleucel works and how safe it is for children and young adults with high-risk B-cell Acute Lymphoblastic Leukemia (B-ALL) who still have minimal residual disease (MRD) after their first round of treatment. The study will follow participants through several phases: screening, pre-treatment, treatment and follow-up, and survival. After receiving tisagenlecleucel, participants will have frequent health checks, especially in the first month, then at Day 29, every 3 months for the first year, every 6 months for the second year, and then yearly until the study ends. The study is expected to last about 8 years from when the first participant starts treatment. Long-term safety follow-up will continue under a separate plan according to health guidelines.
Eligibility Criteria
Inclusion Criteria
- Diagnosed with B-cell Acute Lymphoblastic Leukemia that shows the CD19 marker.
- High-risk B-ALL patients who have received initial treatment but still have minimal residual disease (MRD) of 0.01% or more.
- Aged between 1 and 25 years.
- Have a performance status score of 60% or higher, indicating they can carry out daily activities with some limitations.
- Have adequate organ function, including:
- Proper kidney function for their age and gender.
- Liver enzymes (ALT and AST) within 5 times the normal limit for their age.
- Total bilirubin less than 2 mg/dL (or less than 4 mg/dL for those with Gilbert's Syndrome).
- Good lung function with no or mild shortness of breath and oxygen levels above 90% on room air.
- Good heart function with specific measurements confirmed by heart tests.
- Allowed to have had up to 3 blocks of standard chemotherapy for first-line B-ALL treatment.
Exclusion Criteria
- Have more than 25% leukemia cells in the bone marrow after initial treatment.
- Have more than 5% leukemia cells in the bone marrow or ongoing disease outside the bone marrow after initial consolidation treatment.
- Have Philadelphia chromosome positive ALL.
- Have fewer than 44 chromosomes or other signs of a specific genetic condition called hypodiploidy.
- Have had previous treatment with tyrosine kinase inhibitors.
- Have genetic syndromes that affect bone marrow function, except for Down syndrome.
- Have Burkitt's lymphoma/leukemia or similar conditions.
- Have received any previous anti-CD19 therapy or gene-engineered T cell therapy.
Locations
| FACILITY | ZIP | CITY | STATE |
|---|---|---|---|
| Children s Hospital of Alabama | 35233 | Birmingham | Alabama |
| Phoenix Childrens Hospital | 85016 | Phoenix | Arizona |
| City of Hope National Medical | 91010 | Duarte | California |
| Childrens Hospital Los Angeles | 90027 | Los Angeles | California |
| Mattel Childrens Hospital UCLA | 90095 | Los Angeles | California |
| Childrens Hospital of Orange County | 92868-3874 | Orange | California |
| Rady Children s Hospital | 92123 | San Diego | California |
| UCSF Medical Center | 94143 | San Francisco | California |
| Stanford University Medical Center | 94304 | Stanford | California |
| Childrens Hospital Colorado | 80045 | Aurora | Colorado |
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