B-cell Acute Lymphoblastic Leukemia
Conditions
Interventions
Biological :CTL019
Sponsors
Novartis Pharma. K.K.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Relapsed or refractory pediatric B-cell ALL 2. Adequate organ function 3. For relapsed patients, documentation of CD19 tumor expression within 3 months of study entry. 4. Bone marrow with >= 5% lymphoblasts by morphologic assessment at screening. 5. Life expectancy > 12 weeks. 6. Karnofsky (age >=16 years) or Lansky (age = 50 at screening 7. Signed written informed consent and assent forms 8. Must meet the institutional criteria to undergo leukapheresis or have an acceptable, store leukapheresis product 9. Must have an apheresis product of non-mobilized cells received and accepted by the manufacturing site. 10. Cohort 1 only: 1. First relapse AND hypodiploid cytogenetics OR 2. First relapse AND t(17;19) with defined TCF3-HLF fusion OR 3. First relapse with any cytogenetics provided the relapse occurred ==0.01% by flow cytometry (local assessment)
Exclusion criteria
Exclusion criteria: 1. Isolated extra-medullary disease relapse 2. Patients with concomitant genetic syndrome: such as patients with Fanconi anemia, Kostmann syndrome, Shwachman syndrome or any other known bone marrow failure syndrome. Patients with Down Syndrome will not be excluded. 3. Patients with Burkitt's lymphoma/leukemia (i.e. patients with mature B-cell ALL, leukemia with B-cell [sIg positive and kappa or lambda restricted positivity] ALL, with FAB L3 morphology and /or a MYC translocation) 4. Prior malignancy, except carcinoma in situ of the skin or cervix treated with curative intent and with no evidence of active disease 5. Treatment with any prior gene therapy product 6. Has had treatment with any prior anti-CD19/anti-CD3 therapy, or any other anti-CD19 therapy 7. Active or latent hepatitis B or active hepatitis C (test within 8 weeks of screening), or any uncontrolled infection at screening 8. Human Immunodeficiency Virus (HIV) positive test within 8 weeks of screening 9. Presence of grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). 10. Active CNS involvement by malignancy, defined by CNS-3 per NCCN guidelines. 11. Patient has an investigational medicinal product within the last 30 days prior to screening. 12. Pregnant or nursing (lactating) women. 13. Women of child-bearing potential, defined as physiologically capable of becoming pregnant, unless they agree to use highly effective methods of contraception for at least 12 months after the CTL019 infusion and after CAR T-cells are no longer present by qPCR on two consecutive tests 14. Sexually active males must use a condom during intercourse at least 12 months after the CTL019 infusion after CAR T-cells are no longer present by qPCR on two consecutive tests
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percentage of Participants With Overall Remission Rate (ORR) as Determined by IRC Assessment. [ Time Frame: during the 3 months after tisagenlecleucel administration ] Evaluating the efficacy of tisagenlecleucel therapy from all manufacturing facilities as measured by overall remission rate (ORR) during the 3 months after tisagenlecleucel administration. ORR included complete response (CR) and CR with incomplete blood count recovery (CRi) as determined by an Independent Review Committee ( IRC) assessment. | — |
Countries
Australia, Austria, Belgium, Canada, France, Germany, Italy, Norway, Spain, United states
Contacts
Public ContactKyosuke Yamauchi
Novartis Pharma. K.K.
Outcome results
None listed