Study of UCART19 in Pediatric Patients With Relapsed/Refractory B Acute Lymphoblastic Leukemia (PALL)
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This study aims at evaluating the safety and ability of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL) ahead of planned allogeneic hematopoietic stem cell transplantation (allo-HSCT).
A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B-cell Acute Lymphoblastic Leukaemia
Actual Study Start Date :
June 3, 2016
Estimated Primary Completion Date :
Estimated Study Completion Date :
Resource links provided by the National Library of Medicine
Incidence and Severity of Adverse Events [ Time Frame: From inclusion to Day 84 post UCART19 infusion or until the initiation of allo-HSCT conditioning regimen ]
Adverse events assessed according to NCI-CTCAE v4.03 criteria
Incidence and Severity of Adverse Events [ Time Frame: From Day 85 or the initiation of allo-HSCT conditioning regimen to Month 12 post allo-HSCT ]
Secondary Outcome Measures :
Molecular Remission Rate [ Time Frame: At Day 28 post UCART19 infusion ]
Proportion of patients in whom a molecular Complete Remission (CR) or a Complete Remission with incomplete blood recovery (CRi) is observed (i.e. a CR or CRi combined to a Minimal residual disease <10-4).
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Ages Eligible for Study:
6 Months to 17 Years (Child)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Patient with relapsed or refractory CD19-positive B-acute lymphoblastic leukaemia (B-ALL)
who have exhausted available treatment options
eligible for allo-HSCT with suitable donor available
Estimated life expectancy ≥ 12 weeks
Considered medically fit for allo-HSCT
Eastern Cooperative Oncology Group (ECOG) performance status < 2
CD19-negative B-cell leukemia
Uncontrolled Central Nervous System (CNS) leukemia
Active acute or chronic Graft-versus-Host Disease (GvHD) requiring therapy
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:
Researchers can ask for a study protocol, patient-level and/or study-level clinical trial data including clinical study reports (CSRs).
They can ask all interventional clinical studies:
submitted for new medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US).
Where Servier or an affiliate are the Marketing Authorization Holders (MAH). The date of the first Marketing Authorization of the new medicine (or the new indication) in one of the EEA Member States will be considered within this scope.
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
After Marketing Authorisation in EEA or US if the study is used for the approval.
Researchers should register on Servier Data Portal and fill in the research proposal form. This form in four parts should be fully documented. The Research Proposal Form will not be reviewed until all mandatory fields are completed.
Studies a U.S. FDA-regulated Drug Product:
Studies a U.S. FDA-regulated Device Product:
Product Manufactured in and Exported from the U.S.:
Additional relevant MeSH terms:
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Neoplasms by Histologic Type