Study of UCART19 in Pediatric Patients With Relapsed/Refractory B Acute Lymphoblastic Leukemia (PALL)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02808442
Recruitment Status : Active, not recruiting
First Posted : June 21, 2016
Last Update Posted : November 28, 2018
Information provided by (Responsible Party):
Servier ( Institut de Recherches Internationales Servier )

Brief Summary:
This study aims at evaluating the safety and feasibility of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL).

Condition or disease Intervention/treatment Phase
Refractory B-cell Acute Lymphoblastic Leukemia Relapsed B-cell Acute Lymphoblastic Leukemia Biological: UCART19 Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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 : April 2020
Estimated Study Completion Date : April 2020

Arm Intervention/treatment
Experimental: UCART19 Biological: UCART19
Other Name: S68587

Primary Outcome Measures :
  1. Incidence and Severity of Adverse Events [ Time Frame: From inclusion to Month 12 ]
    Adverse events assessed according to NCI-CTCAE v4.03 criteria

Secondary Outcome Measures :
  1. Molecular Remission Rate [ Time Frame: At Day 28 after the first 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).

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patient with relapsed or refractory CD19-positive B-acute lymphoblastic leukaemia (B-ALL) who have exhausted alternative treatment options.
  • Estimated life expectancy ≥ 12 weeks
  • Lansky (age < 16 years at the time of assent/consent) or Karnofsky (age ≥ 16 years at time of assent/consent) performance status ≥ 50

Exclusion Criteria:

  • Burkitt leukemia
  • CD19-negative B-cell leukemia
  • Active Central Nervous System (CNS) leukemia
  • Active acute or chronic Graft-versus-Host Disease (GvHD) requiring systemic use therapy

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02808442

United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Het Kinderziekenhuis Prinses Elisabeth - UZG
Gent, Belgium, 9000
Hôpital Robert-Debré
Paris, France, 75019
United Kingdom
UCL Great Ormond Hospital
London, United Kingdom
Sponsors and Collaborators
Institut de Recherches Internationales Servier

Additional Information:
Study Data/Documents: Individual Participant Data Set  This link exits the site

Responsible Party: Institut de Recherches Internationales Servier Identifier: NCT02808442     History of Changes
Other Study ID Numbers: UCART19_02 (CL1-68587-001)
2015-004293-15 ( EudraCT Number )
First Posted: June 21, 2016    Key Record Dates
Last Update Posted: November 28, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

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.

Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Time Frame: After Marketing Authorisation in EEA or US if the study is used for the approval.
Access Criteria: 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: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Additional relevant MeSH terms:
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Burkitt Lymphoma
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Epstein-Barr Virus Infections
Herpesviridae Infections
DNA Virus Infections
Virus Diseases
Tumor Virus Infections
Lymphoma, B-Cell
Lymphoma, Non-Hodgkin