Clinical trial NCT02808442: Study of UCART19 in Pediatric Patients With Relapsed/Refractory B Acute Lymphoblastic Leukemia
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Refractory B-cell Acute Lymphoblastic Leukemia
Relapsed B-cell Acute Lymphoblastic Leukemia
Lead sponsor: Servier
Sponsor class: INDUSTRY
Collaborators: name: ADIR, a Servier Group company
First posted: 2016-06-21
Enrollment: 13
Sex: ALL
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study aims to evaluate 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).


Eligibility criteria: 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 within 4 weeks before UCART19 infusion
* Patients with autoimmune disease requiring systemic immunosuppression therapy that cannot be stopped
* History of CRS grade 4 related to previous CAR T cell therapy
* Contraindication to Alemtuzumab administration

Primary outcomes:
measure: Incidence and Severity of Adverse Events
timeFrame: From inclusion to Month 12
Secondary outcomes:
measure: Molecular Remission Rate
timeFrame: At Day 28 after the first UCART19 infusion
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Paris
country: France
facility: Hôpital Robert-Debré
city: Barcelona
country: Spain
facility: Hospital San Juan De Dios
city: London
country: United Kingdom
facility: UCL Great Ormond Hospital
Study officials:
References:
citation: Benjamin R, Graham C, Yallop D, Jozwik A, Mirci-Danicar OC, Lucchini G, Pinner D, Jain N, Kantarjian H, Boissel N, Maus MV, Frigault MJ, Baruchel A, Mohty M, Gianella-Borradori A, Binlich F, Balandraud S, Vitry F, Thomas E, Philippe A, Fouliard S, Dupouy S, Marchiq I, Almena-Carrasco M, Ferry N, Arnould S, Konto C, Veys P, Qasim W; UCART19 Group. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies. Lancet. 2020 Dec 12;396(10266):1885-1894. doi: 10.1016/S0140-6736(20)32334-5.