Dose Escalation Study of UCART19 in Adult Patients With Relapsed / Refractory B-cell Acute Lymphoblastic Leukaemia

Institut de Recherches Internationales Servier25 enrolled

Overview

The study is in two parts: a dose escalation then a safety dose expansion. The purpose of the dose escalation part is to evaluate the safety and tolerability of ascending doses of UCART19 (dose-escalation part) given as a single infusion in patients with relapsed / refractory (R/R) B-cell acute lymphoblastic leukaemia (B-ALL), to determine the maximum tolerated dose (MTD), the recommended dose and the lymphodepletion regimen. The purpose of the safety dose expansion is to assess the safety and tolerability of the RD for UCART19.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

B-cell Acute Lymphoblastic Leukemia

Interventions

UCART19

Eligibility

Sex: ALLMin age: 16 YearsMax age: 69 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Male or female participant * Age ≥ 16 years * Patient with relapsed or refractory CD19 positive B-acute lymphoblastic leukaemia (B-ALL) who have exhausted alternative treatment options * Estimated life expectancy ≥ 12 weeks (according to investigator's judgement) * Eastern Cooperative Oncology Group (ECOG) performance status \< 2 Exclusion Criteria: * Previous treatment with gene or gene-modified cell therapy medicine products or adoptive T cell therapy * Use of previous anti-leukemic therapy (including approved therapies and other investigational products) within 5 half-lives prior to UCART19 administration * CD19 negative B-cell leukaemia * Burkitt cell or mixed lineage acute leukaemia

Locations (9)

Massachusetts General Hospital

Boston, Massachusetts, United States

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

University of Texas MD Anderson Cancer Center

Houston, Texas, United States

Hôpital Saint-Louis

Paris, France

Hôpital Saint-Antoine

Paris, France

Kyushyu University Hospital

Fukuoka, Japan

Hokkaido University Hospital

Sapporo, Japan

King's College Hospital NHS Foundation Trust

London, United Kingdom

The Christie NHS Foundation Trust

Manchester, United Kingdom

Outcomes

Primary Outcomes

Dose escalation part: Dose Limiting Toxicities (DLTs) occurence. Dose expansion part: AE throughout the study.

Time frame: Dose Escalation: Up to day 28 post first UCART19 infusion. Dose Expansion: From inclusion to Month 12

Secondary Outcomes

Incidence and Severity of Adverse Events as a Measure of Safety and Tolerability

Adverse events assessed according to NCI-CTCAE v5.0 criteria

Time frame: From inclusion to Month 12

Objective Remission Rate

Proportion of patients in whom a response among molecular complete remission (mCR), morphologic complete remission (CR) and complete remission with incomplete blood count recovery (CRi)

Time frame: At Day 28, Day 84, Month 4, Month 6, Month 9 and Month12

Duration of remission

Time frame: From the time that response criteria are first met until the date of progression or death (whatever the reason of death), whichever occurs first, assessed up to Month 12

Time to remission

Time frame: From the date of UCART19 administration until the date that response criteria are met, assessed up to Month 12

Progression Free Survival (PFS)

Time frame: From the date of UCART19 administration until the date of progression or the date of death (whatever the reason of death), whichever occur first, assessed up to Month 12

Overall Survival (OS)

Time frame: From the date of UCART19 administration to the date of death from any cause, assessed up to Month 12