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

Phase 1

Completed

Brief Summary: 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
UCART19	UCART19	-

Primary Outcome Measures

Name	Time	Method
Dose escalation part: Dose Limiting Toxicities (DLTs) occurence. Dose expansion part: AE throughout the study.	Dose Escalation: Up to day 28 post first UCART19 infusion. Dose Expansion: From inclusion to Month 12

Secondary Outcome Measures

Name	Time	Method
Incidence and Severity of Adverse Events as a Measure of Safety and Tolerability	From inclusion to Month 12	Adverse events assessed according to NCI-CTCAE v5.0 criteria
Duration of remission	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
Overall Survival (OS)	From the date of UCART19 administration to the date of death from any cause, assessed up to Month 12
Objective Remission Rate	At Day 28, Day 84, Month 4, Month 6, Month 9 and Month12	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)
Progression Free Survival (PFS)	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
Time to remission	From the date of UCART19 administration until the date that response criteria are met, assessed up to Month 12

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-Antoine
🇫🇷
PARIS Cedex 12, France
Hôpital Saint-Louis
🇫🇷
Paris, France
Kyushyu University Hospital
🇯🇵
Fukuoka, Japan
King's College Hospital NHS Foundation Trust
🇬🇧
London, United Kingdom
Hokkaido University Hospital
🇯🇵
Sapporo, Japan
The Christie NHS Foundation Trust
🇬🇧
Manchester, United Kingdom