B-cell Acute Lymphoblastic Leukemia
Conditions
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.
Interventions
BIOLOGICALUCART19
Sponsors
ADIR, a Servier Group company
Institut de Recherches Internationales Servier
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to 69 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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) |
| 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 | — |
| 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 |
| 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 | — |
| Overall Survival (OS) | From the date of UCART19 administration to the date of death from any cause, 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 | — |
Countries
France, Japan, United Kingdom, United States
Outcome results
None listed