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

Phase 1
Completed
Conditions
Adult relapsed /refractory B-A
Registration Number
JPRN-jRCT1080224971
Lead Sponsor
ihon Servier
Brief Summary

This international, multicentre, open-label, dose-escalating, first in human CALM study was conducted in 25 adults with relapsed or refractory CD19 positive B-ALL to assess the safety and tolerability of ascending doses of UCART19, given as a single infusion (with the possibility of re-dosing). MTD was defined as the UCART19 DL3 (3 x 106 cells/kg) and the recommended dose was defined as the UCART19 DL2 (1 x 106 cells/kg).

Detailed Description

Not available

Recruitment & Eligibility

Status
completed
Sex
All
Target Recruitment
25
Inclusion Criteria

Male or female participant

- Age from 18 years

- Patient with relapsed or refractory CD19 positive B acute lymphoblastic leukaemia (B ALL) who have exhausted alternative treatment options

- Estimated life expectancy more than 12 weeks (according to investigator's judgement)

- Eastern Cooperative Oncology Group (ECOG) performance status under 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

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
efficacy<br>safety<br>Dose escalation part: Dose Limiting Toxicities (DLTs) occurence. Dose expansion part: AE throughout the study.
Secondary Outcome Measures
NameTimeMethod
efficacy<br>exploratory<br>pharmacokinetics<br>safety<br>pharmacodynamics<br>pharmacogenomics<br>efficacy, pharmacokinetics, etc
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