A study to evaluate the long-term safety of patients with advanced lymphoid malignancies who have been previously administered with UCART19

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Institut De Recherches Internationales Servier IRIS

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Decision date (initial)

2026-06-09

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

ADIR

External identifiers

EU CT number

2026-526212-37-00

ClinicalTrials.gov

NCT02735083

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the long-term safety of patients with advanced lymphoid leukemia who have been previously administered with UCART19.

Secondary objectives 6

1. To assess the long-term anti leukemic activity (progression-free survival, disease specific survival)
2. To assess the proportion of patients who underwent allogeneic HSCT
3. To assess the time to transplant
4. To assess overall survival
5. To monitor the long-term persistence of UCART19 in blood
6. To monitor the long-term persistence of UCART19 in bone marrow (only in bone marrow samples performed as part of routine care and at the Investigator’s discretion).

Conditions and MedDRA coding

Advanced lymphoid leukemia

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Medicines And Healthcare Products Regulatory Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Written informed consent obtained prior any study-specific procedure (patient or parent(s) or legal representative).
2. Patient dosed with UCART19 who completed or discontinued early from a sponsored or from any investigator-initiated study that tested UCART19, or patients who were administered UCART19 under a special access scheme (compassionate use).
3. Female patients of childbearing potential and male patients with partners of childbearing potential must continue to use an effective method of birth control as well as their partners for a 12-month duration after the last UCART19 administration.

Exclusion criteria 1

1. No exclusion criteria for this study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 4

1. Number, duration, outcome of all adverse events (AE) within 12 months post last UCART19 infusion.
2. Number, duration, outcome of adverse events of special interest (AESI) up to the end of the study.
3. Proportion of patients with adverse events leading to death up to the end of the study.
4. For paediatric patients: assesment of the potential impact on growth curve and puberty.

Secondary endpoints 7

1. Proportion of patients who relapse or progress.
2. Progression-free survival (PFS), disease specific survival (DSS).
3. Duration of remission until the date of progression or death due to any cause, whichever occurs first.
4. Overall survival (OS)
5. Proportion of patients who underwent allogeneic HSCT if not transplanted within the parent study.
6. Time to transplant.
7. Measurement of CD19CAR transgene levels by qPCR (and optionally by flow cytometry) in blood, and in bone marrow (if a BM aspirate is collected by the centre as part of the routine care of the patient’s disease).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Institut De Recherches Internationales Servier IRIS

Sponsor organisation

Institut De Recherches Internationales Servier IRIS

Address

22 Route 128

City

Gif Sur Yvette

Postcode

91190

Country

France

Scientific contact point

Organisation

Institut De Recherches Internationales Servier IRIS

Contact name

Clinical Studies Department

Public contact point

Organisation

Institut De Recherches Internationales Servier IRIS

Contact name

Clinical Studies Department

Third parties 5

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 13 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications