Refractory B-cell Acute Lymphoblastic LeukemiaPhase 1PALLNCT02808442
Study of UCART19 in Pediatric Patients With Relapsed/Refractory B Acute Lymphoblastic Leukemia
A Phase 1 study testing UCART19 in people with Refractory B-cell Acute Lymphoblastic Leukemia and Relapsed B-cell Acute Lymphoblastic Leukemia, run by Institut de Recherches Internationales Servier at 6 sites in 4 countries.
Trial summary
This study aims to evaluate the safety and feasibility of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL). Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- Refractory B-cell Acute Lymphoblastic Leukemia, Relapsed B-cell Acute Lymphoblastic Leukemia
- What is being tested?
- UCART19
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 6 sitesin 4 countries
- Study dates
- 3 Jun 2016to 4 Nov 2020
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patient with relapsed or refractory CD19-positive B-acute lymphoblastic leukaemia (B-ALL) who have exhausted alternative treatment options.
- Estimated life expectancy ≥ 12 weeks
- Lansky (age < 16 years at the time of assent/consent) or Karnofsky (age ≥ 16 years at time of assent/consent) performance status ≥ 50
Who cannot join
- Burkitt leukemia
- CD19-negative B-cell leukemia
- Active Central Nervous System (CNS) leukemia
- Active acute or chronic Graft-versus-Host Disease (GvHD) requiring systemic use therapy within 4 weeks before UCART19 infusion
- Patients with autoimmune disease requiring systemic immunosuppression therapy that cannot be stopped
- History of CRS grade 4 related to previous CAR T cell therapy
- Contraindication to Alemtuzumab administration
Trial locations and contacts
6 sites in 4 countries.
6 sites in 4 countries
United States3 sites
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Children's Hospital Los Angeles
Los Angeles, California, 90027
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B-cell Acute Lymphoblastic Leukaemia
This study aims to evaluate the safety and feasibility of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL).
- Conditions
- Refractory B-cell Acute Lymphoblastic Leukemia, Relapsed B-cell Acute Lymphoblastic Leukemia
- Being tested
- UCART19
- Medical subject headings
- Burkitt Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UCART19Biological
Also called S68587
| Study group | Role | What they receive |
|---|---|---|
| UCART19 | Experimental | Not described Receives: Biological: UCART19 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and Severity of Adverse Events
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Measured: From inclusion to Month 12
Secondary outcomes (1)
Molecular Remission Rate
Proportion of patients in whom a molecular Complete Remission (CR) or a Complete Remission with incomplete blood recovery (CRi) is observed (i.e. a CR or CRi combined to a Minimal residual disease <10-4).
Measured: At Day 28 after the first UCART19 infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2016 (estimated)
- Study start3 Jun 2016
- Primary completion17 Sep 2020
- Study completion4 Nov 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Refractory B-cell Acute Lymphoblastic LeukemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Institut de Recherches Internationales Servier
- Sponsor type
- University, hospital or other
- Collaborators
- ADIR, a Servier Group company
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02808442
- Sponsor's protocol ID
- UCART19_02 (CL1-68587-001)
- Other IDs
- 2015-004293-15, 2015
- Acronym
- PALL
- Status last verified
- Sep 2021
- First submitted
- 16 Jun 2016
- Last updated
- 8 Oct 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data. Access can be requested for all interventional clinical studies: * used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US). * where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope. In addition, access can be requested for all interventional clinical studies in patients: * sponsored by Servier * with a first patient enrolled as of 1 January 2004 onwards * for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.
Publications
Benjamin R, Graham C, Yallop D, Jozwik A, Mirci-Danicar OC, Lucchini G, Pinner D, Jain N, Kantarjian H, Boissel N, Maus MV, Frigault MJ, Baruchel A, Mohty M, Gianella-Borradori A, Binlich F, Balandraud S, Vitry F, Thomas E, Philippe A, Fouliard S, Dupouy S, Marchiq I, Almena-Carrasco M, Ferry N, Arnould S, Konto C, Veys P, Qasim W; UCART19 Group. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies. Lancet. 2020 Dec 12;396(10266):1885-1894. doi: 10.1016/S0140-6736(20)32334-5.
PubMed 33308471Li YR, Zhu Y, Fang Y, Lyu Z, Yang L. Emerging trends in clinical allogeneic CAR cell therapy. Med. 2025 Aug 8;6(8):100677. doi: 10.1016/j.medj.2025.100677. Epub 2025 May 13.
PubMed 40367950
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Open Label, Non-comparative Study to Evaluate the Safety and the Ability of UCART19 to Induce Molecular Remission in Paediatric Patients With Relapsed/Refractory B-cell Acute Lymphoblastic Leukaemia
- ClinicalTrials.gov ID
- NCT02808442
- Acronym / sponsor ID
- PALL / UCART19_02 (CL1-68587-001)
- Conditions
- Refractory B-cell Acute Lymphoblastic Leukemia, Relapsed B-cell Acute Lymphoblastic Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Institut de Recherches Internationales ServierUniversity, hospital or other
- Collaborators
- ADIR, a Servier Group company
- Study start
- 3 Jun 2016 (actual)
- Primary completion
- 17 Sep 2020 (actual)
- Study completion
- 4 Nov 2020 (actual)
- First posted
- 21 Jun 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study aims to evaluate the safety and feasibility of UCART19 to induce molecular remission in pediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UCART19 | Experimental | Biological: UCART19 |
Who may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Patient with relapsed or refractory CD19-positive B-acute lymphoblastic leukaemia (B-ALL) who have exhausted alternative treatment options.
- Estimated life expectancy ≥ 12 weeks
- Lansky (age < 16 years at the time of assent/consent) or Karnofsky (age ≥ 16 years at time of assent/consent) performance status ≥ 50
Key exclusion criteria (7 listed)
- Burkitt leukemia
- CD19-negative B-cell leukemia
- Active Central Nervous System (CNS) leukemia
- Active acute or chronic Graft-versus-Host Disease (GvHD) requiring systemic use therapy within 4 weeks before UCART19 infusion
- Patients with autoimmune disease requiring systemic immunosuppression therapy that cannot be stopped
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UCART19Also: S68587 | Biological | UCART19 |
Primary outcome
Incidence and Severity of Adverse Events
Time frame: From inclusion to Month 12
Adverse events assessed according to NCI-CTCAE v5.0 criteria
1 secondary outcome
Molecular Remission Rate
Time frame: At Day 28 after the first UCART19 infusion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2021.
- Results publication: PubMed 33308471Benjamin R, Graham C, Yallop D, Jozwik A, Mirci-Danicar OC, Lucchini G, Pinner D, Jain N, Kantarjian H, Boissel N, Maus MV, Frigault MJ, Baruchel A, Mohty M, Gianella-Borradori A, Binlich F, Balandraud S, Vitry F, Thomas E, Philippe A, Fouliard S, Dupouy S, Marchiq I, Almena-Carrasco M, Ferry N, Arnould S, Konto C, Veys P, Qasim W; UCART19 Group. Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies. Lancet. 2020 Dec 12;396(10266):1885-1894. doi: 10.1016/S0140-6736(20)32334-5.
- Derived from this study: PubMed 40367950Li YR, Zhu Y, Fang Y, Lyu Z, Yang L. Emerging trends in clinical allogeneic CAR cell therapy. Med. 2025 Aug 8;6(8):100677. doi: 10.1016/j.medj.2025.100677. Epub 2025 May 13.
- Individual participant dataSharing planned: Yes. Qualified scientific and medical researchers can request access to anonymized patient-level and study-level clinical trial data. Access can be requested for all interventional clinical studies: * used for Marketing Authorization (MA) of medicines and new indications approved after 1 January 2014 in the European Economic Area (EEA) or the United States (US). * where Servier is the Marketing Authorization Holder (MAH). The date of the first MA of the new medicine (or the new indication) in one of the EEA Member States will be considered for this scope. In addition, access can be requested for all interventional clinical studies in patients: * sponsored by Servier * with a first patient enrolled as of 1 January 2004 onwards * for New Chemical Entity or New Biological Entity (new pharmaceutical form excluded) for which development has been terminated before any Marketing authorization (MA) approval.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing UCART19 in people with Refractory B-cell Acute Lymphoblastic Leukemia and Relapsed B-cell Acute Lymphoblastic Leukemia, run by Institut de Recherches Internationales Servier at 6 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States, France, Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and Severity of Adverse Events (time frame: From inclusion to Month 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02808442, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2021.