B-cell Acute Lymphoblastic LeukemiaPhase 1/2NCT04150497
Phase 1/2 Study of UCART22 in Patients With Relapsed or Refractory CD22+ B-cell Acute Lymphoblastic Leukemia (BALLI-01)
A Phase 1/2 study testing UCART22 and CLLS52 in people with B-cell Acute Lymphoblastic Leukemia, run by Cellectis S.A. at 19 sites in 2 countries.
Trial summary
This is a first-in-human, open-label, dose escalation and expansion study of UCART22 administered intravenously to patients with relapsed or refractory B-cell acute Lymphoblastic Leukemia (B-ALL). Read more about this study
- Who is it for?
- Ages 15 to 50 years
- Conditions the study is for
- B-cell Acute Lymphoblastic Leukemia
- What is being tested?
- UCART22 and CLLS52
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 52 participants
- Where
- 19 sitesin 2 countries
- Study dates
- 14 Oct 2019to 30 Jun 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 15 to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- B-ALL blast cells expressing CD22
- Diagnosed with R/R B-ALL
- Prior therapy must include at least one standard chemotherapy regimen and at least one salvage regimen
Who cannot join
- -Prior cellular therapy or investigational cellular or gene therapy within 90 days prior to enrollment
Trial locations and contacts
19 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
19 sites in 2 countries
United States14 sites, 13 recruiting
- Recruiting
University of Colorado - Aurora Cancer Center
Aurora, Colorado, 80045
- Recruiting
Sarah Cannon - St. David's South Austin Medical Center
Austin, Texas, 78704
- Recruiting
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
- Recruiting
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263
- Recruiting
University of Chicago
Chicago, Illinois, 60647
- Recruiting
Sarah Cannon - Colorado Blood Cancer Institute
Denver, Colorado, 80218
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
- Recruiting
University of California, Los Angeles (UCLA) - Medical Center
Los Angeles, California, 90095
- Recruiting
University of Wisconsin Carbone Cancer Center
Madison, Wisconsin, 53792
- Recruiting
Sarah Cannon - HCA Research Institute
Nashville, Tennessee, 37203
- Recruiting
Memorial Sloan Kettering Cancer Center (MSKCC) David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center
New York, New York, 10021
- Withdrawn
Weill Medical College of Cornell University
New York, New York, 10065
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
- Recruiting
Sarah Cannon - Texas Transplant Institute at Methodist Hospital
San Antonio, Texas, 78229
France5 sites, 5 recruiting
- Recruiting
CHU de Nantes - Hôtel-Dieu
Nantes, 44093
- Recruiting
Hôpital Saint Louis, Unité d'Hématologie Adolescents et Jeunes Adultes Département d'Hématologie
Paris, 75010
- Recruiting
Hôpital Robert Debré - Service d'hémato-immunologie
Paris, 75019
- Recruiting
Hôpital Lyon Sud
Pierre-Bénite, 69310
- Recruiting
CHU Rennes - Hopital Pontchaillou
Rennes, 35033
About this study
Official title: Open Label Dose-escalation and Dose-expansion Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of UCART22 (Allogeneic Engineered T-cells Expressing Anti-CD22 Chimeric Antigen Receptor) in Patients With Relapsed or refractoryCD22+ B-cell Acute Lymphoblastic Leukemia (B-ALL)
This is a first-in-human, open-label, dose escalation and expansion study of UCART22 administered intravenously to patients with relapsed or refractory B-cell acute Lymphoblastic Leukemia (B-ALL). The purpose of this study is to evaluate the safety and clinical activity of UCART22 and determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D)
- Conditions
- B-cell Acute Lymphoblastic Leukemia
- Being tested
- UCART22, CLLS52
- Keywords
- B-cell Acute Lymphoblastic Leukemia (B-ALL), Relapse/Refractory B-ALL, Universal Chimeric Antigen Receptor T-Cell (UCAR-T) Therapy, Allogeneic, Transcription Activator-Like Effector Nuclease (TALEN®)
- Medical subject headings
- Burkitt Lymphoma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UCART22Biological
Allogeneic engineered T-cells expressing anti-CD22 Chimeric Antigen Receptor given following a lymphodepleting regimen
CLLS52Biological
Also called Alemtuzumab
A monoclonal antibody that recognizes a CD52 antigen
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation | Experimental | Several tested doses of UCART22 until the Maximum Tolerated Dose (MTD) is identified and establish Recommended Phase 2 Dose (RP2D)
Dose Expansion: UCART22 administered at the RP2D Receives: Biological: UCART22; Biological: CLLS52 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of AE/SAE/DLT [Safety and Tolerability]
Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study in relation to UCART22 and/or lymphodepletion
Measured: 24 Months
Dose escalation part: Occurrence of Dose Limiting Toxicities (DLTs)
Measured: Up to D28 post initial UCART22 infusion
Secondary outcomes (5)
Investigator assessed overall response rate according to the Response criteria for Acute Lymphoblastic Leukemia (ALL)
Measured: At Day 28, Day 56, Day 84, Month 3, Month 6, Month 9, Month 12, Month 15, Month 18, Month 21 and Month 24
Duration of Response
Measured: From the date of the initial response to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24
Progression Free Survival
Measured: From the first day of study treatment to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24
Overall Survival
Measured: From the first day of study treatment to the date of death from any cause, assessed up to Month 24
Pharmacokinetic (PK) profile/exposure levels of CLLS52 (Alemtuzumab) used during lymphodepletion
Measured: Lymphodepletion to Day 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2019
- Study start14 Oct 2019
- Primary completion30 Jun 2026 (estimated)
- Study completion30 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 52 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cellectis S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nitin Jain, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04150497
- Sponsor's protocol ID
- UCART22_01
- Status last verified
- Sep 2025
- First submitted
- 1 Nov 2019
- Last updated
- 9 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label Dose-escalation and Dose-expansion Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of UCART22 (Allogeneic Engineered T-cells Expressing Anti-CD22 Chimeric Antigen Receptor) in Patients With Relapsed or refractoryCD22+ B-cell Acute Lymphoblastic Leukemia (B-ALL)
- ClinicalTrials.gov ID
- NCT04150497
- Acronym / sponsor ID
- UCART22_01
- Conditions
- B-cell Acute Lymphoblastic Leukemia
- Keywords
- B-cell Acute Lymphoblastic Leukemia (B-ALL), Relapse/Refractory B-ALL, Universal Chimeric Antigen Receptor T-Cell (UCAR-T) Therapy, Allogeneic, Transcription Activator-Like Effector Nuclease (TALEN®)
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 52 participants (estimated)
- Lead sponsor
- Cellectis S.A.Industry (pharma, biotech, devices)
- Study start
- 14 Oct 2019 (actual)
- Primary completion
- 30 Jun 2026 (estimated)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 4 Nov 2019 (actual)
- 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 is a first-in-human, open-label, dose escalation and expansion study of UCART22 administered intravenously to patients with relapsed or refractory B-cell acute Lymphoblastic Leukemia (B-ALL). The purpose of this study is to evaluate the safety and clinical activity of UCART22 and determine the Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation | Experimental | Biological: UCART22, Biological: CLLS52Several tested doses of UCART22 until the Maximum Tolerated Dose (MTD) is identified and establish Recommended Phase 2 Dose (RP2D) Dose Expansion: UCART22 administered at the RP2D |
Who may qualify
- Ages
- 15 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- B-ALL blast cells expressing CD22
- Diagnosed with R/R B-ALL
- Prior therapy must include at least one standard chemotherapy regimen and at least one salvage regimen
Key exclusion criteria (1 listed)
- -Prior cellular therapy or investigational cellular or gene therapy within 90 days prior to enrollment
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 |
|---|---|---|
| UCART22Allogeneic engineered T-cells expressing anti-CD22 Chimeric Antigen Receptor given following a lymphodepleting regimen | Biological | Dose Escalation |
| CLLS52Also: AlemtuzumabA monoclonal antibody that recognizes a CD52 antigen | Biological | Dose Escalation |
Primary outcome
Incidence of AE/SAE/DLT [Safety and Tolerability]
Time frame: 24 Months
Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study in relation to UCART22 and/or lymphodepletion
Dose escalation part: Occurrence of Dose Limiting Toxicities (DLTs)
Time frame: Up to D28 post initial UCART22 infusion
5 secondary outcomes
Investigator assessed overall response rate according to the Response criteria for Acute Lymphoblastic Leukemia (ALL)
Time frame: At Day 28, Day 56, Day 84, Month 3, Month 6, Month 9, Month 12, Month 15, Month 18, Month 21 and Month 24
Duration of Response
Time frame: From the date of the initial response to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24
Progression Free Survival
Time frame: From the first day of study treatment to the date of disease progression or death from any cause, whichever occurs first, assessed up to Month 24
Overall Survival
Time frame: From the first day of study treatment to the date of death from any cause, assessed up to Month 24
Pharmacokinetic (PK) profile/exposure levels of CLLS52 (Alemtuzumab) used during lymphodepletion
Time frame: Lymphodepletion to Day 56
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2025.
- Individual participant dataSharing planned: No
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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.
Nitin Jain, MD principal investigator
M.D. Anderson Cancer Center
19 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing UCART22 and CLLS52 in people with B-cell Acute Lymphoblastic Leukemia, run by Cellectis S.A. at 19 sites in 2 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 1 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: looking for participants now, as of the sponsor's last update on 9 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, France. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Cellectis Central Contact (+1 (347) 752-4044, clinicaltrials@cellectis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of AE/SAE/DLT [Safety and Tolerability] (time frame: 24 Months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 9 Sep 2025.