Phase 1/2 Study of UCART22 in Patients With Relapsed or Refractory CD22+ B-cell Acute Lymphoblastic Leukemia (BALLI-01)
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)
1 other identifier
interventional
52
2 countries
19
Brief 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). 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)
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Oct 2019
Longer than P75 for phase_1
19 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
October 14, 2019
CompletedFirst Submitted
Initial submission to the registry
November 1, 2019
CompletedFirst Posted
Study publicly available on registry
November 4, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2026
ExpectedStudy Completion
Last participant's last visit for all outcomes
June 30, 2026
September 9, 2025
September 1, 2025
6.7 years
November 1, 2019
September 8, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
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
24 Months
Dose escalation part: Occurrence of Dose Limiting Toxicities (DLTs)
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)
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
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
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
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
Lymphodepletion to Day 56
Study Arms (1)
Dose Escalation
EXPERIMENTALSeveral 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
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- Prior cellular therapy or investigational cellular or gene therapy within 90 days prior to enrollment
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Cellectis S.A.lead
Study Sites (19)
University of California, Los Angeles (UCLA) - Medical Center
Los Angeles, California, 90095, United States
University of Colorado - Aurora Cancer Center
Aurora, Colorado, 80045, United States
Sarah Cannon - Colorado Blood Cancer Institute
Denver, Colorado, 80218, United States
University of Chicago
Chicago, Illinois, 60647, United States
Dana Farber Cancer Institute
Boston, Massachusetts, 02215, United States
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263, United States
Memorial Sloan Kettering Cancer Center (MSKCC) David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center
New York, New York, 10021, United States
Weill Medical College of Cornell University
New York, New York, 10065, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Sarah Cannon - HCA Research Institute
Nashville, Tennessee, 37203, United States
Sarah Cannon - St. David's South Austin Medical Center
Austin, Texas, 78704, United States
MD Anderson Cancer Center
Houston, Texas, 77030, United States
Sarah Cannon - Texas Transplant Institute at Methodist Hospital
San Antonio, Texas, 78229, United States
University of Wisconsin Carbone Cancer Center
Madison, Wisconsin, 53792, United States
CHU de Nantes - Hôtel-Dieu
Nantes, 44093, France
Hôpital Saint Louis, Unité d'Hématologie Adolescents et Jeunes Adultes Département d'Hématologie
Paris, 75010, France
Hôpital Robert Debré - Service d'hémato-immunologie
Paris, 75019, France
Hôpital Lyon Sud
Pierre-Bénite, 69310, France
CHU Rennes - Hopital Pontchaillou
Rennes, 35033, France
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Nitin Jain, MD
M.D. Anderson Cancer Center
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 1, 2019
First Posted
November 4, 2019
Study Start
October 14, 2019
Primary Completion (Estimated)
June 30, 2026
Study Completion (Estimated)
June 30, 2026
Last Updated
September 9, 2025
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will not share