Phase 1 Study of UCART22 in Patients With Relapsed or Refractory CD22+ B-cell Acute Lymphoblastic Leukemia (BALLI-01)

Cellectis S.A. (Industry)
Overall Status
Recruiting ID

Study Details

Study Description

Brief Summary

This is a Phase 1, 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)

Condition or Disease Intervention/Treatment Phase
  • Biological: UCART22
Phase 1

Study Design

Study Type:
Anticipated Enrollment :
30 participants
Intervention Model:
Single Group Assignment
None (Open Label)
Primary Purpose:
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)
Actual Study Start Date :
Oct 14, 2019
Anticipated Primary Completion Date :
Jul 31, 2023
Anticipated Study Completion Date :
Jul 31, 2023

Arms and Interventions

Arm Intervention/Treatment
Experimental: Dose Escalation

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

Biological: UCART22
Allogeneic engineered T-cells expressing anti-CD22 Chimeric Antigen Receptor given following a lymphodepleting regimen

Outcome Measures

Primary Outcome Measures

  1. Incidence of AE/SAE/DLT [Safety and Tolerability) [24 Months]

    Incidence, nature, and severity of adverse events and serious adverse events (SAEs) throughout the study

  2. For Dose Expansion: confirmation of the RP2D in patients with R/R B-ALL who have failed a CD19 directed therapy [24 Months]

Secondary Outcome Measures

  1. Response Assessment determined using the response criteria as defined in NCCN guidelines version 1.2020 for Acute Lymphoblastic Leukemia [At Day 28, Day 56, Day 84, Month 3, Month 6, Month 9, Month 12, Month 15, Month 18, Month 21 and Month 24]

  2. 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]

  3. 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]

  4. Overall Survival [From the first day of study treatment to the date of death from any cause, assessed up to Month 24]

Eligibility Criteria


Ages Eligible for Study:
15 Years to 70 Years
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Inclusion Criteria:

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

Exclusion Criteria:

Prior cellular therapy or investigational cellular or gene therapy within 60 days prior to enrollment

Contacts and Locations


Site City State Country Postal Code
1 University of California, Los Angeles (UCLA) - Medical Center Los Angeles California United States 90095
2 University of Chicago Chicago Illinois United States 60647
3 Dana Farber Cancer Institute Boston Massachusetts United States 02215
4 Memorial Sloan Kettering Cancer Center (MSKCC) David H. Koch Center for Cancer Care at Memorial Sloan Kettering Cancer Center New York New York United States 10021
5 Weill Medical College of Cornell University New York New York United States 10065
6 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
7 MD Anderson Cancer Center Houston Texas United States 77030
8 CHU de Nantes - Hôtel-Dieu Nantes France 44093
9 Hôpital Saint Louis, Unité d'Hématologie Adolescents et Jeunes Adultes Département d'Hématologie Paris France 75010
10 Hôpital Robert Debré - Service d'hémato-immunologie Paris France 75019
11 CHU Rennes - Hopital Pontchaillou Rennes France 35033

Sponsors and Collaborators

  • Cellectis S.A.


  • Principal Investigator: Nitin Jain, MD, M.D. Anderson Cancer Center

Study Documents (Full-Text)

None provided.

More Information


None provided.
Responsible Party:
Cellectis S.A. Identifier:
Other Study ID Numbers:
  • UCART22_01
First Posted:
Nov 4, 2019
Last Update Posted:
Aug 25, 2022
Last Verified:
Aug 1, 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:
Studies a U.S. FDA-regulated Drug Product:
Studies a U.S. FDA-regulated Device Product:
Keywords provided by Cellectis S.A.
Additional relevant MeSH terms:

Study Results

No Results Posted as of Aug 25, 2022