Phase 1/2 Study of UCART22 in Patients With Relapsed or Refractory CD22+ B-cell Acute Lymphoblastic Leukemia (BALLI-01)

• ClinicalTrials.gov Identifier: NCT04150497
• Recruitment Status: Recruiting
• First Posted: November 4, 2019
• Last Update Posted: September 25, 2023
• Sponsor: Cellectis S.A.
• Information provided by (Responsible Party): Cellectis S.A.

Tracking Information

• First Submitted Date: November 1, 2019
• First Posted Date: November 4, 2019
• Last Update Posted Date: September 25, 2023
• Actual Study Start Date: October 14, 2019
• Estimated Primary Completion Date: December 31, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: September 23, 2022)

• 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 ]

Original Primary Outcome Measures (submitted: November 1, 2019)

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

Current Secondary Outcome Measures (submitted: September 23, 2022)

• 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 ]

• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Phase 1/2 Study of UCART22 in Patients With Relapsed or Refractory CD22+ B-cell Acute Lymphoblastic Leukemia (BALLI-01)
• 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)
• 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)
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: B-cell Acute Lymphoblastic Leukemia

Intervention

• Biological: UCART22
  Allogeneic engineered T-cells expressing anti-CD22 Chimeric Antigen Receptor given following a lymphodepleting regimen
• Biological: CLLS52
  A monoclonal antibody that recognizes a CD52 antigen
  Other Name: Alemtuzumab

Study Arms

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

Interventions:

• Biological: UCART22
• Biological: CLLS52

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: September 23, 2022): 40
• Original Estimated Enrollment (submitted: November 1, 2019): 30
• Estimated Study Completion Date: January 31, 2026
• Estimated Primary Completion Date: December 31, 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 15 Years to 70 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

Contact: Cellectis Central Contact; +1 (347) 752-4044; clinicaltrials@cellectis.com

• Listed Location Countries: France, United States

Administrative Information

• NCT Number: NCT04150497
• Other Study ID Numbers: UCART22_01
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Cellectis S.A.
• Original Responsible Party: Same as current
• Current Study Sponsor: Cellectis S.A.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

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

• PRS Account: Cellectis S.A.
• Verification Date: September 2023