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

• Sponsor: Cellectis S.A. (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT04150497
• Collaborator: (none)
• Enrollment: 30
• Locations: 11
• Arm: 1
• Anticipated Duration (Months): 45.5
• Patients Per Site: 2.7
• Patients Per Site Per Month: 0.1

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
B-cell Acute Lymphoblastic Leukemia	Biological: UCART22	Phase 1

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 30 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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

Criteria

• Ages Eligible for Study: 15 Years to 70 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

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.

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Cellectis S.A.
• ClinicalTrials.gov Identifier: NCT04150497
• 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: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Cellectis S.A.: 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®)
• Additional relevant MeSH terms: Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Leukemia, Lymphoid

Study Results

No Results Posted as of Aug 25, 2022