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

Phase 1

Recruiting

• Conditions: B-cell Acute Lymphoblastic Leukemia
• Interventions: Biological: UCART22; Biological: CLLS52

• Registration Number: NCT04150497
• Lead Sponsor: Cellectis S.A.

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 available

Study Timeline

• Study Start: 2019-10-14
• Study First Submitted: 2019-11-01
• Study First Submitted QC: 2019-11-01
• Study First Posted: 2019-11-04
• Status Verified: 2023-09
• Last Update Submitted: 2024-08-23
• Last Update Posted: 2024-08-26
• Primary Completion: 2026-01-31
• Study Completion: 2026-01-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Dose Escalation	CLLS52	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
Dose Escalation	UCART22	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

Primary Outcome Measures

Name	Time	Method
Dose escalation part: Occurrence of Dose Limiting Toxicities (DLTs)	Up to D28 post initial UCART22 infusion
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 in relation to UCART22 and/or lymphodepletion

Secondary Outcome Measures

Name	Time	Method
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
Pharmacokinetic (PK) profile/exposure levels of CLLS52 (Alemtuzumab) used during lymphodepletion	Lymphodepletion to Day 56
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

Trial Locations

Locations (18)

University of California, Los Angeles (UCLA) - Medical Center; 🇺🇸 Los Angeles, California, United States

MD Anderson Cancer Center; 🇺🇸 Houston, Texas, United States

Sarah Cannon - Texas Transplant Institute at Methodist Hospital; 🇺🇸 San Antonio, Texas, United States

Sarah Cannon - Colorado Blood Cancer Institute; 🇺🇸 Denver, Colorado, United States

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

University of Colorado - Aurora Cancer Center; 🇺🇸 Aurora, Colorado, United States

Dana Farber Cancer Institute; 🇺🇸 Boston, Massachusetts, United States

University of Chicago; 🇺🇸 Chicago, Illinois, United States

Roswell Park Comprehensive Cancer Center; 🇺🇸 Buffalo, New York, United States

Sarah Cannon - St. David's South Austin Medical Center; 🇺🇸 Austin, Texas, United States

Weill Medical College of Cornell University; 🇺🇸 New York, New York, 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, United States

CHU de Nantes - Hôtel-Dieu; 🇫🇷 Nantes, France

Hôpital Lyon Sud; 🇫🇷 Pierre-Benite, France

CHU Rennes - Hopital Pontchaillou; 🇫🇷 Rennes, France

Sarah Cannon - HCA Research Institute; 🇺🇸 Nashville, Tennessee, United States

Hôpital Saint Louis, Unité d'Hématologie Adolescents et Jeunes Adultes Département d'Hématologie; 🇫🇷 Paris, France

Hôpital Robert Debré - Service d'hémato-immunologie; 🇫🇷 Paris, France