Evaluating a new CAR-T therapy for patients with relapsed or refractory acute myeloid leukemia
Phase I, Open Label Dose Escalation and Dose-Expansion Study to Evaluate the Safety, Expansion, Persistence, and Clinical Activity of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor), Administered in Patients With Relapsed/Refractory Acute Myeloid Leukemia
This study is testing a new CAR-T therapy to see if it can help people with relapsed or hard-to-treat acute myeloid leukemia feel better and improve their health.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 65 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Cellectis S.A. Industry-sponsored |
| Drugs / interventions | rituximab, Chimeric Antigen Receptor |
| Locations | 9 sites (San Francisco, California and 8 other locations) |
| Trial ID | NCT03190278 on ClinicalTrials.gov |
What this trial studies
This Phase 1 clinical trial is an open-label, dose-escalation and dose-expansion study that assesses the safety and efficacy of Universal Chimeric Antigen Receptor T-cells (UCART123v1.2) targeting CD123 in patients with relapsed or refractory acute myeloid leukemia (AML). The study aims to determine the Maximum Tolerated Dose (MTD) and the Recommended Phase 2 Dose (RP2D) while monitoring clinical activity and safety outcomes. Participants will receive the UCART123v1.2 treatment after meeting specific eligibility criteria, including having CD123+ blast cells and adequate organ function.
Who should consider this trial
Good fit: Ideal candidates include patients with relapsed or primary refractory AML who have CD123+ blast cells and meet specific health criteria.
Not a fit: Patients with acute promyelocytic leukemia, central nervous system leukemia, or those who have had multiple prior stem cell transplants may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could provide a new treatment option for patients with relapsed or refractory acute myeloid leukemia, potentially improving survival rates.
How similar studies have performed: Other studies using CAR-T therapies have shown promising results, indicating potential success for this novel approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Main Inclusion Criteria: * Patients with relapsed or primary refractory AML (as defined in World Health Organization \[WHO\] criteria) with ≥5% bone marrow blasts * Patients with CD123+ blast cells (verified by flow cytometry) * Eastern Cooperative Oncology Group Performance Status (ECOG-PS) of ≤1 * Adequate organ function, including bone marrow, renal, hepatic, pulmonary, and cardiac function based on the last assessment performed within screening period * (Dose-escalation) Identified donor and transplant strategy prior to lymphodepletion (LD) * Other criteria may apply Main Exclusion Criteria: * Patients with acute promyelocytic leukemia (APL) or central nervous system (CNS) Leukemia * Previous investigation gene or cell therapy (including CAR) * \> 1 prior allogeneic stem cell transplantations (SCTs) * Prior treatment with rituximab or other anti-cluster of differentiation 20 (anti-CD20) therapy within 3 months * Any known active or uncontrolled infection * Other criteria may apply
Where this trial is running
San Francisco, California and 8 other locations
- University of California, San Francisco (UCSF) - Helen Diller Family Comprehensive Cancer Center — San Francisco, California, United States (Recruiting)
- Sylvester Comprehensive Cancer Center — Miami, Florida, United States (Withdrawn)
- H. Lee Moffitt Cancer Center & Research Institute — Tampa, Florida, United States (Recruiting)
- Northwestern University — Chicago, Illinois, United States (Recruiting)
- Dana-Farber Cancer Institute — Boston, Massachusetts, United States (Recruiting)
- Roswell Park Cancer Institute — Buffalo, New York, United States (Recruiting)
- Weill Medical College of Cornell University — New York, New York, United States (Recruiting)
- University of Pennsylvania - Abramson Cancer Center — Philadelphia, Pennsylvania, United States (Recruiting)
- MD Anderson Cancer Center — Houston, Texas, United States (Recruiting)
Study contacts
- Principal investigator: Gail Roboz, Dr — Weill Medical College of Cornell University
- Study coordinator: Cellectis Central Contact
- Email: clinicaltrials@cellectis.com
- Phone: 1-347-752-4044
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.