NCT05066165

Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia

Phase 1/2a, Single Dose Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Intellia Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study will be conducted to evaluate the safety, tolerability, cellular kinetics (CK), activity, and pharmacodynamics (PD) of NTLA-5001 in participants with Acute Myeloid Leukemia (AML).

Detailed description

This 2-part first in human (FIH) study is comprised of two open-label arms. It is a multi-center, Phase 1/2a study evaluating the safety and activity of NTLA-5001 in subjects with persistent or recurrent Acute Myeloid Leukemia after first-line or later therapy.

Conditions

• Acute Myeloid Leukemia

Interventions

Type	Name	Description
GENETIC	Arm 1: NTLA-5001	Autologous WT1-directed TCR T cells engineered ex vivo using CRISPR/Cas9 as intravenous infusion after pre-conditioning chemotherapy. Cyclophosphamide and Fludarabine will be administered on Day -5, -4, and -3 as intravenous infusion.
GENETIC	Arm 2: NTLA-5001	Autologous WT1-directed TCR T cells engineered ex vivo using CRISPR/Cas9 as intravenous infusion after pre-conditioning chemotherapy. Cyclophosphamide and Fludarabine will be administered on Day -5, -4, and -3 as intravenous infusion.

Timeline

• Start date: 2021-12-17
• Primary completion: 2022-07-21
• Completion: 2022-08-31
• First posted: 2021-10-04
• Last updated: 2023-12-28
• Results posted: 2023-12-28

Locations

10 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study