Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia

Phase 1

Terminated

• Conditions: Acute Myeloid Leukemia

• Registration Number: NCT05066165
• Lead Sponsor: Intellia Therapeutics

Brief 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.

Study Timeline

• Study First Submitted: 2021-09-23
• Study First Submitted QC: 2021-09-23
• Study First Posted: 2021-10-04
• Study Start: 2021-12-17
• Primary Completion: 2022-07-21
• Study Completion: 2022-08-31
• Results First Submitted: 2023-08-31
• Status Verified: 2023-12
• Results First Submitted QC: 2023-12-14
• Last Update Submitted: 2023-12-14
• Results First Posted: 2023-12-28
• Last Update Posted: 2023-12-28

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Primary Outcome Measures

Name	Time	Method
Participants That Experienced Dose-limiting Toxicities (DLTs)	Primary DLT assessment from NTLA-5001 infusion up to 28 days post-infusion	DLTs were defined as events with onset within 28 days of infusion. AEs were collected from time of informed consent through the Week 112 visit. AEs were coded using Medical Dictionary for Regulatory Activities (MedDRA) version 24.0. Severity of AEs was assessed by the investigator using the National Cancer Institute Common Terminology Criteria for Adverse Events, Version 5.0 toxicity grading criteria. The measure reported below for the primary outcome consists of DLT data only. Adverse events are reported in the Adverse Event section of this presentation.

Secondary Outcome Measures

Name	Time	Method
Frequency of NTLA-5001 T-cell Receptor (TCR) Transgene Copy Number in the Peripheral Blood	From NTLA-5001 infusion up to 4 weeks post-infusion	Frequency of edited TCR transgene copy number in the peripheral blood of the participants was determined by droplet digital polymerase chain reaction (ddPCR).
Tumor Response in Participants With AML	From NTLA-5001 infusion up to 4 weeks post-infusion	Rate of objective response, where response is complete response without measurable residual disease (CRMRD-) (Arm 1). Rate of objective response, where response is CRMRD-, complete response, complete remission with incomplete hematologic recovery, morphologic leukemia-free state, and partial remission (Arm 2).
Response Duration in Participants With AML	From NTLA-5001 infusion up to 4 weeks post-infusion	Bone marrow results were planned to be used to determine the duration of response / remission (DOR) for subjects with objective response, from first response until MRD was measured above the lower level of detection for the central laboratory assay, or death from any cause, whichever occurred first (Arm 1). Bone marrow results were planned to be used to determine DOR for subjects with composite CR, from first response to progression or death due to any cause, whichever occurred first (Arm 2).
Persistence of NTLA-5001 T Cell Receptor (TCR) Transgene Copy in Peripheral Blood	From NTLA-5001 infusion up to 4 weeks post-infusion	Persistence of edited TCR transgene copy in the peripheral blood of the participants determined by ddPCR.
Disease Progression in Participants With AML	From NTLA-5001 infusion up to 4 weeks post-infusion	Bone marrow results were used to determine the time to clinical progression. Progressive disease was defined as an increase from baseline of at least 25% of bone marrow blasts or an absolute increase of at least 5,000 cells/μL in the number of circulating leukemia cells

Trial Locations

Locations (10)

Research Site 8; 🇬🇧 London, United Kingdom

Research Site 9; 🇬🇧 London, United Kingdom

Research Site 2; 🇺🇸 Los Angeles, California, United States

Research Site 5; 🇺🇸 Tampa, Florida, United States

Research Site 6; 🇺🇸 Portland, Oregon, United States

Research Site 1; 🇺🇸 Boston, Massachusetts, United States

Research Site 3; 🇺🇸 Houston, Texas, United States

Research Site 10; 🇬🇧 Leeds, United Kingdom

Research Site 4; 🇺🇸 Milwaukee, Wisconsin, United States

Research Site 7; 🇬🇧 Manchester, United Kingdom