Study Investigating NTLA-5001 in Subjects With Acute Myeloid Leukemia

Intellia Therapeutics

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Acute Myeloid Leukemia

Treatments: Genetic: Arm 2: NTLA-5001; Genetic: Arm 1: NTLA-5001

Study type: Interventional

Funder types: Industry

Identifiers

NCT05066165

ITL-5001-CL-001

Details and patient eligibility

About

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).

Full 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.

Enrollment: 6 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria (abbreviated):

• Has AML as defined by World Health Organization
• Has detectable disease following first-line therapy
• Is ≥ 18 years of age.
• Carries the human leukocyte antigen-A0201 (HLA-A*02:01) allele.
• Has ECOG performance status of 0 to 1.
• Has adequate absolute total lymphocyte count
• Has adequate cardiac, renal, and liver organ function

Exclusion Criteria (abbreviated):

• Has received AML-directed therapy or immunomodulatory therapy within a specified window prior to study entry.
• Has received allogeneic hematopoietic cell transplant within 84 days, with ongoing GVHD, with recent DLI, or on active immunosuppression.
• Has CNS involvement by tumor.
• Has severe autoimmunity requiring immunomodulatory therapy.
• Has active disseminated intravascular coagulation (DIC), bleeding or coagulopathy.
• Has leukocytosis ≥ 20,000 blasts/μL despite hydroxyurea or has rapidly progressive disease
• Has human immunodeficiency virus (HIV) infection, or any uncontrolled infection.
• Female subjects are pregnant or breastfeeding; or are of childbearing potential and are unwilling to use protocol specified method of contraception.
• Male subjects who have female partners of childbearing potential and are unwilling to use protocol specified method of contraception.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

---

6 participants in 2 patient groups

Arm 1: NTLA-5001

Experimental group

Description:

Up to three escalation cohorts in phase 1 followed by one expansion cohort in phase 2. Subjects have AML and bone marrow blast count \<5%, administered by IV infusion following lymphodepleting chemotherapy.

Treatment:

Genetic: Arm 1: NTLA-5001

Arm 2: NTLA-5001

Experimental group

Description:

Up to three escalation cohorts in phase 1 followed by one expansion cohort in phase 2. Subjects have AML and bone marrow blast count ≥5%, administered by IV infusion following lymphodepleting chemotherapy.

Treatment:

Genetic: Arm 2: NTLA-5001