A Phase I, Open-label, Multicenter Study to Evaluate the Safety, Tolerability, Cellular Kinetics, Immunogenicity, Pharmacodynamics, and Preliminary Efficacy of AZD0120 in Participants With Multiple Myeloma

Not Applicable

Recruiting

• Conditions: Multiple Myeloma
• Interventions: Biological: AZD0120

• Registration Number: NCT07073547
• Lead Sponsor: AstraZeneca

Brief Summary

This is an interventional, modular, open-label, multicenter study to primarily evaluate the safety and tolerability of AZD0120 in adult participants with multiple myeloma (MM).

Detailed Description

This modular study aims to evaluate the safety, tolerability, cellular kinetics, pharmacodynamic effect, immunogenicity, and preliminary efficacy of AZD0120 in subjects with newly diagnosed or early relapsed or primary refractory multiple myeloma. Module 1 consists of early line MM (including newly diagnosed MM and early relapsed or primary refractory MM) with AZD0120 (for newly diagnosed multiple myeloma (NDMM), the intervention is with AZD0120 ± maintenance). Module 2 consists of NDMM with AZD0120 ± maintenance.

Study Timeline

• Study First Submitted: 2025-07-10
• Study First Submitted QC: 2025-07-10
• Study First Posted: 2025-07-18
• Study Start: 2025-07-31
• Status Verified: 2025-10
• Last Update Submitted: 2025-10-06
• Last Update Posted: 2025-10-07
• Primary Completion: 2028-06-19
• Study Completion: 2028-06-19

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

Age:

• Males and females ≥18 years of age at the time of consent

Type of Participant and Disease Characteristics:

• Participant must have documented diagnosis of MM per IMWG diagnostic criteria
• ECOG performance status of 0 or 1.
• Adequate organ and bone marrow function.

For NDMM participants:

• Participants on Module 1: Newly diagnosed multiple myeloma (NDMM) without prior anti- myeloma therapy (no more than 2 cycles of induction therapy before enrollment are acceptable)
• For participants on Module 2: Newly diagnosed MM with a maximum of 6 cycles and minimum of 4 cycles of induction therapy completed prior to screening
• Classified as high-risk MM

For Early Relapsed or Primary Refractory MM (1 or 2 prior lines of therapy) participants:

• Have received and failed 1 or 2 lines of anti-myeloma therapy
• Have received a proteasome inhibitor (PI) and immunomodulatory drug (IMiD) as part of their previous therapy
• Have documented evidence of progressive disease based on investigator's determination of response by the IMWG criteria within 1 year of starting treatment, or on or within 6 months of completing treatment of the subject's last line of anti-myeloma therapy, or have confirmed progressive disease within 6 months prior to screening and who are subsequently determined to be refractory or non-responsive to their most recent anti-myeloma treatment regimen

General

Exclusion Criteria

• Have received prior treatment with CAR T therapy directed at any target
• Known active, or prior history of central nervous system (CNS) involvement or exhibits clinical signs of meningeal involvement of multiple myeloma
• Active or history of plasma cell leukemia at the time of screening
• Seropositive for human immunodeficiency virus (HIV)
• Active Hepatitis B infection
• Active Hepatitis C infection
• Serious underlying medical condition

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AZD0120	AZD0120	AZD0120 will be administrated in one infusion

Primary Outcome Measures

Name	Time	Method
Adverse Events (AEs)	2 years	Incidence and severity of adverse events (AEs)
Serious Adverse Events (SAEs)	2 years	Incidence and severity of serious adverse events (SAEs)
Dose Limiting Toxicities (DLT)	28 days	Incidence of dose limiting toxicities events

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetic - AUC0-28d, AUCinf, AUClast	2 years	Area under the concentration time-curve of AZD0120 level
Pharmacokinetic - Cmax	2 years	Maximum AZD0120 level
Pharmacokinetic - Tmax	2 years	Time to reach Maximum AZD0120 level
Pharmacokinetic - Clast	2 years	Observed concentration of AZD0120 at last quantifiable concentration
Pharmacokinetic - Tlast	2 years	Time to last quantifiable concentration of AZD0120 level
Pharmacokinetic - Quantification of CAR transgene levels	2 years	Determination of transgene level
Efficacy - Objective Response Rate (ORR)	2 years	Defined as proportion of participants who achieve an overall response of PR or better according to the IMWG 2016 criteria
Efficacy - Complete Response Rate (CRR)	2 years	Defined as proportion of participants who achieve a CR response or sCR response according to the IMWG 2016 criteria
Efficacy - Minimal Residual Disease (MRD)	9 months	Negative rate at 9 months (± 3 months): defined as the proportion of participants with MRD negative status at 9 months (± 3 months)
Efficacy - Duration of Response (DoR)	2 years	Defined as the time from first documented confirmed response until date of documented PD per IMWG 2016 criteria or death due to any cause, whichever occurs first
Efficacy - Time to Response (TTR)	2 years	Defined as the time from infusion until the date of first documented objective response, as assessed per IMWG 2016 criteria
Humoral Immunogenicity	2 years	Prevalence and incidence ADAs against AZD0120 and the impact on PK, efficacy, and safety, as data allow
Module 2 Adverse Events (AEs) Maintenance	2 years	Incidence and severity of AEs for maintenance following AZD0120 infusion in participants with NDMM
Module 2 Serious Adverse Events (SAEs) Maintenance	2 years	Incidence and severity of SAEs for maintenance following AZD0120 infusion in participants with NDMM

Trial Locations

Locations (1)

Research Site; 🇺🇸 Houston, Texas, United States