An Open-label Study of AZD0120 in Adults With Multiple Sclerosis

NCT07224373 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: D831DC0000129707
• Study Type: interventional
• Target: 24
• Locations: 5 countries
• Sites: 19

Brief Summary

This trial is a Phase 1b, open-label, multi-center, clinical study of AZD0120, a BCMA/CD19 dual targeting CAR+ T-cell therapy, to evaluate the safety and tolerability in adult participants with Multiple Sclerosis.

Conditions

Multiple Sclerosis

Keywords

AZD0120; Multiple Sclerosis; MS; RMS; PMS

Outcome Measures

Primary Outcomes (4)

• Evaluate the safety, and tolerability of AZD0120 in participants with MS (disease cohort 1: RMS; disease cohort 2: PMS)

  Incidence and severity of Dose Limiting Toxicity (DLT) over 104 weeks following AZD0120 administration.

  Day 1 to day 29, and over 104 weeks

• Evaluate the safety, and tolerability of AZD0120 in participants with MS (disease cohort 1: RMS; disease cohort 2: PMS)

  Incidence and severity of Adverse Events (AE) over 104 weeks following AZD0120 administration.

  Day 1 to day 29, and over 104 weeks

• Evaluate the safety, and tolerability of AZD0120 in participants with MS (disease cohort 1: RMS; disease cohort 2: PMS)

  Incidence and severity of Serious Adverse Events (SAE) over 104 weeks following AZD0120 administration.

  Day 1 to day 29, and over 104 weeks

• Evaluate the safety, and tolerability of AZD0120 in participants with MS (disease cohort 1: RMS; disease cohort 2: PMS)

  Incidence and severity of Treatment Emergent Adverse Events (TEAE) over 104 weeks following AZD0120 administration.

  Day 1 to day 29, and over 104 weeks

Secondary Outcomes (33)

• Evaluate the optimum regimen with AZD0120 in MS participants to determine the RP2D in each disease cohort

  Over 104 weeks

• Evaluate the optimum regimen with AZD0120 in MS participants to determine the RP2D in each disease cohort

  Over 104 weeks

• Evaluate the preliminary efficacy of AZD0120 in RMS and PMS

  Over 104 weeks

• Evaluate the preliminary efficacy of AZD0120 in RMS and PMS

  Over 104 weeks

• Evaluate the preliminary efficacy of AZD0120 in RMS and PMS

  Over 104 weeks

Study Arms (2)

Arm/Group 1: AZD0120 RMS

EXPERIMENTAL

AZD0120 RMS

Biological: AZD0120 - Regimen 1; Biological: AZD0120 - Regimen 2

Arm/Group 2: AZD0120 PMS

EXPERIMENTAL

AZD0120 PMS

Biological: AZD0120 - Regimen 1; Biological: AZD0120 - Regimen 2

Interventions

AZD0120 - Regimen 1 BIOLOGICAL

Regimen 1, infusion of AZD0120

Arm/Group 1: AZD0120 RMS; Arm/Group 2: AZD0120 PMS

AZD0120 - Regimen 2 BIOLOGICAL

Regimen 2, infusion of AZD0120

Arm/Group 1: AZD0120 RMS; Arm/Group 2: AZD0120 PMS

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Diagnosis of RMS according to the 2024 McDonald Criteria (Montalban et al 2025) or diagnosis of relapsing, active SPMS according to Lublin et al 2014.
• Participants should have an EDSS of ≤ 6.5 at screening.
• Evidence of active disease (clinical relapses and MRI activities within 2 years prior to screening), or intolerance, while on a high efficacy disease-modifying therapy for ≥ 6 months.
• Diagnosis of PPMS according to the 2024 McDonald Criteria (Montalban et al 2025) or non-relapsing SPMS according to Lublin et al 2014.
• Participants must have an EDSS of ≥ 3.0 and ≤ 6.5 at screening.
• Inadequate response ≥ 1 heDMT with ≥ 6 months treatment or intolerance.

You may not qualify if:

• Participants are excluded from the study if any of the following criteria apply:
• Any prior CAR-T or CAR-NK cell exposure.
• Underwent splenectomy within 12 months prior to signing the ICF.
• Received a solid organ transplant at any time or on an active transplant waiting list.
• Prior treatment with autologous hematopoietic stem cell transplantation or total lymphoid irradiation.
• Cardiac conditions or any other significant cardiac condition that would present undue risk to the participant in the investigator's opinion:
• Any other central nervous system disease including epilepsy, convulsive seizures, organic encephalopathy syndrome, non-MS related paralysis, aphasia, stroke, severe brain injury, dementia, Parkinson's disease or associated movement disorder, psychosis, CNS vasculitis, or any other neurological disease that may impact the ability to evaluate neurotoxicity. History of a seizure disorder even if the seizure disorder is well controlled with anti-epileptics.
• Participant has significant psychiatric condition (active or history of).
• History of other immune-mediated disease that required continued systemic immunosuppression/systemic disease-modifying agents.
• Evidence of clinically significant bleeding or active bleeding diathesis within 90 days before screening
• History of malignancy or ongoing treatment for prior malignancy.
• Inborn error of immunity and/or primary immunodeficiency.
• Seropositive for HIV or HTLV (including any history of HIV or HTLV).
• Active viral (any etiology, HBV, HCV) hepatitis are excluded.
• Major surgery within 4 weeks prior to apheresis or lymphodepletion or has surgery planned during the study or within 4 weeks after study treatment administration.

Sponsors & Collaborators

• AstraZeneca: lead

Study Sites (19)

Research Site

Tucson, Arizona, 85719, United States

WITHDRAWN

Research Site

Aurora, Colorado, 80045, United States

NOT YET RECRUITING

Research Site

Washington D.C., District of Columbia, 20010, United States

NOT YET RECRUITING

Research Site

St Louis, Missouri, 63110, United States

RECRUITING

Research Site

New York, New York, 10016, United States

RECRUITING

Research Site

New York, New York, 10032, United States

RECRUITING

Research Site

Cleveland, Ohio, 44195, United States

NOT YET RECRUITING

Research Site

Seattle, Washington, 98122, United States

RECRUITING

Research Site

Milwaukee, Wisconsin, 53226, United States

NOT YET RECRUITING

Research Site

Liverpool, 2170, Australia

NOT YET RECRUITING

Research Site

Melbourne, 3000, Australia

RECRUITING

Research Site

Melbourne, 3004, Australia

NOT YET RECRUITING

Research Site

Waratah, 2298, Australia

NOT YET RECRUITING

Research Site

Montreal, Quebec, H3A 1A1, Canada

NOT YET RECRUITING

Research Site

Leipzig, 04103, Germany

WITHDRAWN

Research Site

Magdeburg, 39120, Germany

WITHDRAWN

Research Site

Würzburg, DE-97072, Germany

WITHDRAWN

Research Site

Cambridge, CB2 2QQ, United Kingdom

WITHDRAWN

Research Site

London, SE5 9RS, United Kingdom

WITHDRAWN

MeSH Terms

Conditions

Multiple Sclerosis

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

1-877-240-9479; information.center@astrazeneca.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 21, 2025
• First Posted: November 4, 2025
• Study Start: December 9, 2025
• Primary Completion (Estimated): December 7, 2028
• Study Completion (Estimated): December 7, 2028
• Last Updated: May 22, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (9); GB (2); AU (4); DE (3); CA (1)