NCT07081646

Brief Summary

Open-label Phase 1b/2 study with primary objective of this study is to evaluate the safety, tolerability and efficacy of AZD0120 in participants with light chain (AL) amyloidosis.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
91

participants targeted

Target at P75+ for phase_1

Timeline
57mo left

Started Aug 2025

Longer than P75 for phase_1

Geographic Reach
3 countries

18 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress15%
Aug 2025Feb 2031

First Submitted

Initial submission to the registry

July 7, 2025

Completed
16 days until next milestone

First Posted

Study publicly available on registry

July 23, 2025

Completed
26 days until next milestone

Study Start

First participant enrolled

August 18, 2025

Completed
4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 15, 2029

Expected
1.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

February 14, 2031

Last Updated

March 24, 2026

Status Verified

March 1, 2026

Enrollment Period

4 years

First QC Date

July 7, 2025

Last Update Submit

March 23, 2026

Conditions

Keywords

AZD0120AmyloidosisAL AmyloidosisCAR-TCell Therapy

Outcome Measures

Primary Outcomes (2)

  • Phase 1b: Number of Participants With incidence and severity of Treatment-emergent Adverse Events

    Through study completion, a minimum of 6 months

  • Phase 2: Proportion of Participants Experiencing a Complete Response

    Through study completion, a minimum of 6 months

Secondary Outcomes (2)

  • Phase 1b: Levels of AZD0120 in blood over time in participants with AL amyloidosis

    Through study completion, a minimum of 6 months

  • Phase 2: Percentage of participants achieving hematologic response

    Through study completion, a minimum of 6 months

Study Arms (1)

AZD0120

EXPERIMENTAL

Participants will receive weight-based dose of AZD0120.

Drug: AZD0120

Interventions

Participants will receive AZD0120 via intravenous (IV) infusion.

AZD0120

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Confirmed histopathological diagnosis of AL amyloidosis
  • One or more organs currently or historically impacted by AL amyloidosis according to consensus guidelines
  • Measurable hematologic disease: dFLC \> 20 mg/L or serum M-protein \> 5g/L
  • Relapsed disease or refractory disease defined as a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy.
  • ECOG performance status of 0 to 1
  • Must be able and willing to adhere to the study visit schedule and other protocol requirements
  • Women of child-bearing potential (WCBP) must have a negative serum pregnancy test prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use effective methods of birth control throughout the study.

You may not qualify if:

  • Have any other form of amyloidosis other than AL amyloidosis
  • Mayo Stage IIIb AL amyloidosis
  • Oxygen saturation \< 95% on room air
  • Systolic blood pressure \<100mmHg
  • NYHA class III or IV
  • Extensive GI involvement with evidence of active GI bleeding/risk of bleeding as determined by Investigator
  • Prior therapies:
  • CAR T cell therapy directed at any target
  • Prior BCMA-targeting therapy
  • Prior treatment with any FDA approved or investigational T cell engaging therapies (including T cell-directed bispecific or trispecific therapies) at any target within the last 6 months.
  • Toxicity from previous anti-cancer or anti-PC-directed therapy did not resolve to baseline levels or to Grade 1 or less except for alopecia or peripheral neuropathy.
  • Active plasma cell leukemia at the time of screening
  • Symptomatic multiple myeloma (defined as clonal bone marrow plasma cells ≥10% plus at least one myeloma-defining event per IMWG 2014)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (18)

Research Site

Phoenix, Arizona, 85028, United States

RECRUITING

Research Site

San Francisco, California, 94143, United States

NOT YET RECRUITING

Research Site

Tampa, Florida, 33612, United States

RECRUITING

Research Site

Boston, Massachusetts, 02215, United States

RECRUITING

Research Site

Detroit, Michigan, 48201, United States

RECRUITING

Research Site

Rochester, Minnesota, 55905, United States

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

New York, New York, 10065, United States

RECRUITING

Research Site

Cleveland, Ohio, 44195, United States

RECRUITING

Research Site

Nashville, Tennessee, 37232, United States

RECRUITING

Research Site

Calgary, Alberta, T2N 5G2, Canada

WITHDRAWN

Research Site

Calgary, Alberta, T2N 5G2, Canada

NOT YET RECRUITING

Research Site

Toronto, Ontario, M5G 2M9, Canada

WITHDRAWN

Research Site

Toronto, Ontario, M5G 2M9, Canada

NOT YET RECRUITING

Research Site

London, NW3 2PF, United Kingdom

WITHDRAWN

Research Site

London, NW3 2PF, United Kingdom

NOT YET RECRUITING

MeSH Terms

Conditions

Immunoglobulin Light-chain AmyloidosisAmyloidosis

Condition Hierarchy (Ancestors)

Neoplasms, Plasma CellNeoplasms by Histologic TypeNeoplasmsProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic DiseasesLymphoproliferative DisordersImmunoproliferative DisordersImmune System DiseasesParaproteinemias

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 7, 2025

First Posted

July 23, 2025

Study Start

August 18, 2025

Primary Completion (Estimated)

August 15, 2029

Study Completion (Estimated)

February 14, 2031

Last Updated

March 24, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will share

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Shared Documents
STUDY PROTOCOL, SAP, CSR

Locations