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NCT07081646PHASE1, PHASE2Recruiting

A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.

Alexion Pharmaceuticals, Inc.

Start Date

8/18/2025

Completion Date

2/14/2031

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.

Eligibility Criteria

Age Range: 18 years to No maximum

Inclusion Criteria: * 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 or refractory disease with a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy. * ECOG performance status of 0 to 2 * 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 and/or urine pregnancy test result prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use highly effective methods of birth control throughout the study. Exclusion Criteria: * 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: 1. CAR T cell therapy directed at any target 2. Prior BCMA-targeting therapy 3. 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)

Interventions

DRUG

AZD0120

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Conditions

Relapsed AL AmyloidosisRefractory AL AmyloidosisLight Chain AmyloidosisAmyloidosis

Locations

Research Site

Phoenix, Arizona 85028

United States

Research Site

San Francisco, California 94143

United States

Research Site

Tampa, Florida 33612

United States

Research Site

Boston, Massachusetts 02215

United States

Research Site

Detroit, Michigan 48201

United States

Research Site

Rochester, Minnesota 55905

United States

Research Site

St Louis, Missouri 63110

United States

Research Site

New York, New York 10016

United States

Research Site

New York, New York 10032

United States

Research Site

New York, New York 10065

United States

Research Site

Cleveland, Ohio 44195

United States

Research Site

Nashville, Tennessee 37232

United States

Research Site

Calgary, Alberta T2N 5G2

Canada

Research Site

Calgary, Alberta T2N 5G2

Canada

Research Site

Toronto, Ontario M5G 2M9

Canada

Research Site

Toronto, Ontario M5G 2M9

Canada

Research Site

London, NW3 2PF

United Kingdom

Research Site

London, NW3 2PF

United Kingdom