RecruitingPhase 1Phase 2NCT07081646

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

A Phase 1b/2 Study of AZD0120 (Also Known as GC012F), a Chimeric Antigen Receptor T Cell Therapy Targeting CD19 and B Cell Maturation Antigen in Participants With Relapsed or Refractory AL Amyloidosis.


Sponsor

Alexion Pharmaceuticals, Inc.

Enrollment

91 participants

Start Date

Aug 18, 2025

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 18 Years

Inclusion Criteria7

  • 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.

Exclusion Criteria13

  • 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)

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Interventions

DRUGAZD0120

Participants will receive AZD0120 via intravenous (IV) infusion.


Locations(18)

Research Site

Phoenix, Arizona, United States

Research Site

San Francisco, California, United States

Research Site

Tampa, Florida, United States

Research Site

Boston, Massachusetts, United States

Research Site

Detroit, Michigan, United States

Research Site

Rochester, Minnesota, United States

Research Site

St Louis, Missouri, United States

Research Site

New York, New York, United States

Research Site

New York, New York, United States

Research Site

New York, New York, United States

Research Site

Cleveland, Ohio, United States

Research Site

Nashville, Tennessee, United States

Research Site

Calgary, Alberta, Canada

Research Site

Calgary, Alberta, Canada

Research Site

Toronto, Ontario, Canada

Research Site

Toronto, Ontario, Canada

Research Site

London, United Kingdom

Research Site

London, United Kingdom

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NCT07081646


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