Recruiting
Phase 1
Phase 2

AZD0120

Sponsor:

Alexion Pharmaceuticals, Inc.

Code:

NCT07081646

Conditions

Relapsed AL Amyloidosis

Refractory AL Amyloidosis

Light Chain Amyloidosis

Amyloidosis

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

AZD0120

Study Details

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.

Conditions

Relapsed AL Amyloidosis

Refractory AL Amyloidosis

Light Chain Amyloidosis

Amyloidosis

Study ID

NCT07081646

Start date

Aug 18, 2025

Status verified date

Jun, 2026

Completion date

Feb 14, 2031

Anticipated

Primary completion date

Aug 15, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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)

Study Design

Enrollment

91 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: AZD0120

Participants will receive weight-based dose of AZD0120.

Interventions

AZD0120

Participants will receive AZD0120 via intravenous (IV) infusion.

Primary outcome measure

  • Phase 1b: Number of Participants With incidence and severity of Treatment-emergent Adverse Events [ Time Frame: Through study completion, a minimum of 6 months ]
  • Phase 2: Proportion of Participants Achieving Complete Response [ Time Frame: Through study completion, a minimum of 6 months ]

Central Contacts and Locations

Central contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

1-855-752-2356clinicaltrials@alexion.com

Locations

Research Site

Recruiting

Phoenix, Arizona, United States, 85028

Research Site

Recruiting

San Francisco, California, United States, 94143

Research Site

Recruiting

Tampa, Florida, United States, 33612

Research Site

Recruiting

Boston, Massachusetts, United States, 02215

Research Site

Recruiting

Detroit, Michigan, United States, 48201

Research Site

Recruiting

Rochester, Minnesota, United States, 55905

Research Site

Recruiting

St Louis, Missouri, United States, 63110

Research Site

Recruiting

New York, New York, United States, 10016

Research Site

Recruiting

New York, New York, United States, 10032

Research Site

Recruiting

New York, New York, United States, 10065

Research Site

Recruiting

Cleveland, Ohio, United States, 44195

Research Site

Recruiting

Nashville, Tennessee, United States, 37232

Research Site

Recruiting

Calgary, Alberta, Canada, T2N 5G2

Research Site

Recruiting

Toronto, Ontario, Canada, M5G 2M9

More Information

Sponsor

Alexion Pharmaceuticals, Inc.

Last update posted

Jul 2, 2026

Last verified

Jun, 2026

Keywords

  • AZD0120
  • AL Amyloidosis
  • CAR-T
  • Cell Therapy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc. on 2026-07-02.