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

Not Applicable

Not yet recruiting

• Conditions: Relapsed AL Amyloidosis; Amyloidosis; Refractory AL Amyloidosis; Light Chain Amyloidosis
• Interventions: Drug: AZD0120

• Registration Number: NCT07081646
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-07
• Study First Submitted: 2025-07-07
• Study First Submitted QC: 2025-07-15
• Last Update Submitted: 2025-07-15
• Study First Posted: 2025-07-23
• Last Update Posted: 2025-07-23
• Study Start: 2025-10-15
• Primary Completion: 2029-08-15
• Study Completion: 2031-02-14

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 91

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 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 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

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AZD0120	AZD0120	Participants will receive weight-based dose of AZD0120.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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