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Clinical Trials in the UK / NCT07081646
Recruiting Phase 1/2

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

NCT07081646 · tracked via the Priya Life Science UK tracker
Sponsor
Alexion Pharmaceuticals, Inc.
Phase
Phase 1/2
Started
2025-08-18
Last updated
2026-07-02

Condition(s) studied

Relapsed AL AmyloidosisRefractory AL AmyloidosisLight Chain AmyloidosisAmyloidosis

Investigational drug(s) / intervention(s)

AZD0120

AZD0120: Participants will receive AZD0120 via intravenous (IV) infusion.

Study 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

Sex
ALL
Min age
18 Years
Max age
Healthy volunteers
No
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)

Primary outcome measure(s)

Trial sites (18)

FacilityCityRegionStatus
Research Site Phoenix Arizona Recruiting
Research Site San Francisco California Recruiting
Research Site Tampa Florida Recruiting
Research Site Boston Massachusetts Recruiting
Research Site Detroit Michigan Recruiting
Research Site Rochester Minnesota Recruiting
Research Site St Louis Missouri Recruiting
Research Site New York New York Recruiting
Research Site New York New York Recruiting
Research Site New York New York Recruiting
Research Site Cleveland Ohio Recruiting
Research Site Nashville Tennessee Recruiting
Research Site Calgary Alberta Withdrawn
Research Site Calgary Alberta Recruiting
Research Site Toronto Ontario Withdrawn
Research Site Toronto Ontario Recruiting
Research Site London United Kingdom Withdrawn
Research Site London United Kingdom Recruiting

More Alexion Pharmaceuticals, Inc. trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07081646 on ClinicalTrials.gov ↗ ← All trials in the UK