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Looking for participantsPhase1/Phase2

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

Sponsor: Alexion Pharmaceuticals, Inc.

NCT ID: NCT07081646

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
AZD0120 (drug)
How long the study runs
Study runs about 66 months (dates as stated)
About the drug or intervention
AZD0120 — drug: Participants will receive AZD0120 via intravenous (IV) infusion.
Patient visit burden
Not specified by the sponsor

In plain English

This is an early-stage study (Phase 1b/2) of CAR T cell therapy (a treatment where your own immune cells are changed in a lab to attack cancer) targeting two proteins called CD19 and BCMA. It is for people whose AL amyloidosis (a rare condition where abnormal proteins build up in organs) has come back or has not responded to earlier treatment. The study is run by Alexion Pharmaceuticals, Inc.

Who can take part

  • A confirmed diagnosis of AL amyloidosis based on tissue testing
  • At least one organ affected by AL amyloidosis, now or in the past
  • Measurable disease in the blood (dFLC over 20 mg/L or serum M-protein over 5 g/L)
  • Disease has come back or not responded to at least one earlier treatment, and more treatment is needed
  • Able to carry out daily activities with little or some effort (performance status 0 to 2)
  • Able and willing to follow the study visit schedule
  • Negative pregnancy test before treatment for women who can become pregnant, and highly effective birth control used throughout the study by all sexually active participants who can have children

Who may not be able to

  • Any type of amyloidosis other than AL amyloidosis
  • Mayo Stage IIIb AL amyloidosis (an advanced stage of the illness)
  • Oxygen levels below 95% on room air
  • Top blood pressure number (systolic) below 100 mmHg
  • Severe heart failure symptoms (NYHA class III or IV)
  • Extensive gut involvement with active bleeding or a high risk of bleeding
  • Previous CAR T cell therapy of any kind
  • Previous treatment targeting BCMA
  • Treatment with any approved or experimental T cell engaging therapies (including bispecific or trispecific therapies) in the last 6 months
  • Side effects from earlier cancer or plasma cell treatment that have not got better, except hair loss or nerve tingling/numbness
  • Active plasma cell leukaemia at screening
  • Symptomatic myeloma (bone marrow plasma cells of 10% or more plus at least one myeloma-defining event)

What taking part involves

  • • Not stated — ask the trial team

Time commitment: Not stated — ask the trial team about visit schedule, duration, and what taking part involves.

Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.

Type of study
Testing a treatment
Ages
18 Years and over
Who
All
Number of participants
91
Started
2025-08-18
Last checked
2026-06

Plain English Summary

What is this study?

  • • Testing a new treatment for relapsed al amyloidosis
  • • Phase1/Phase2 - 91 participants
  • • 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

Who can take part?

  • • Ages 18 Years and over
  • • Diagnosed with relapsed al amyloidosis

Where?

  • • London - Research Site
  • • London - Research Site

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

Relapsed AL AmyloidosisRefractory AL AmyloidosisLight Chain AmyloidosisAmyloidosis

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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Still need:

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  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 18 Years and over
  • Who can join: All genders
  • How fit you need to be: ECOG 0 or better

Biomarkers mentioned

have a negative

Treatment history

Treatments you must have had:

  • ✓ a negative serum and/or urine pregnancy test result

What the study is looking for

  • ✓Confirmed histopathological diagnosis of AL amyloidosis
  • ✓One or more organs currently or historically impacted by AL amyloidosis according to consensus guidelines
  • ✓Measurable blood 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...
  • ✓Fit enough for normal activity (ECOG 0) to 2

Who cannot take part

  • ✗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
See the full criteria
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)

Where Is This Study? (2 UK sites)

Research Site

London NW3 2PF, United Kingdom

WITHDRAWN

Research Site

London NW3 2PF, United Kingdom

Recruiting

How to Get in Touch

Alexion Pharmaceuticals, Inc. (Sponsor)

Sponsor contact

CONTACT

1-855-752-2356 clinicaltrials@alexion.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-06