ClinicalTrials.gov record NCT07081646
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.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 1b/2 interventional study tests AZD0120 (GC012F), a CAR T cell therapy targeting CD19 and BCMA, in adults with relapsed or refractory light‑chain (AL) amyloidosis. Participants receive a weight‑based intravenous dose in a single treatment group with no masking. The study evaluates safety, tolerability, and measures of hematologic response, with follow-up through study completion (minimum six months).
What it is studying. The study is testing the safety, tolerability, and anti‑disease activity of AZD0120, a CAR T cell therapy that targets CD19 and B cell maturation antigen (BCMA), in people with relapsed or refractory AL amyloidosis.
What the researchers hope to learn. To evaluate the safety and tolerability of AZD0120 (including incidence and severity of treatment‑emergent adverse events); to measure AZD0120 levels in blood over time; and to assess hematologic response rates and the proportion of participants achieving complete response.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- AZD0120
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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:
- 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)
Study description and what participation involves
The registry record has no detailed description beyond the summary above.
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants receive a weight‑based intravenous (IV) infusion of AZD0120 and are followed for safety and response. They will have blood tests over time (including drug-level measurements and tests to track disease response), regular clinic visits, and monitoring for side effects. Women will have pregnancy testing before treatment, and sexually active participants must use effective contraception during the study. The study includes follow-up visits for at least six months to assess adverse events and response.
How the study is structured. Open‑label, single‑group Phase 1b/2 treatment trial. All participants receive a weight‑based intravenous infusion of AZD0120; there is no placebo or randomization and no masking. Participants are monitored for adverse events and for hematologic and complete response outcomes, with at least six months of follow-up reported.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in Canada, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on AL amyloidosis
Important dates
- Study start
- Aug 18, 2025
- Primary completion
- Aug 15, 2029 (estimated)
- Study completion
- Feb 14, 2031 (estimated)
- Target enrollment
- 91 participants (estimated)
- Last updated on registry
- Jul 2, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Alexion Pharmaceuticals, Inc. (Sponsor) 📞 1-855-752-2356 ✉ clinicaltrials@alexion.com
Participating sites (18)
- Research SiteRecruiting
Phoenix, Arizona, 85028 · United States
- Research SiteRecruiting
San Francisco, California, 94143 · United States
- Research SiteRecruiting
Tampa, Florida, 33612 · United States
- Research SiteRecruiting
Boston, Massachusetts, 02215 · United States
- Research SiteRecruiting
Detroit, Michigan, 48201 · United States
- Research SiteRecruiting
Rochester, Minnesota, 55905 · United States
- Research SiteRecruiting
St Louis, Missouri, 63110 · United States
- Research SiteRecruiting
New York, New York, 10016 · United States
- Research SiteRecruiting
New York, New York, 10032 · United States
- Research SiteRecruiting
New York, New York, 10065 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- Open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- primary objective
- The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
- dFLC
- The difference between the abnormal ('involved') and normal ('uninvolved') free light chain levels in the blood. Used in some conditions to decide whether disease is measurable and to track response.
- Relapsed or refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- ECOG performance status
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- CAR T
- A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
- Prior treatment
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- bispecific
- An engineered antibody that attaches to two targets at once, often linking an immune cell to a diseased cell so the immune cell can attack it.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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