Actively Recruiting
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.
Led by Alexion Pharmaceuticals, Inc. · Updated on 2026-03-24
91
Participants Needed
18
Research Sites
78 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying AZD0120, a CAR T cell therapy targeting CD19 and BCMA, in people with relapsed or refractory light chain (AL) amyloidosis. This phase 1b/2 open-label study aims to evaluate the safety, tolerability, and effectiveness of AZD0120 in this patient group. The trial is sponsored by Alexion Pharmaceuticals, Inc. and focuses on participants who have measurable hematologic disease and prior therapy experience. Participants will receive a weight-based dose of AZD0120 through an intravenous infusion. The study includes both phase 1b and phase 2 periods. During these phases, researchers will monitor treatment-emergent adverse events and assess the proportion of participants achieving a complete response or hematologic response. The treatment is given once, and participants will be closely observed throughout the study. Throughout the study, participants will have regular visits for safety assessments, laboratory tests, and blood level monitoring of AZD0120. The primary outcomes include tracking adverse events in phase 1b and measuring complete response rates in phase 2, both over at least six months. Secondary outcomes include monitoring AZD0120 levels in the blood and hematologic response rates. Participants will be monitored closely for a minimum of six months following treatment to evaluate treatment effects and safety.
CONDITIONS
Brief Title
A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 needing additional therapy after at least 1 line of anti-plasma cell-directed therapy
- ECOG performance status of 0 to 1
- Able and willing to follow the study visit schedule and protocol requirements
- Women of child-bearing potential must have a negative pregnancy test before treatment
- All sexually active women of child-bearing potential and male participants must agree to use effective birth control during the study
You will not qualify if you...
- Any form of amyloidosis other than AL amyloidosis
- Mayo Stage IIIb AL amyloidosis
- Oxygen saturation less than 95% on room air
- Systolic blood pressure less than 100 mmHg
- New York Heart Association (NYHA) class III or IV heart failure
- Extensive gastrointestinal involvement with active bleeding risk
- Prior CAR T cell therapy targeting any antigen
- Prior BCMA-targeting therapy
- Prior treatment with any FDA approved or investigational T cell engaging therapies within the last 6 months
- Toxicity from previous anti-cancer or anti-plasma cell therapy not resolved to baseline or Grade 1 or less, except alopecia or peripheral neuropathy
- Active plasma cell leukemia at screening
- Symptomatic multiple myeloma defined by clonal bone marrow plasma cells ≥10% plus myeloma-defining events per IMWG 2014 criteria
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Through study completion, a minimum of 6 months
Participants receive AZD0120 via intravenous infusion as part of the treatment for relapsed or refractory AL amyloidosis.
Visit schedule as per treatment protocol
Trial Site Locations
Total: 18 locations
1
Research Site
Phoenix, Arizona, United States, 85028
Actively Recruiting
2
Research Site
San Francisco, California, United States, 94143
Not Yet Recruiting
3
Research Site
Tampa, Florida, United States, 33612
Actively Recruiting
4
Research Site
Boston, Massachusetts, United States, 02215
Actively Recruiting
5
Research Site
Detroit, Michigan, United States, 48201
Actively Recruiting
6
Research Site
Rochester, Minnesota, United States, 55905
Actively Recruiting
7
Research Site
St Louis, Missouri, United States, 63110
Actively Recruiting
8
Research Site
New York, New York, United States, 10016
Actively Recruiting
9
Research Site
New York, New York, United States, 10032
Actively Recruiting
10
Research Site
New York, New York, United States, 10065
Actively Recruiting
11
Research Site
Cleveland, Ohio, United States, 44195
Actively Recruiting
12
Research Site
Nashville, Tennessee, United States, 37232
Actively Recruiting
13
Research Site
Calgary, Alberta, Canada, T2N 5G2
Withdrawn
14
Research Site
Calgary, Alberta, Canada, T2N 5G2
Not Yet Recruiting
15
Research Site
Toronto, Ontario, Canada, M5G 2M9
Withdrawn
16
Research Site
Toronto, Ontario, Canada, M5G 2M9
Not Yet Recruiting
17
Research Site
London, United Kingdom, NW3 2PF
Withdrawn
18
Research Site
London, United Kingdom, NW3 2PF
Not Yet Recruiting
Research Team
A
Alexion Pharmaceuticals, Inc. (Sponsor)
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here