Key Highlights
- New potential option for AL Amyloidosis
- Dual-targeting (BCMA + CD19)
Designed for patients who have received just 1 prior line, who are experiencing rising light chains or M spike.
Next Generation CAR-T Therapy Clinical Trial #23773
Refer eligible patients for a Phase 1b/2 study evaluating AZD0120 (BCMA/CD19 CAR-T) with early signals of deep response and favorable safety in RRMM.
A Potential Breakthrough Treatment for AL Amyloidosis Patients
Patients with AL Amyloidosis who have rising light chains or M spike could be eligible. AZD0120 is designed to:
- Target two antigens (BCMA + CD19) to reduce increase efficacy
- Deliver rapid and deep responses
- Maintain a manageable safety profile
Early Clinical Results for Multiple Myeloma Show Promise
The results from Phase 1b in RRMM indicate this could provide a new option for treating AL Amyloidosis. Highlights from the RRMM study below or read the published study.
New Option for AL Amyloidosis
- CAR-T has shown to be effective in RRMM
- Few options for relapsing AL Amlyloidosis
Efficacy in RRMM
- ORR: 100% (n=15 evaluable)
- 33% sCR / 47% VGPR
- 100% MRD negativity (evaluable pts)
- Median time to response: about 1 month
Safety in RRMM
- No Grade ≥3 CRS
- No ICANS observed
- CRS mostly Grade 1–2, short duration (about 2 days)
Note: Data is early phase with limited follow-up. ORR (Overall Response Rate), sCR (Stringent Complete Response), VGPR (Very Good Partial Response), MRD negativity (Minimal or Measurable Residual Disease negativity), CRS (Cytokine Release Syndrome), ICANS (Immune Effector Cell-Associated Neurotoxicity Syndrome),
Which Patients May Be Eligible?
Inclusion Criteria Summary:
- Confirmed histopathological AL Amyloidosis
- Measureable disease
- dFLC > 20 mg/L, or
- Serum M-protein > 5 g/L
- One or more organs affected
- 1 prior anti-plasma cell therapy
- ECOG 0–1
Exclusion Criteria Summary:
- Significant cardiac disease
- Extensive GI involvement
- Recent stem cell transplant
Not sure if your patient qualifies? Contact a Clinical Research Coordinator for rapid eligibility review: Natalie.Abele@moffitt.org
What does trial participation look like for patients?
- Submit referral request and send patient records to Moffitt
- Patients will be pre-screened for basic eligibility by the clinical research coordinator
- Initial appointment with Dr. Blue
- Workup and clinical screening appointments
- Following enrollment in the trial, patient is scheduled for apheresis and chemotherapy
- Apheresis (cell collection) is a half-day appointment at Magnolia Campus
- Collected cells are sent for manufacturing, patients will remain at home during cell manufacturing process.
- Patient receives infusion of cells followed by a 12 day inpatient stay at Magnolia Campus
The patient and their caregiver will need to remain within 1 hour of Moffitt's Magnolia Campus for about 28 days, for careful monitoring and multiple visits. The trial sponsor will reimburse travel/lodging expenses and our team can support with finding accommodations for patients who live further away.
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Expenses Reimbursed
Upon enrollment, travel and lodging expenses are covered by the trial sponsor through reimbursement.
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Follow-up Care
2 years of active follow-up, then visits every 6 months until year 15 post therapy.
Partner with Moffitt
A referral for a clinical trial means we will work together to give your patient the best chance at beating their cancer.
This trial represents a promising new treatment option for patients with AL Amyloidosis. We are encouraged by the results seen in multiple myeloma and hope to bring more treatment options for patients with this rare disease.
Brandon Blue, MD
Principal Investigator
Have a patient you think would be a candidate for this clinical trial?
We are here to help, you can start a referral by submitting this online form.
If you have questions for the Research Coordinator about the trial, please contact Natalie.Abele@moffitt.org.
For questions about referring your patient or to learn more about Moffitt you can speak with a Physician Liaison:
