Phase 1b/2 study of AZD0120 (CAR T-cell therapy targeting CD19 and BCMA) in AL amyloidosis

• Research type

  Research Study

• Full title

  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.

• IRAS ID

  1012635

• Contact name

  Richa Manwani

• Contact email

  richa.manwani@alexion.com

• Sponsor organisation

  Alexion Pharmaceuticals, Inc.

• Clinicaltrials.gov Identifier

  NCT07081646

• Research summary

  This study aims to learn more about whether an experimental therapy called AZD0120 is safe and effective in patients with relapsed or refractory AL amyloidosis. AL amyloidosis occurs when plasma cells in the bone marrow (which normally make antibodies that fight infections) become defective and make abnormal light chain proteins, forming a protein called ‘amyloid’. Amyloid can deposit in different organs, such as the heart, kidneys and liver, causing damage and affecting their function. The study is open to patients whose disease has returned (relapsed) or does not respond to treatment (refractory).
  AZD0120 is a type of treatment called Chimeric Antigen Receptor or CAR T-cell therapy. CAR T-cell therapy uses the participant’s own immune cells which are genetically modified to attack plasma cells that make amyloid. T- cells are collected from the patient’s blood (through a process called leukapheresis), the CAR gene is put into the T cells so they can find and attack abnormal plasma cells that make amyloid. Patients receive two chemotherapy drugs (called lymphodepletion) which make space for the CAR T-cells. The CAR T-cells are then given into a vein and patients are monitored closely in the hospital for 10 days, with daily reviews and regular blood tests. Patients will continue to be monitored by their team after discharge for any side-effects and to assess how their disease is responding (through blood, urine and bone marrow tests). Patients who receive CAR-T are monitored for a long period of time (for 15 years after CAR-T) to check their response and monitor for any side-effects.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  25/LO/0756

• Date of REC Opinion

  16 Dec 2025

• REC opinion

  Further Information Favourable Opinion