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 CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis.
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.
Miscellaneous
Phase I/II
Adults
Chemotherapy - cytotoxic,
Mol. targeted/Immunotherapy/Biologics
AZD0120,
Cyclophosphamide,
Fludarabine
Baljevic, Muhamed
National
Vanderbilt University
03-17-2026
Eligibility
18 Years and older
ALL
false
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 disease or refractory disease defined as a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy.
ECOG performance status of 0 to 1
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 pregnancy test prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use 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)
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 disease or refractory disease defined as a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy.
ECOG performance status of 0 to 1
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 pregnancy test prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use 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)
