A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis

Launched by ALEXION PHARMACEUTICALS, INC. · Jul 15, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new type of treatment called CAR T cell therapy for people with a condition called AL amyloidosis, which happens when abnormal proteins build up in organs and cause damage. This treatment is designed to target specific proteins (CD19 and BCMA) on the cells causing the disease. The main goal is to see if this treatment is safe, well-tolerated, and effective for people whose AL amyloidosis has returned after previous treatments or who didn’t respond to earlier therapies.

People who might be eligible to join this study are adults with a confirmed diagnosis of AL amyloidosis affecting one or more organs, and whose disease has either come back or not improved after at least one prior treatment. They need to be in fairly good overall health and able to follow the study schedule. The study is not yet open for recruiting, and certain conditions like more severe heart or lung problems, other types of amyloidosis, or recent similar treatments would exclude someone from joining. Participants can expect careful monitoring to check how they respond to the therapy and to watch for any side effects. This trial offers a potential new option for those with limited treatment choices, but it’s important to discuss all risks and benefits thoroughly with the study team.

Gender

ALL

Eligibility criteria

• 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)