Cardiovascular

Light Chain Amyloidosis

Also known as AL amyloidosis, primary amyloidosis, immunoglobulin light-chain amyloidosis

Light Chain Amyloidosis is caused by a clonal population of plasma cells in the bone marrow producing misfolded immunoglobulin light chains that aggregate into amyloid fibrils and deposit in vital organs, most critically the heart and kidne

ORPHA:85443 ↗Prevalence Approximately 8–12 per million per yearOnset Adulthood; median diagnosis in the sixth decadeAcquired (clonal plasma cell disorder)

54

studies recruiting now

as of 7 Sept 2026

295

studies registered in total

as of 7 Sept 2026

11

countries with a recruiting site

as of 7 Sept 2026

10 Jun 2026

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 54 recruiting studies

Where recruiting studies are running

Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.

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Patient organisations

Amyloidosis Research ConsortiumPatient association
Visit website ↗

Registry: Amyloidosis Foundation Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.

About Light Chain Amyloidosis

Light Chain Amyloidosis is caused by a clonal population of plasma cells in the bone marrow producing misfolded immunoglobulin light chains that aggregate into amyloid fibrils and deposit in vital organs, most critically the heart and kidneys. Unlike hereditary amyloidosis, AL is not inherited but arises de novo and is closely related to multiple myeloma, requiring haematological treatment directed at the underlying plasma cell clone. Cardiac involvement, present in up to 70% of patients at diagnosis, is the primary determinant of prognosis and drives treatment urgency; the introduction of daratumumab-based combination regimens has markedly improved haematological response rates and cardiac outcomes.

Common clinical features

Heart failure with preserved or reduced ejection fractionPeriorbital purpura (raccoon eyes) pathognomonic but uncommonMacroglossiaNephrotic-range proteinuria and progressive renal impairmentPeripheral neuropathy and carpal tunnel syndromeOrthostatic hypotension due to autonomic neuropathyFatigue, weight loss, and early satietyElevated NT-proBNP disproportionate to degree of overt heart failure

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

2 approved treatments and 21 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Tafamidis (Vyndamax)Approved: Tafamidis Meglumine (Vyndaqel)
Phase 3Cyclophosphamide (Cyclophosphamide)
Phase 3Dexamethasone (Aeroseb-dex)
Phase 3Lenalidomide (Lenalidomide accord)
Phase 3Daratumumab (Daratumumab component of darzalex faspro)
Phase 3Anselamimab
Phase 3Birtamimab
Phase 2/3Doxycycline (Doxirobe)
Phase 2Amiodarone Hydrochloride (Amidox)

+ 13 more in development

Before you apply

Things trial teams commonly ask about for Light Chain Amyloidosis. Not eligibility rules; those are set by each study.

  • Confirmation of AL amyloidosis requires both demonstration of amyloid deposits (Congo red biopsy) and proof of a clonal plasma cell disorder (serum/urine protein electrophoresis, free light chain assay, bone marrow biopsy); all results are required for trial screening.
  • Cardiac staging (Mayo 2004 or 2012 criteria using NT-proBNP and troponin) is used to stratify eligibility; Stage IIIb/IV patients may be excluded from some trials due to safety concerns.
  • Prior haematological treatment lines and response status affect eligibility for relapsed/refractory trials; a detailed treatment history including cycle counts and best responses is essential.

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).