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AL amyloidosis is described in the packet as a plasma cell disorder characterized by the aggregation and deposition of insoluble amyloid fibrils derived from misfolded monoclonal immunoglobulin light chains, usually produced by a plasma cell tumor. The packet lists synonyms including light-chain amyloidosis and primary systemic amyloidosis, and identifiers from Orphanet (Orphanet:85443), GARD (GARD:5797), and MeSH. The prevalence field is categorized as uncommon, 1 to 9 in 100,000 people. Two subtypes are listed, primary systemic amyloidosis and primary localized amyloidosis.
Data assembled from 5 of 12 sources · Last updated Nov 4, 2026, 4:52 AM UTC
European rare disease database
Genetic and Rare Diseases Info Center
According to the packet definition, the disease involves misfolding of monoclonal immunoglobulin light chains, usually produced by a plasma cell tumor, with deposition of the resulting amyloid fibrils. The packet lists no known genes and no inheritance patterns, so no genetic cause or inheritance pattern is described.
No diagnostic methods, GeneReviews diagnosis content, or newborn screening information are certified in this packet. How AL amyloidosis is diagnosed is therefore not described here.
The packet contains no certified FDA approval records, so FDA-approved options are unknown in this packet. Orphan-drug records list Darzalex Faspro (daratumumab and hyaluronidase-fihj, Janssen) with status approved for light-chain (AL) amyloidosis. Orphan designation, which is not approval, is recorded for birtamimab (Prothena) and for N-(3,4-dihydroxyphenyl)-3,4-dihydroxybenzamide (ProtaMed). Three further orphan designations are recorded as withdrawn. No foundational therapies or GeneReviews management content are certified. Patient assistance programs may exist for some therapies; none are certified in this packet.
70 trials found
Natural history and prognosis are not certified in this packet, so no statement about outcomes is made here.
The packet reports 60 ClinicalTrials.gov records for AL amyloidosis, all counted as active. Examples include NCT05898646, a Phase 2 daratumumab maintenance study sponsored by Mayo Clinic (recruiting; 2023-09-06 to 2031-11-30); NCT05451771, a Phase 1 venetoclax-dexamethasone study in relapsed or refractory t(11;14) amyloidosis sponsored by Columbia University (active, not recruiting; 2022-10-26 to 2027-09-01); NCT06569147, a Phase 1 elranatamab study sponsored by Dana-Farber Cancer Institute (recruiting; 2024-11-01 to 2029-07-01); and NCT05145816, a Phase 1/2a belantamab mafodotin study sponsored by the University of Texas Southwestern Medical Center (recruiting; 2024-02-15 to 2027-09-01). The packet's publication digest classifies 285 papers, with reviews and meta-analyses the dominant type. Trial statuses are as recorded in the packet and may have changed.
AI-curated news mentioning AL amyloidosis
Updated Oct 9, 2026
A recent study published in PubMed explores myocardial work in patients with light-chain and transthyretin-related amyloidosis who have preserved ejection fraction. This research enhances understanding of cardiac function in these rare diseases.
A North Texas man with AL Amyloidosis achieved complete remission after participating in a clinical trial for CAR-T cell therapy, NX 201. The trial reported a striking 95% remission rate among participants, highlighting the potential of this innovative treatment for a rare and life-threatening condition.
A clinical trial of CAR-T cell therapy NXC-201 shows promising results for patients with AL Amyloidosis, with approximately 95% achieving complete remission. This innovative treatment targets the accumulation of abnormal proteins that damage vital organs.
A clinical trial of CAR-T cell therapy, NX 201, shows promising results for patients with AL amyloidosis, with about 95% achieving complete remission. This experimental treatment could significantly impact the management of this rare and life-threatening disease.
A recent study highlights raccoon eye as a unique presenting sign of AL amyloidosis linked to a low-burden clonal plasma cell neoplasm. This finding may enhance diagnostic awareness for clinicians encountering similar symptoms.