Investigating the Pathogenic Role of N-glycosylation in AL Amyloidosis: Molecular Bases, Diagnosis, and Treatment
Recruiting now
Conditions studied: AL Amyloidosis, MGUS, Multiple Myeloma, Monoclonal Gammopathies
In brief
Immunoglobulin light chain (AL) amyloidosis is caused by a typically small, minimally proliferating bone marrow plasma cell clone secreting a patient-unique, unstable, aggregation-prone, toxic light chain (LC). The amyloidogenicity of LCs is encrypted in their sequence, yet molecular determinants of LC pathogenicity remain obscure. N-glycosylation has been long suspected to be a determinant of LC amyloidogenicity based on anecdotal reports of individual AL patients with a clonal LC displaying this post-translational modification. It is hypothesized that N-glycosylation fundamentally contributes to determining the amyloidogenicity of immunoglobulin LCs in a subset of patients with AL and might influence its clinical phenotype. It is further proposed that the synthesis and secretion of unstable LCs that also have to be N-glycosylated might reverberate on the biology of the plasma cell clone, possibly modulating the sensitivity toward different drugs and might represent itself a therapeutic target. The objective of our study is now to elucidate the molecular role of LC N-glycosylation in AL amyloidosis, exploit it for risk assessment, and define its potential impact on the biology of the underlying plasma cell clone and its drug sensitivity.
Key facts
- Study ID
- NCT07448779
- Run by
- Fondazione IRCCS Policlinico San Matteo di Pavia
- People needed
- 100
- Starts
- 2025-11-17
- Expected to finish
- 2027-05-30
- Last updated by the study team
- 2026-03-04
Who can join
Age: 18 and older, up to 99. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Diagnosis of monoclonal gammopathy (e.g. AL amyloidosis, MGUS, MM, others)
- Planned peripheral blood sampling +/- bone marrow aspiration
- Age > 18 years
- Willingness to allow use of clinical data and diagnostic leftovers of clinical specimens for research purposes through signing a written informed consent.
You may not qualify if…
- Lack of monoclonal gammopathy
- Patients fulfilling the criteria for complete hematologic response after anti-clonal therapy
- Age <18 years
- Failure to show willingness to allow use of clinical data and diagnostic leftovers of clinical specimens for research purposes.
Where it is running
- Fondazione IRCCS Policlinico San Matteo di Pavia — Pavia, PV, Italy (enrolling)
Full record on ClinicalTrials.gov
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