One Gene, Two Diseases: the Pathologic Role of IGLV1-44 in AL Amyloidosis and POEMS
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: AL Amyloidosis, POEMS Syndrome, Multiple Myeloma. Basic parameters: 18 years — 99 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
By detailed sequence analysis and subsequent biophysical characterization of prototypic light chains, this project aims to identify sequence fingerprints in IGLV1-44 light chains leading to AL amyloidosis and POEMS syndrome. This understanding might help improve the risk stratification and early diagnosis of patients overexpressing pathologic IGLV1-44 LCs. Moreover, the development of nanobodies efficient in recognizing and stabilizing IGLV1-44 light chains which exert direct toxicity in cardiac AL amyloidosis and POEMS syndrome might form the basis for future development of therapeutic agents capable of counteracting IGLV1-44 light chain proteotoxicity.
Primary outcome measures
- identify sequence fingerprints in IGLV1-44 light chains leading to AL amyloidosis and POEMS syndrome [Time frame: two years]
Eligibility criteria
Inclusion criteria
- Biopsy-proven diagnosis of systemic AL amyloidosis, POEMS syndrome or multiple myeloma
- 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.
Exclusion criteria
- Undefined monoclonal gammopathy or non-AL amyloidosis
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Italy · 1 center
- Fondazione IRCCS Policlinico San Matteo — Pavia
Identifiers
NCT: NCT07448935 · ALPS