How the IGLV1-44 light chain causes AL amyloidosis and POEMS
One Gene, Two Diseases: the Pathologic Role of IGLV1-44 in AL Amyloidosis and POEMS
This project tests whether specific IGLV1-44 light‑chain sequences drive AL amyloidosis and POEMS and whether nanobodies can recognize and stabilize those light chains in adults with AL amyloidosis, POEMS, or multiple myeloma.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 100 (estimated) |
| Ages | 18 Years to 99 Years |
| Sex | All |
| Sponsor | Fondazione IRCCS Policlinico San Matteo di Pavia Academic / other |
| Locations | 1 site (Pavia) |
| Trial ID | NCT07448935 on ClinicalTrials.gov |
What this trial studies
This observational project collects blood (and optional bone marrow) from adults with biopsy‑proven AL amyloidosis, POEMS syndrome, or multiple myeloma to perform detailed sequence analysis and biophysical characterization of IGLV1-44 light chains. The team aims to identify sequence 'fingerprints' that distinguish light chains that cause cardiac proteotoxicity and POEMS manifestations. Findings are intended to improve risk stratification and earlier diagnosis for patients expressing pathogenic IGLV1-44 variants. Parallel laboratory work will develop and test nanobodies that recognize and stabilize pathogenic IGLV1-44 light chains as a possible starting point for future therapies.
Who should consider this trial
Good fit: Adults (≥18 years) with biopsy‑proven systemic AL amyloidosis, POEMS syndrome, or multiple myeloma who agree to blood sampling and optional bone marrow aspiration are eligible.
Not a fit: Patients with non‑AL amyloidosis, undefined monoclonal gammopathy, or those already in complete hematologic response are unlikely to receive benefit from this project.
Why it matters
Potential benefit: If successful, this work could enable earlier diagnosis and better risk prediction and provide a foundation for nanobody-based therapies that neutralize toxic IGLV1-44 light chains.
How similar studies have performed: Previous studies have linked IGLV1-44 sequence patterns to AL amyloidosis and POEMS, but using nanobodies to stabilize these light chains is largely preclinical and remains novel.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Pavia
- Fondazione IRCCS Policlinico San Matteo — Pavia, Italy (Recruiting)
Study contacts
- Study coordinator: Mario Nuvolone, MD
- Email: m.nuvolone@smatteo.pv.it
- Phone: 0382502935
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.