Promoting Diagnosis and Management of AL in Italy (ProDigALIty)

NCT06383143 | Recruiting

• 1 other identifier: AC-020-IT
• Study Type: observational
• Target: 760
• Locations: 1 country
• Sites: 4

Brief Summary

The investigators plan to establish a dedicated network of Italian Hematologic Departments interconnected with the Amyloidosis Research and Treatment Center in Pavia to:

1. 1.Implement a biomarker-based screening strategy to promote early diagnosis of AL amyloidosis among at-risk patients, including patients with monoclonal gammopathy of undetermined significance, MGUS, and altered free light chain ratio (aFLCR), and patients with smoldering multiple myeloma (SMM)
2. 2.Expedite and facilitate patients' referral and their enrollment in ongoing pre-clinical/clinical studies, also to reflect a broader spectrum of the real-world population of patients with AL amyloidosis in Italy;
3. 3.Investigate the clinical utility of novel diagnostic technologies, including light chain sequencing and N-glycosylation analysis

Conditions

AL Amyloidosis; Smoldering Multiple Myeloma; Monoclonal Gammopathy of Undetermined Significance

Outcome Measures

Primary Outcomes (3)

• Assess proportion of patients with newly diagnosed AL identified through the biomarker-based screening of at-risk patient

  Part A of the study: the proportion of patients with newly diagnosed AL through the biomarker-based screening of at-risk patients with a known monoclonal gammopathy will be identified.

  2 years

• Assess the proportion of patients with deep haematological response after frontline therapy (best response evaluation) in the new enhanced, contemporary, real-world series of AL patients enrolled during the study.

  Part B of the study: The proportion of patients with deep hematological response after frontline therapy will be assessed

  2 years

• 3. Identify associations of clonal light chain features with different clinical features at baseline.

  Part C of the study: Associations of clonal light chain features with different clinical features at baseline, including organ tropism and clonal burden, will be assessed.

  2 years

Secondary Outcomes (6)

• Description of the baseline characteristics and 6-months outcome, as well as the time to AL development for patients with AL identified through the biomarker-based screening

  6 months

• Description of the baseline characteristics of MGUS/SMM patients with abnormal FLCR

  2 years

• Identification of clinical and biological correlates of hematological response

  2 years

• To verify whether implementing a dedicated pipeline for referral of AL patients to the National Referral Center will increase the proportion of patients from spoke centers

  2 years

• Identification of associations of clonal light chain features with event-free survival

  2 years

Study Arms (1)

Patients with MGUS and aFLCR and with SMM

Part A: Patients with MGUS and aFLCR and patients with SMM undergoing an active, biomarker-based screening of presymptomatic amyloid organ involvement. Part B: Newly diagnosed patients with AL amyloidosis identified through Part A or with clinically overt AL amyloidosis evaluated in the frame of routine clinical assessments and referred to the Amyloidosis Research and Treatment Center in Pavia or managed locally at the participating Italian Hematologic Departments

Other: no intervention

Interventions

no intervention OTHER

no intervention

Patients with MGUS and aFLCR and with SMM

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The participating Departments of Hematology will be involved in screening at-risk patients with monoclonal gammopathies and refer suspected/confirmed AL cases to the Amyloidosis Center in Pavia.

You may qualify if:

• diagnosis of MGUS with altered FLCR or SMM;
• treatment-naïve;
• age ≥18 years;
• ability to understand and willingness to sign an informed consent;
• planned follow-up at participating center.

You may not qualify if:

• Diagnosis of symptomatic monoclonal gammopathies;
• Previous treatment for monoclonal gammopathies.
• PART B
• diagnosis of systemic AL amyloidosis;
• treatment-naïve;
• age ≥18 years;
• ability to understand and willingness to sign an informed consent;
• planned follow-up at participating center.
• non-AL amyloidosis;
• previous treatment for AL amyloidosis.

Sponsors & Collaborators

• Fondazione IRCCS Policlinico San Matteo di Pavia: lead

Study Sites (4)

Azienda Ospedaliera Policlinico Consorziale

Bari, Italy

RECRUITING

Azienda Ospedaliero Universitaria Policlinico G.Rodolico - San Marco

Catania, Italy

RECRUITING

Fondazione Irccs Policlinico San Matteo

Pavia, Italy

RECRUITING

EMATOLOGIA - Città della Scienza e Salute - Torino

Torino, Italy

RECRUITING

MeSH Terms

Conditions

Immunoglobulin Light-chain Amyloidosis; Smoldering Multiple Myeloma; Monoclonal Gammopathy of Undetermined Significance

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Paraproteinemias; Precancerous Conditions; Hypergammaglobulinemia; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD

Study Record Dates

• First Submitted: April 19, 2024
• First Posted: April 25, 2024
• Study Start: May 1, 2023
• Primary Completion: May 1, 2026
• Study Completion: May 1, 2026
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

IT (4)