A Study to Investigate the Relationship Between Duration of Treatment and Response in Patients With Multiple Myeloma (MM) or Systemic AL Amyloidosis Treated in Real-life Practice

NCT ID: NCT04659798

Last Updated: 2024-07-08

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 240 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2021-01-22
• Study Completion Date: 2023-09-05

Brief Summary

The study will provide information on outcomes in people with multiple myeloma, or systemic AL amyloidosis, or both, under standard care. AL is short for amyloid light-chain. Standard care means the participant will be treated according to their clinic's standard practice. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study.

The aim of the study is to learn if treatment duration makes a difference in how participants with multiple myeloma or systemic AL amyloidosis respond to their treatment.

During the study, participants will be treated according to their clinic's standard practice. Participants must have started their treatment up to 12 months before taking part in this study. During the study, the participants will visit their clinic every 3 months. These are extra visits to their clinic's standard visits.

Detailed Description

This is a prospective non-interventional study to assess the DoT and response in participants with MM or systemic AL amyloidosis in standard clinical practice.

This study will enroll approximately 250 participants (220 with MM and 30 with systemic AL amyloidosis). Participants will be enrolled in 2 groups:

Participants with MM Participants with AL amyloidosis The study will have a prospective data collection of the participants from clinical records and scheduled visits following the routine clinical practice. All participants will receive treatment at study start and this treatment must have been started within 12 months before the participant's enrollment.

This multi-center study will be conducted in Spain. Participants will be followed until 12 months after enrollment. The overall time to participate in this study is approximately 1 year.

Conditions

Multiple Myeloma; Immunoglobulin Light-chain Amyloidosis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Participants with MM

Participants diagnosed with MM who have received treatment within 12 months preceding the enrollment will be observed prospectively. Data will be collected from the participants medical charts and via electronic case report forms (eCRFs).

No interventions assigned to this group

Participants with AL Amyloidosis

Participants diagnosed with AL Amyloidosis who have received treatment within 12 months preceding the enrollment will be observed prospectively. Data will be collected from the participants medical charts and via eCRFs.

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

1. Diagnosis of MM and/or AL amyloidosis according to the IMWG for MM and BSH guidelines for AL amyloidosis.

MM diagnostic criteria:

• Smouldering MM- Both criteria must be met:

1. Serum M protein (Immunoglobulin G [IgG] or IgA greater than or equal to (>=) 30 gram per liter (g/L) or urinary monoclonal protein (M protein) >= 500 milligram per 24 hours (mg/24 h) and/or clonal bone marrow (BM) plasma cells (PCs) 1 percent (%) - 60%.

2. Absence of myeloma-defining events or amyloidosis.

• Multiple myeloma- Clonal BM plasma cells >= 10% or biopsy-proven bony or extramedullary plasmacytoma and any one or more of the following myeloma-defining events:

1. Evidence of end organ damage that can be attributed to the underlying plasma cell proliferative disorder, specifically:

• Hypercalcemia: serum calcium greater than (>) 0.25 millimole per liter (mmol/L) (> 1 milligram per deciliter [mg/dL]) higher than the upper limit of normal or >2.75 mmol/L (>11 mg/dL).
• Renal insufficiency: creatinine clearance (CrCl) less than (<) 40 milliliter per minute (mL/min) or serum creatinine >177 micromole/liter (mcmol/L) (>2 mg/dL).
• Anemia: haemoglobin (Hb) value of >20 g/L below the lower limit of normal or a Hb value of <100 g/L.
• Bone lesions: one or more osteolytic lesions on skeletal radiography, computed tomography (CT) or positron emission tomography-computed tomography (PET-CT).

2. Any one or more of the following biomarkers of malignancy:

• >=60% clonal BM plasma cells.
• Involved/uninvolved serum-free light chain ratio >=100.
• >1 focal lesions on magnetic resonance imaging (MRI) studies (each focal lesion must be >=5 millimeter (mm) in size).

AL amyloidosis diagnosis:

• In suspected AL amyloidosis, a histological diagnosis is essential and, where possible, a biopsy should be taken from an apparently affected organ. Alternatively, a subcutaneous abdominal fat aspirate and bone marrow biopsy may be examined for amyloid.
• Congo red staining with classical apple green birefringence under polarized light should be used to test for the presence of amyloid on any histological specimen.
• The diagnosis of amyloid requires an experienced laboratory as false negative and false positive diagnoses on the basis of histology are not infrequent. Other (non-AL) amyloid fibril types should be excluded by using immunohistochemistry, deoxyribonucleic acid (DNA) analysis, protein sequencing or mass spectrometry.

2. Under treatment for MM or systemic AL amyloidosis at the time of study entry.

3. Have started treatment up to 12 months before inclusion for MM or systemic AL amyloidosis, irrespective of the treatment regimen.

4. Having first, second, third or fourth line of treatment for MM or systemic AL amyloidosis, irrespective of the treatment regimen.

5. In case of participants with Newly Diagnosed Multiple Myeloma (NDMM) who are candidates to autologous stem cell transplant (ASCT), the ASCT has to be performed before study entry.

Exclusion Criteria

1. Participants with planned cessation of treatment for MM or systemic AL amyloidosis from participation to the study (example, due to pregnancy).

2. Participating in blinded clinical trials, or in clinical trials with no possibility of obtaining information required in this study, or in clinical trials in which participation in other studies is not allowed.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Takeda

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Director

Role: STUDY_DIRECTOR

Takeda

Locations

H. Puerta del Mar

Cadiz, Andalusia, Spain

Hospital Regional Universitario de Malaga (Carlos de Haya)

Málaga, Andalusia, Spain

H. Universitari Son Espases

Palma de Mallorca, Balearic Islands, Spain

H. Universitario de Araba

Vitoria-Gasteiz, Basque Country, Spain

C.H.U. Canarias

San Cristóbal de La Laguna, Canary Islands, Spain

H. Universitario Marques de Valdecilla

Santander, Cantabria, Spain

H. Universitario de Leon

León, Castille and León, Spain

C.H. Salamanca

Salamanca, Castille and León, Spain

H. Universitario Lucus Agusti

Lugo, Galicia, Spain

C.H.U. Santiago

Santiago de Compostela, Galicia, Spain

Hospital General Universitario Santa Lucia

Cartagena, Murcia, Spain

H. Universitario de Cabuenes

Gijón, Principality of Asturias, Spain

Hospital Universitario de Burgos

Burgos, , Spain

H. Universitario Fundacion Jimenez Diaz

Madrid, , Spain

Countries

Spain

Related Links

https://clinicaltrials.takeda.com/study-detail/5ffeb154565ce300294c6ac5

To obtain more information on the study, click here/on this link

Other Identifiers

U1111-1258-6881

Identifier Type: REGISTRY

Identifier Source: secondary_id

C16054

Identifier Type: -

Identifier Source: org_study_id