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

NCT04659798 | Completed

• 2 other identifiers: C16054U1111-1258-6881
• Study Type: observational
• Target: 240
• Locations: 1 country
• Sites: 14

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.

Conditions

Multiple Myeloma; Immunoglobulin Light-chain Amyloidosis

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (2)

• DoT

  DoT is defined as the time between the start of a treatment/regimen and the end of that regimen.

  From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)

• PFS

  PFS: time between start of treatment or regimen and progression or death. PFS for MM will be evaluated according to International Myeloma Working Group(IMWG) and defined per European Society for Medical Oncology(ESMO)guideline. Progressive disease(PD)for MM: increase of 25% from lowest confirmed response value in one of the following criteria: Serum M protein(absolute increase must be\>=0.5 g/dL; Serum M protein increases \>=1g/dL, if the lowest M component was 5g/dL; Urine M protein(absolute increase must be\>=200 mg/24h).PFS for AL amyloidosis will be evaluated as per Guidelines of the British Society for Haematology(BSH).PD for AL amyloidosis: progression from complete response(CR)as any detectable M protein or abnormal free light chain(FLC) ratio(involved light chain must double).Progression from partial response(PR)is defined as a 50% increase in serum M protein to\>5.0g/L or 50% increase in urine M protein to\>200mg/dL(a visible peak must be present)or FLC increase of 50% to\>100mg/L.

  From date of the initiation of treatment or regimen the participant is receiving at the time of study entry until progression, death, or 1 year after end of treatment (approximately 1 year)

Secondary Outcomes (8)

• Time to Next Treatment (TTNT)

  From start of current treatment or regimen until start of next treatment or regimen (approximately 1 year)

• Number of Participants Categorized Based on Treatment Regimens Prescribed in Routine Clinical Practice

  From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)

• Number of Participants Based on Treatment Response Achieved

  From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)

• Number of Participants Categorized Based on Differences Between Prescribed and Actual DoT, and Reasons for Treatment Discontinuation

  From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)

• Number of Participants Satisfied With the Treatment

  From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)

Study Arms (2)

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).

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.

Eligibility Criteria

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

Study Population

Participants diagnosed with MM or systemic AL amyloidosis.

You may qualify if:

• 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:
• 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%.
• 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:
• 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).
• 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).

You may not qualify if:

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

Sponsors & Collaborators

• Takeda: lead

Study Sites (14)

H. Puerta del Mar

Cadiz, Andalusia, 11009, Spain

Location

Hospital Regional Universitario de Malaga (Carlos de Haya)

Málaga, Andalusia, 29010, Spain

Location

H. Universitari Son Espases

Palma de Mallorca, Balearic Islands, 7120, Spain

Location

H. Universitario de Araba

Vitoria-Gasteiz, Basque Country, 1009, Spain

Location

C.H.U. Canarias

San Cristóbal de La Laguna, Canary Islands, 38320, Spain

Location

H. Universitario Marques de Valdecilla

Santander, Cantabria, 39008, Spain

Location

H. Universitario de Leon

León, Castille and León, 24071, Spain

Location

C.H. Salamanca

Salamanca, Castille and León, 37007, Spain

Location

H. Universitario Lucus Agusti

Lugo, Galicia, 27003, Spain

Location

C.H.U. Santiago

Santiago de Compostela, Galicia, 15706, Spain

Location

Hospital General Universitario Santa Lucia

Cartagena, Murcia, 30202, Spain

Location

H. Universitario de Cabuenes

Gijón, Principality of Asturias, 33394, Spain

Location

Hospital Universitario de Burgos

Burgos, 9006, Spain

Location

H. Universitario Fundacion Jimenez Diaz

Madrid, 28040, Spain

Location

Related Links

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

MeSH Terms

Conditions

Multiple Myeloma; Immunoglobulin Light-chain Amyloidosis

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Medical Director

  Takeda

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 2, 2020
• First Posted: December 9, 2020
• Study Start: January 22, 2021
• Primary Completion: April 24, 2023
• Study Completion: September 5, 2023
• Last Updated: July 8, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

ES (14)