Clinical trial NCT04659798: 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
Official title: Prospective Non-interventional Study to Investigate the Relationship Between Duration of Treatment (DoT) and Response in Patients With Multiple Myeloma or Systemic AL Amyloidosis Treated in Real-life Clinical Practice
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Multiple Myeloma
Immunoglobulin Light-chain Amyloidosis
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2020-12-09
Enrollment: 240
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants diagnosed with MM or systemic AL amyloidosis.

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.

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.

Primary outcomes:
measure: DoT
timeFrame: From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)
measure: PFS
timeFrame: 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:
measure: Time to Next Treatment (TTNT)
timeFrame: From start of current treatment or regimen until start of next treatment or regimen (approximately 1 year)
measure: Number of Participants Categorized Based on Treatment Regimens Prescribed in Routine Clinical Practice
timeFrame: From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)
measure: Number of Participants Based on Treatment Response Achieved
timeFrame: From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)
measure: Number of Participants Categorized Based on Differences Between Prescribed and Actual DoT, and Reasons for Treatment Discontinuation
timeFrame: From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)
measure: Number of Participants Satisfied With the Treatment
timeFrame: From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)
measure: Number of Participants Categorized Based on Clinical and Sociodemographic Characteristics Influencing the Treatment Prescribed and Satisfaction With Treatment
timeFrame: From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)
measure: Percentage of Participants With More Than one Neoplasm
timeFrame: From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)
measure: Percentage of Participants who Achieve the Prescribed DoT According to Treatment Strategy
timeFrame: From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)
Other outcomes:
Locations:
city: Cadiz
country: Spain
facility: H. Puerta del Mar
state: Andalucia
city: Malaga
country: Spain
facility: Hospital Regional Universitario de Malaga (Carlos de Haya)
state: Andalucia
city: Gijon
country: Spain
facility: H. Universitario de Cabuenes
state: Asturias
city: La Laguna
country: Spain
facility: C.H.U. Canarias
state: Canarias
city: Santander
country: Spain
facility: H. Universitario Marques de Valdecilla
state: Cantabria
city: Leon
country: Spain
facility: H. Universitario de Leon
state: Castilla Y Leon
city: Salamanca
country: Spain
facility: C.H. Salamanca
state: Castilla Y Leon
city: Lugo
country: Spain
facility: H. Universitario Lucus Agusti
state: Galicia
city: Santiago de Compostela
country: Spain
facility: C.H.U. Santiago
state: Galicia
city: Palma de Mallorca
country: Spain
facility: H. Universitari Son Espases
state: Islas Baleares
city: Cartagena
country: Spain
facility: Hospital General Universitario Santa Lucia
state: Murcia
city: Vitoria
country: Spain
facility: H. Universitario de Araba
state: Pais Vasco
city: Burgos
country: Spain
facility: Hospital Universitario de Burgos
city: Madrid
country: Spain
facility: H. Universitario Fundacion Jimenez Diaz
Study officials:
affiliation: Takeda
name: Medical Director
role: STUDY_DIRECTOR
References: