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
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 240
- 试验地点
- 14
- 主要终点
- PFS
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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 >=
- •>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.
- •Under treatment for MM or systemic AL amyloidosis at the time of study entry.
- •Have started treatment up to 12 months before inclusion for MM or systemic AL amyloidosis, irrespective of the treatment regimen.
- •Having first, second, third or fourth line of treatment for MM or systemic AL amyloidosis, irrespective of the treatment regimen.
- •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.
排除标准
- •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.
结局指标
主要结局
PFS
时间窗: 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)
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.
DoT
时间窗: From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year)
DoT is defined as the time between the start of a treatment/regimen and the end of that regimen.
次要结局
- 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))
- Percentage of Participants With More Than one Neoplasm(From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year))
- 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 Categorized Based on Clinical and Sociodemographic Characteristics Influencing the Treatment Prescribed and Satisfaction With Treatment(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))
- Percentage of Participants who Achieve the Prescribed DoT According to Treatment Strategy(From start date of treatment reported at study entry until discontinuation of treatment for any reason (approximately 1 year))