UMIN-CTR Clinical Trial

Public title

Prospective observational study to assess the prognosis of patients with plasma cell neoplasms in Japan

Acronym

Prospective observational study for MM in Japan (JSH-MM-15)

Scientific Title

Prospective observational study to assess the prognosis of patients with plasma cell neoplasms in Japan

Scientific Title:Acronym

Prospective observational study for MM in Japan (JSH-MM-15)

Region

Japan

Condition

Newly diagnosed plasma cell neoplasms

Classification by specialty

Hematology and clinical oncology

Classification by malignancy

Malignancy

Genomic information

NO

Narrative objectives1

The objective of this study is to figure out the prognosis and the prognostic factor in patients with plasma cell neoplasms.

Basic objectives2

Efficacy

Basic objectives -Others

Trial characteristics_1

Exploratory

Trial characteristics_2

Pragmatic

Developmental phase

Not applicable

Primary outcomes

3-year overall survival in patients with symptomatic myeloma who have received drug therapies in novel drug era

Key secondary outcomes

In patients with symptomatic myeloma who have received drug therapies:
overall response rate(ORR), progression-free survival(PFS), time to next treatment(TNT), treatment-free interval(TFI), overall survival(OS)
according to the difference of induction therapies, clinical stages, and cytogenetic risk groups
in addition to exploring the incidence of second primary malignancies(SPM) after initial treatment

In patients with plasma cell neoplasms except for symptomatic myeloma, systemic amyloidosis and POEMS syndrome, i.e., MGUS, smoldering myeloma, solitary plasmacytoma, multiple plasmacytoma and plasma cell leukemia:
overall survival(OS),
disease progression

Study type

Observational

Basic design

Randomization

Randomization unit

Blinding

Control

Stratification

Dynamic allocation

Institution consideration

Blocking

Concealment

No. of arms

Purpose of intervention

Type of intervention

Interventions/Control_1

Interventions/Control_2

Interventions/Control_3

Interventions/Control_4

Interventions/Control_5

Interventions/Control_6

Interventions/Control_7

Interventions/Control_8

Interventions/Control_9

Interventions/Control_10

Age-lower limit

Not applicable

Age-upper limit

Not applicable

Gender

Male and Female

Key inclusion criteria

1)diagnosed as having plasma cell neoplasms including MGUS, smoldering myeloma, symptomatic myeloma, non-secretary myeloma, solitary plasmacytoma of bone, extramedullary plasmacytoma based on International Myeloma Working Group(IMWG) criteria in 2014
2)newly diagnosed on January 1 in 2016 or later

Key exclusion criteria

none

Target sample size

1100

Name of lead principal investigator

1st name	Shinsuke
Middle name
Last name	Iida

Organization

Nagoya City University Graduate School of Medical Sciences

Division name

Hematology and Oncology

Zip code

467-8601

Address

1 Kawasumi,Mizuho-cho,Mizuho-ku,Nagoya City,Aichi 467-8601,Japan

TEL

052-853-8738

Email

iida@med.nagoya-cu.ac.jp

Name of contact person

1st name	Hirohiko
Middle name
Last name	Shibayama

Organization

National Hospital Organization Osaka National Hospital

Division name

Hematology

Zip code

540-0006

Address

2-1-14, Hoenzaka, Chuo-ku, Osaka City, Japan

TEL

06-6942-1331

Homepage URL

http://www.jshem.or.jp/shikkan/mm.html

Email

shibayama.hirohiko.ec@mail.hosp.go.jp

Institute

THE JAPANESE SOCIETY OF HEMATOLOGY

Institute

Department

Personal name

Organization

THE JAPANESE SOCIETY OF HEMATOLOGY

Organization

Division

Category of Funding Organization

Non profit foundation

Nationality of Funding Organization

Japan

Co-sponsor

Name of secondary funder(s)

Organization

Nagoya City University Graduate School of Medical Sciences and Nagoya City Hospital Institutional Review Board

Address

1, Kawasumi, Mizuho-cho, Mizuho-ku, Nagoya City, Aichi, Japan

Tel

052-858-7215

Email

clinical_research@med.nagoya-cu.ac.jp

Secondary IDs

NO

Study ID_1

Org. issuing International ID_1

Study ID_2

Org. issuing International ID_2

IND to MHLW

Institutions

Date of disclosure of the study information

2016

Year

05

Month

01

Day

URL releasing protocol

http://www.jshem.or.jp/shikkan/mm.html

Publication of results

Published

URL related to results and publications

https://pmc.ncbi.nlm.nih.gov/articles/PMC11136844/

Number of participants that the trial has enrolled

1951

Results

A prospective observational study of multiple myeloma diagnosed between 2016 and 2018 was conducted. Three year OS rate as a primary endpoint in 1284 patients was 70.0% [95%CI 67.4,72.6%]. It was 90.3% [95%CI 86.6,93.1%] in patients who received upfront autologous stem cell transplantation [ASCT], versus 61.4% [95%CI 58.0,64.6%] in those not receiving upfront ASCT.

Results date posted

2024

Year

11

Month

03

Day

Results Delayed

Results Delay Reason

Date of the first journal publication of results

Baseline Characteristics

The median age of the patients was 71 years (ranging from 33 to 96 years), and 1,044 (75.4%) patients were 65 years or older. Female patients constituted 47.7% of the cohort. Eastern Cooperative Oncology Group Performance Status (ECOG PS) scores were distributed as 78.1% for 0-2 and 21.9% for 3-4. Regarding the ISS, 20.1%, 35.8%, and 40.1% of patients were classified into stages 1, 2, and 3, respectively. The M-protein types were IgG in 55.9% of cases and non-IgG in 40.4%. Various symptoms were observed in the patients, with hypercalcemia in 13.0%, renal insufficiency in 25.3%, anemia in 61.4%, and bone diseases in 61.5% of cases. Accompanying symptoms such as AL amyloidosis, extramedullary tumors, and central nervous system (CNS) invasion were observed in 6.1%, 8.4%, and 0.6% of the patients, respectively. The distribution of Freiburg comorbidity index (FCI) scores was as follows: 61.7% had an FCI of 0, 29.2% had an FCI of 1, 7.9% had an FCI of 2, and 1.1% had an FCI of 3.

Participant flow

Between January 2016 and December 2018, a total of 1,951 patients with PCN were registered from 67 hospitals across Japan. After excluding 52 patients due to ineligibility or insufficient data, 1,899 patients were included in the analysis. Among them, 1,349 patients were diagnosed with symptomatic PCN and required some form of treatment, while the remaining 550 patients were initially diagnosed with non-symptomatic PCN. During the study period, 59 patients with non-symptomatic PCN progressed to symptomatic PCN, with 36 of them experiencing this progression by December 2018. Therefore, a total of 1,385 patients with symptomatic PCN (1,349 plus 36) were analyzed in this report. Among these patients, 1,274 were diagnosed with symptomatic multiple myeloma (MM), 14 with non-secretary MM, 70 with multiple plasmacytoma, and 27 with plasma cell leukemia (PCL).

Adverse events

No particular adverse event has been reported associated with this prospective observational study.

Outcome measures

The primary endpoint of this study was the 3-year overall survival (OS) of newly diagnosed patients with symptomatic PCN who were treated with systemic chemotherapy. Secondary endpoints included progression-free survival (PFS), time to next treatment (TNT), treatment-free interval (TFI), and the best overall response rates (ORR) based on the International Myeloma Working Group (IMWG) uniform response criteria as determined by the first-line treatment.

Plan to share IPD

IPD sharing Plan description

Recruitment status

Completed

Date of protocol fixation

2016

Year

03

Month

28

Day

Date of IRB

2016

Year

03

Month

28

Day

Anticipated trial start date

2016

Year

05

Month

01

Day

Last follow-up date

2022

Year

04

Month

30

Day

Date of closure to data entry

Date trial data considered complete

Date analysis concluded

Other related information

Prospective observational study
In patients with symptomatic myeloma, several factors such as age, clinical stage, cytogenetic risk group and difference of initial therapies are considered to influence on the efficacy endpoints such as overall survival and progression-free survival.
In patients with MGUS and asymptomatic myeloma, several factors such as paraprotein level, marrow plasma cell percentage, serum free light chain ratio are considered to influence on the disease progression.

Registered date

2016

Year

04

Month

27

Day

Last modified on

2024

Year

11

Month

03

Day

Link to view the page

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000025347