Patient Reported Outcomes and Patient Voice Among Patients Diagnosed With Low Risk Myelodysplastic Syndrome (LR-MDS) or Unexplained Anemia In Japan

NCT07008820 | Recruiting

• 1 other identifier: CA056-1133
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

This study will examine quality of life, experiences, and unmet needs among individuals diagnosed with Low Risk Myelodysplastic Syndrome who are erythropoietin stimulating agent naïve and non-transfusion dependent and among individuals with suspected myelodysplastic syndromes with unexplained anemia in Japan

Conditions

Myelodysplastic Syndrome (MDS)

Keywords

Myelodysplastic Syndrome (MDS)

Outcome Measures

Primary Outcomes (6)

• Participant quality of life as assessed by the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC-QLQ C30) patient reported outcome questionnaire

  Day 1

• Participant anemia symptoms as assessed by the Functional Assessment of Cancer Therapy - Anemia (FACT-An) patient reported outcome questionnaire

  Day 1

• Symptom severity as assessed by the Patient Global Impression-Severity (PGI-S) patient reported outcome questionnaire

  Day 1

• General health status of participants as assessed by the EQ-5D-5L patient reported outcome questionnaire

  Day 1

• Participant baseline socio-demographics

  Baseline

• Participant baseline clinical characteristics

  Baseline

Secondary Outcomes (1)

• Qualitative interview results

  Up to 1 month

Study Arms (2)

Cohort 1

Low Risk Myelodysplastic Syndrome erythropoietin stimulating agent naïve and non-transfusion dependent

Other: Health-related quality of life questionnaires; Other: Qualitative interviews

Cohort 2

Suspected Low Risk Myelodysplastic Syndrome with unexplained anemia

Other: Health-related quality of life questionnaires; Other: Qualitative interviews

Interventions

Health-related quality of life questionnaires OTHER

Questionnaires include: EORTC-QLQ C30 FACT-An PGI-S EQ-5D-5L

Cohort 1; Cohort 2

Qualitative interviews OTHER

For participants that meet eligibility criteria

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This study will include adult participants diagnosed with Low Risk Myelodysplastic Syndrome who are erythropoietin stimulating agent naïve and non-transfusion dependent or adults with suspected myelodysplastic syndromes with unexplained anemia in Japan

You may qualify if:

• Low Risk Myelodysplastic Syndromes (LR MDS) erythropoietin stimulating agent (ESA) naïve non-transfusion dependent (NTD) patrticipants: identified with confirmed via bone marrow aspirate and \< 5% blasts in bone marrow. Lower-risk is defined by International Prognostic Scoring System (IPSS) or Revised International Prognostic Scoring System (IPSS-R), as follows:
• Very low, low, or intermediate-risk (score ≤ 3.5) as assessed by IPSS-R
• Low or intermediate-1 (score ≤ 1) as assessed by IPSS
• Unexplained anemia with suspected MDS: identified anemia with general anemia (iron/vitamin deficiency, bleeding, renal, etc.) excluded
• Participants with hemoglobin in the most recent blood test \< 10.0 g/dl, or the average of hemoglobin \< 10.0g/dl in the most recent 2 blood tests conducted within 30 days prior to enrollment in this study
• Participants who are ≥ 18 years of age at the time of signing the informed consent form.
• Participants who are able and willing to provide informed consent.
• Participants who are able to complete the protocol requirements.
• \. Participants with severity scores of "moderate" or greater on at least one Patient Global Impression-Severity (PGI-S) item.

You may not qualify if:

• Patients meeting the following criteria will be excluded:
• Participants who have difficulty obtaining informed consent or execution of this study because of insufficient Japanese language proficiency.
• Participants who are considered to have difficulty answering HRQoL questionnaires and/or responding to questions during cognitive interview
• Participants who received red-blood cell transfusion (RBC-T) within 16 weeks prior to enrollment.
• Note: RBC-T of 1 to 2 units within the 16 weeks prior to enrollment are allowed, provided those 1-2 RBC-T units are administered for an acute event/illness (i.e., surgical procedure, bleeding, infection) or presence of comorbidity (including cardiovascular, pulmonary, cerebrovascular), and not for the treatment of low hemoglobin (with or without symptoms) alone.
• Participants who received prior drug treatment related to anemia; drugs include the following: erythropoiesis stimulating agent, erythroid maturation agent, hypomethylating agent, immunomodulatory drugs, immuno-suppressive agent.
• Participants with myelodysplastic/myeloproliferative neoplasms (MDS/MPN) according to World Health Organization (WHO) 2016 classification (i.e., chronic myelomonocytic leukemia, atypical chronic myeloid leukemia, breakpoint cluster region-Abelson 12, juvenile myelomonocytic leukemia, MDS/MPN unclassifiable).
• Participants with secondary MDS (i.e., MDS that is known to have arisen as a result of chemical injury or treatment with chemotherapy and/or radiation for other diseases).
• Participants with a known history of diagnosis of acute myeloid leukemia.
• Participants with major surgery within 8 weeks prior to enrollment. Patients must have completely recovered from any previous surgery prior to enrollment.
• Participants who have any condition or receive concomitant medication that confounds the ability to interpret data from the study.

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (1)

Mebix. Inc

Minato-ku, Tokyo, 1050001, Japan

RECRUITING

Related Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Bristol Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Central Study Contacts

BMS Study Connect Contact Center www.BMSStudyConnect.com

CONTACT

855-907-3286; Clinical.Trials@bms.com

First line of the email MUST contain NCT # and Site #

CONTACT

Study Design

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

Study Record Dates

• First Submitted: May 27, 2025
• First Posted: June 6, 2025
• Study Start: May 13, 2025
• Primary Completion: February 28, 2026
• Study Completion: February 28, 2026
• Last Updated: December 4, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

JP (1)