Clinical trial NCT07008820: Patient Reported Outcomes and Patient Voice Among Patients Diagnosed With Low Risk Myelodysplastic Syndrome (LR-MDS) or Unexplained Anemia In Japan
Official title: Patient Reported Outcomes and Patient Voice Among Patients Diagnosed With Low Risk Myelodysplastic Syndrome (LR-MDS) or Unexplained Anemia In Japan
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Myelodysplastic Syndrome (MDS)
Keywords: Myelodysplastic Syndrome (MDS)
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2025-06-06
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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

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


Eligibility criteria: Inclusion Criteria:

The inclusion criteria for the quantitative phase will include either 1 or 2 and all of 3-6:

1. Low Risk Myelodysplastic Syndromes (LR MDS) erythropoietin stimulating agent (ESA) naïve non-transfusion dependent (NTD) patients: identified with confirmed via bone marrow aspirate and \< 5% blasts in bone marrow. Lower-risk is defined by International Prognostic Scoring System 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
2. Unexplained anemia with suspected MDS: identified anemia with general anemia (iron/vitamin deficiency, bleeding, renal, etc.) excluded
3. Patients with Hb \< 9.5 g/dl.
4. Patients who are ≥ 18 years of age at the time of signing the informed consent form.
5. Patients who are able and willing to provide informed consent.
6. Patients who are able to complete the protocol requirements.

For the qualitative phase (approximately 10 participants), the inclusion criteria will also include:

1. Patients with severity scores of "moderate" or greater on at least one Patient Global Impression-Severity (PGI-S) item.

Exclusion Criteria:

Patients meeting the following criteria will be excluded:

1. Patients who have difficulty obtaining informed consent or execution of this study because of insufficient Japanese language proficiency.
2. Patients who are ≧80 years of age at enrollment
3. Patients 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.
4. Patients 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.
5. Patients 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).
6. Patients 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).
7. Patients with a known history of diagnosis of acute myeloid leukemia.
8. Patients with major surgery within 8 weeks prior to enrollment. Patients must have completely recovered from any previous surgery prior to enrollment.
9. Patients with history of cerebrovascular accident (including ischemic, embolic, and hemorrhagic cerebrovascular accident), transient ischemic attack, deep venous thrombosis (including proximal and distal), pulmonary or arterial embolism, arterial thrombosis, or other venus thrombosis within 6 months prior to enrollment. Note: prior superficial thrombophlebitis is not an exclusion criterion.
10. Patients who have any condition or receive concomitant medication that confounds the ability to interpret data from the study.

Primary outcomes:
measure: 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
timeFrame: Day 1
measure: Participant anemia symptoms as assessed by the Functional Assessment of Cancer Therapy - Anemia (FACT-An) patient reported outcome questionnaire
timeFrame: Day 1
measure: Symptom severity as assessed by the Patient Global Impression-Severity (PGI-S) patient reported outcome questionnaire
timeFrame: Day 1
measure: General health status of participants as assessed by the EQ-5D-5L patient reported outcome questionnaire
timeFrame: Day 1
measure: Participant baseline socio-demographics
timeFrame: Baseline
measure: Participant baseline clinical characteristics
timeFrame: Baseline
Secondary outcomes:
measure: Qualitative interview results
timeFrame: Up to 1 month
Other outcomes:
Locations:
city: Minato-ku
country: Japan
facility: Local Institution - 0001
state: Tokyo
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol Myers Squibb
role: STUDY_DIRECTOR
References: